[{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Lonza Group","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Lonza Expands Partnership with Rocket Pharmaceuticals for Childhood Disorder Treatment","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Ideaya Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"IDEAYA Biosciences and Boston Children\\'s Hospital Collaborate on Preclinical Evaluation of IDE196 for Sturge Weber Syndrome - a Rare Disease Associated with Genetic Mutation of GNAQ","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed First to Advance Cannabinol (CBN) into Therapeutic Clinical Trials","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inovio Publishes Patient Benefit Results From A Pilot Clinical Study","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Vaccine","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Vaccine","graph2":"Phase I"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inovio Receives Authorization from FDA To Begin Trial for INO-3107","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Vaccine","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Vaccine","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Sanifit","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"First patient dosed in pivotal phase 3 trial of SNF472 in calciphylaxis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Announces Completed Enrollment in Phase 1 Clinical Trial of INM-755 CBN","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Submits Clinical Trial Application to Evaluate INM-755 in Second Phase 1 Trial","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Announces Completion of Initial Phase 1 Clinical Trial of INM-755 CBN Cream in Healthy Subjects","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I"},{"orgOrder":0,"company":"LifeMax Laboratories","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"LifeMax Receives Fast Track Designation For LM-030 for the Treatment of Netherton Syndrome","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Azitra","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Azitra Announces Rare Pediatric Disease Designation for ATR-12 for the Treatment of Netherton Syndrome","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Emendo Biotherapeutics","sponsor":"AnGes","pharmaFlowCategory":"D","amount":"$61.0 million","upfrontCash":"Undisclosed","newsHeadline":"Emendo Biotherapeutics Raises $61 Million to Advance Next Generation Genome Editing Therapeutics","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"January 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Undisclosed"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Announces Publication of Manuscript Evaluating Mosaicism in Fanconi Anemia","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Announces Science Translational Medicine Publication of its Program in Danon Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Presents Positive Updates on FA and LAD-I Gene Therapy Programs at the 23rd ASGCT Annual Meeting","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"AlgoTherapeutix","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AlgoTherapeutix Announces Orphan Drug Designation Granted by FDA for ATX01 in Erythromelalgia","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"FRANCE","productType":"Small molecule","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ProQR Announces Virtual Presentation of Phase 1\/2 Sepofarsen Data Through the Association for Research in Vision and Ophthalmology (ARVO)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Approves First Treatment for Adult Onset Still\u2019s Disease, a Severe and Rare Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"SWITZERLAND","productType":"Large molecule","productStatus":"Approved","date":"June 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Takeda Pharmaceutical","sponsor":"Carmine Therapeutics","pharmaFlowCategory":"D","amount":"$900.0 million","upfrontCash":"Undisclosed","newsHeadline":"Takeda Partners up with Carmine Therapeutics to Develop and Market Rare Disease Candidates","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"JAPAN","productType":"Cell and Gene therapy","productStatus":"Undisclosed","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Discovery"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Announces Dosing Initiation in 2nd Phase 1 Trial with INM-755","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Benitec Biopharma Provides Update on BB-301 Tissue Transduction Study","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"IND Enabling"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Announces First Patient Treated in Phase 1 Trial of RP-L301 Gene Therapy for Pyruvate Kinase Deficiency","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Benitec Begins Early Study of BB-301 Gene Therapy for OPMD","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"IND Enabling"},{"orgOrder":0,"company":"Protara Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Protara Therapeutics Receives Rare Pediatric Disease Designation for TARA-002 for the Treatment of Lymphatic Malformations","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"INOVIO Receives Orphan Drug Designation From for DNA Medicine INO-3107 To Treat Rare Disease Recurrent Respiratory Papillomatosis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Vaccine","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Vaccine","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Cerecor","sponsor":"Calidi Biotherapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Cerecor Receives Orphan Drug Designation for CERC-006 in Lymphatic Malformations","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Oncopeptides","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Oncopeptides Starts the First Study with Melflufen Outside Multiple Myeloma and Enrolls First Patient in Phase 1\/2 AL Amyloidosis Study","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"SWEDEN","productType":"Peptide","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Peptide","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Receives FDA Fast Track Designation for RP-L401 Gene Therapy for Infantile Malignant Osteopetrosis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Announces First Patient Treated in Higher Dose Cohort in Phase 1 Clinical Trial of RP-A501 for Danon Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I"},{"orgOrder":0,"company":"Protara Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Protara Therapeutics Provides Comparability and U.S. Regulatory Updates for TARA-002 to Advance in Oncology and Rare Disease Indications","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AMRYT Announces Positive Top Line Results from Pivotal Phase 3 \"ease\" Trial of Filsuvez\u00ae in Epidermolysis Bullosa","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Raziel Therapeutics","sponsor":"Fosun Pharmaceutical","pharmaFlowCategory":"D","amount":"$74.0 million","upfrontCash":"$27.0 million","newsHeadline":"Raziel Announces China Market License Agreement with Fosun Pharma for RZL 012","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"ISRAEL","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Protara Therapeutics","sponsor":"Cowen","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Protara Therapeutics Announces Proposed Concurrent Public Offerings of Common Stock and Preferred Stock","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Announces Completion of Subject Treatments in Second Phase 1 Clinical Trial of INM-755 CBN Cream in Healthy Subjects","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants AT-007 Pediatric Rare Disease Designation and Orphan Designation for Treatment of PMM2-CDG","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Presents Positive LAD-l Clinical Update and Comprehensive IMO Preclinical Review at the ESID 2020 Meeting","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Sanifit","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sanifit Presents New Data for SNF472 in Treatment of Vascular Calcification at ASN Kidney Week 2020","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Amryt Announces Positive Results from Phase 3 Trial of Filsuvez\u00ae in Epidermolysis Bullosa","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"GSK","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GlaxoSmithKline's Nucala Filings Accepted by European Medicines Agency for Three Additional Eosinophil-Driven Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"Approved","date":"October 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"XOMA","sponsor":"Chiesi Group","pharmaFlowCategory":"D","amount":"$150.0 million","upfrontCash":"$1.2 million","newsHeadline":"XOMA Acquires Royalty Interest in Four Lysosomal Storage Disorder Enzymes Being Developed Under the Chiesi Group\u2013Bioasis Strategic Alliance","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Large molecule","productStatus":"Undisclosed","date":"November 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Undisclosed"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ProQR Announces Virtual Presentations at American Academy of Ophthalmology (AAO)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals to Present Data from its Fanconi Anemia, Leukocyte Adhesion Deficiency-I and Pyruvate Kinase Deficiency Programs at the 62nd American Society of Hematology Annual Meeting","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Roth Capital Partners","pharmaFlowCategory":"D","amount":"$8.0 million","upfrontCash":"Undisclosed","newsHeadline":"InMed Pharmaceuticals Announces the Closing of US$8 Million Public Offering","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"California Institute for Regenerative Medicine","pharmaFlowCategory":"D","amount":"$3.7 million","upfrontCash":"Undisclosed","newsHeadline":"Rocket Pharma Gets Funding from the California Institute for Regenerative Medicine for Phase 1 Clinical Trial of RP-L401 for Infantile Malignant Osteopetrosis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I"},{"orgOrder":0,"company":"University of Oxford","sponsor":"Evox Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Evox Therapeutics Announces Launch of Strategic Collaboration with the University of Oxford","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"UNITED KINGDOM","productType":"Undisclosed","productStatus":"Undisclosed","date":"November 2020","url1":"","url2":"","graph1":"Undisclosed","graph2":"Discovery"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"INOVIO Announces Dosing of First Subject in Phase 1\/2 Clinical Trial for INO-3107","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Saniona Reports Positive Topline Results from Tesomet Phase 2 Open-Label Extension Study in Hypothalamic Obesity","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios' Phase 3 ACTIVATE Trial of Mitapivat Met Primary Endpoint in Adults with Pyruvate Kinase Deficiency Who Are Not Regularly Transfused","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Sofinnova Investments","pharmaFlowCategory":"D","amount":"$55.0 million","upfrontCash":"Undisclosed","newsHeadline":"Atsena Therapeutics Raises $55 Million Series A Financing to Advance LCA1 Gene Therapy Clinical Program","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Adrestia Therapeutics","sponsor":"GSK","pharmaFlowCategory":"D","amount":"$1,150.0 million","upfrontCash":"Undisclosed","newsHeadline":"GSK and Ahren Announce Co-Led Series A Investment in Adrestia, and Multi-Project Discovery Collaboration for Five Projects","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery Platform","country":"UNITED KINGDOM","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Undisclosed","graph2":"Discovery Platform"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Stealth BioTherapeutics Granted Pre-NDA Meeting For Elamipretide as a Treatment For Cardiomyopathy In Barth Syndrome","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Peptide","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ProQR Completes Enrollment of its Pivotal Trial of Sepofarsen for the Treatment of LCA10","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios Announces Phase 3 ACTIVATE-T Trial of Mitapivat Achieved Primary Endpoint in Adults with Pyruvate Kinase Deficiency","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"H.C. Wainwright & Co","pharmaFlowCategory":"D","amount":"$4.7 million","upfrontCash":"Undisclosed","newsHeadline":"Stealth BioTherapeutics Announces Pricing Of $4.7 Million Registered Direct Offering Of American Depositary Shares","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Peptide","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Ensoma","sponsor":"5AM Ventures","pharmaFlowCategory":"D","amount":"$70.0 million","upfrontCash":"Undisclosed","newsHeadline":"Ensoma Launches to Pioneer Next-Generation In Vivo Approach to Deliver First \u201cOff-the-shelf\u201d Genomic Medicines","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"D","amount":"$4.0 million","upfrontCash":"Undisclosed","newsHeadline":"InMed Announces Closing of US$4.5 Million Private Placement","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"February 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I"},{"orgOrder":0,"company":"Novadip Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Novadip Biosciences receives IND approval from the FDA for regenerative bone product NVD-003","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"IND Enabling","country":"BELGIUM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"IND Enabling"},{"orgOrder":0,"company":"Precigen","sponsor":"National Institutes of Health","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Precigen Receives FDA Orphan Drug Designation for PRGN-2012 AdenoVerse\u2122 Immunotherapy for Recurrent Respiratory Papillomatosis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Vaccine","productStatus":"New Molecular Entity","date":"March 2021","url1":"","url2":"","graph1":"Vaccine","graph2":"Phase I"},{"orgOrder":0,"company":"Swedish Orphan Biovitrum AB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"NICE Issues Positive Recommendation for Use of Kineret to Treat Still\u2019s Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"SWEDEN","productType":"Large molecule","productStatus":"Approved","date":"March 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"N-Lorem Foundation Partners with Ultragenyx to Bolster Development of Personalized Medicines for Patients with Ultra-Rare Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Large molecule","productStatus":"Undisclosed","date":"April 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Discovery"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ProQR Announces Virtual Presentation at American Association for Pediatric Ophthalmology and Strabismus (AAPOS) 2021","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"NETHERLANDS","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"AGC Biologics","sponsor":"Rocket Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"AGC Biologics Expands Partnership with Rocket Pharmaceuticals","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II"},{"orgOrder":0,"company":"Allen Institute","sponsor":"BioMarin Pharmaceutical","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Allen Institute and BioMarin Team Up To Develop Gene Therapies For Rare Brain Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Undisclosed","country":"","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Undisclosed"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Pharma Submits Clinical Trial Applications to Evaluate INM-755 (Cannabinol) Cream in a Phase 2 Trial for Epidermolysis Bullosa","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"April 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ilaris\u00ae Receives Health Canada Approval as First of Its Kind Treatment for Rare, Inflammatory Disorder, Adult-Onset Still's Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"SWITZERLAND","productType":"Large molecule","productStatus":"Approved","date":"April 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Affinia Therapeutics","sponsor":"EcoR1 Capital","pharmaFlowCategory":"D","amount":"$110.0 million","upfrontCash":"Undisclosed","newsHeadline":"Affinia Therapeutics Closes $110 Million Series B Financing","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Saniona to Host Research and Development (R&D) Day on Leveraging Ion Channel Targeting Expertise for Rare Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Strongbridge Biopharma","sponsor":"Xeris Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Xeris Pharmaceuticals, Inc. to Acquire Strongbridge Biopharma in Stock and CVR Transaction","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"May 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Disc medicine","pharmaFlowCategory":"D","amount":"$200.0 million","upfrontCash":"Undisclosed","newsHeadline":"Disc Medicine Expands Hematology Pipeline with Worldwide Licensing Agreement for Bitopertin, a First-in-Class Modulator of Heme Synthesis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"SWITZERLAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Saniona Hosts Research and Development Day on Proprietary Ion Channel Drug Discovery Engine and Pipeline Programs","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Priority Review for New Drug Application for Oleogel-S10 for the Treatment of Epidermolysis Bullosa","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"IRELAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Confirms NDA for Oleogel-S10 Will not Require an Advisory Committee Meeting","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Baymedica","sponsor":"InMed Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"InMed Pharmaceuticals Signs Non-Binding Letter of Intent to Acquire BayMedica, a Commercial Manufacturer of Rare Cannabinoids","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"H.C. Wainwright & Co.","pharmaFlowCategory":"D","amount":"$12.0 million","upfrontCash":"Undisclosed","newsHeadline":"InMed Pharmaceuticals Announces $12 Million Private Placement Priced At-the-Market under Nasdaq Rules","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Glycomine","sponsor":"ImmuneID","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Glycomine Closes $68 Million Series B to Advance into Clinical Trials a Novel Treatment for PMM2-CDG, a Rare Glycosylation Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Saniona Initiates Phase 1 Clinical Trial of SAN711","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ReCode to Present Preclinical Data from mRNA-Based Program for Primary Ciliary Dyskinesia at the Move Virtual Scientific Conference","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Beckley Psytech","sponsor":"Integrated","pharmaFlowCategory":"D","amount":"$80.0 million","upfrontCash":"Undisclosed","newsHeadline":"Beckley Psytech Completes Oversubscribed $80m (\u00a358m) Fundraise to Develop Portfolio of Psychedelic Medicine Breakthroughs","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I"},{"orgOrder":0,"company":"Disc medicine","sponsor":"OrbiMed","pharmaFlowCategory":"D","amount":"$90.0 million","upfrontCash":"Undisclosed","newsHeadline":"Disc Medicine Announces $90 Million Series B Financing to Advance Portfolio of Clinical-Stage Therapies for Hematologic Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Institute for Life Changing Medicines","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Moderna and The Institute for Life Changing Medicines Announce a New Collaboration to Develop an mRNA Therapeutic","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"RD Fund","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"RD Fund Launches Opus Genetics with $19M Seed Funding to Advance Gene Therapy Treatments for Blinding Conditions","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Catalyst Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Catalyst Biosciences Orphan Drug Designation for MarzAA for the Treatment of Factor VII Deficiency","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Results Show Long-Lasting and Holistic Improvements in Children with AADC Deficiency Treated with PTC-AADC Gene Therapy","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II"},{"orgOrder":0,"company":"Retrotope","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Retrotope Reports Data from Phase 2\/3 Clinical Trial of RT001 and Concurrent Natural History Study in Patients with Infantile Neuroaxonal Dystrophy (INAD)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Enzyvant","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Enzyvant Receives FDA Approval for RETHYMIC\u00ae (allogeneic processed thymus tissue-agdc), a One-Time Regenerative Tissue-Based Therapy for Pediatric Congenital Athymia","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"October 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Kedrion","sponsor":"Kedrion","pharmaFlowCategory":"D","amount":"$17.0 million","upfrontCash":"Undisclosed","newsHeadline":"Kedrion Biopharma Grows in North America As it Completes Acquisition of Prometic","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"October 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Kedrion","sponsor":"Kedrion","pharmaFlowCategory":"D","amount":"$17.0 million","upfrontCash":"Undisclosed","newsHeadline":"Kedrion enters into Agreement with Liminal BioSciences","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"May 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"ChromaDex, Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ChromaDex Shares Findings from First Clinical Study on Nicotinamide Riboside (NR) in Children, Highlighting Improvements for Prematurely Aging Patients with Ataxia-Telangiectasia (AT)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"October 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"AFT Pharmaceuticals","sponsor":"Quoin Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Quoin Pharmaceuticals Signs Exclusive Licensing and Distribution Agreement with AFT Pharmaceuticals for its Lead Asset, QRX003","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"NEW ZEALAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Genpharm","sponsor":"Quoin Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Quoin Pharmaceuticals and Genpharm Services Sign Exclusive License and Distribution Agreement for Quoin\u2019s Lead Asset, QRX003","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.A.E","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"InveniAI","sponsor":"Bristol Myers Squibb","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Relief Therapeutics and InveniAI Sign a Strategic Collaboration Agreement to Identify New Product Development Opportunities using Artificial Intelligence","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Undisclosed","productStatus":"Undisclosed","date":"November 2021","url1":"","url2":"","graph1":"Undisclosed","graph2":"Discovery"},{"orgOrder":0,"company":"GSK","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"European Commission Approves Nucala (Mepolizumab) in Three Additional Eosinophil-Driven Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"Approved","date":"November 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"New Analysis Published in Multiple Sclerosis and Related Disorders Supports the Efficacy of UPLIZNA\u00ae (Inebilizumab-cdon) in Patients Previously Treated with Rituximab for Neuromyelitis Optica Spectrum Disorder (NMOSD)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Large molecule","productStatus":"Approved","date":"November 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Orpharm","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Quoin Pharmaceuticals Signs Exclusive Distribution Agreement with Orpharm LLC for its Lead Asset, QRX003, for Netherton Syndrome","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Adverum Biotechnologies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Adverum Biotechnologies Granted Orphan Drug Designation by FDA for Gene Therapy Candidate in Preclinical Development for the Treatment of Blue Cone Monochromacy","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Amryt Announces Oleogel-S10 EMA Regulatory Update","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"January 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Argonaut Manufacturing Services","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"n-Lorem Foundation Partners with Argonaut to Manufacture ASO Medicines for Patients with Nano-rare Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Undisclosed"},{"orgOrder":0,"company":"Hemab Therapeutics","sponsor":"Haemnet","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Hemab Therapeutics Announces First Drug Candidate: HMB-001, a Novel Bispecific Antibody with Potential for Treatment of Rare Bleeding Disorders","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"DENMARK","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Akouos","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Akouos Presents Nonclinical Data Demonstrating Potential Applicability of Precision Genetic Medicine Platform for a Broad Range of Inner Ear Conditions at the Association for Research in Otolaryngology 45th Annual Mid-Winter Meeting","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Cytiva","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"n-Lorem Foundation Partners with Cytiva to Bring Individualized ASO Medicines to Patients with Nano-rare Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Discovery"},{"orgOrder":0,"company":"Erkim","sponsor":"Quoin Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Quoin Pharmaceuticals Signs Exclusive Distribution Agreement with ER-Kim for its Lead Asset, QRX003, for Netherton Syndrome","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"TURKEY","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ProQR Announces Top-Line Results from Phase 2\/3 Illuminate Trial of Sepofarsen in CEP290-mediated LCA10","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"NETHERLANDS","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Saniona Initiates Multiple Ascending Dose Stage of SAN711 Phase 1 Clinical Trial","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Neopharma","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Quoin Pharmaceuticals Signs Exclusive Distribution Agreement with Neopharm for its Lead Asset, QRX003, for Netherton Syndrome","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"TargED Biopharmaceuticals","sponsor":"Andera Partners","pharmaFlowCategory":"D","amount":"$43.7 million","upfrontCash":"Undisclosed","newsHeadline":"TargED Biopharmaceuticals Raises EUR 39 Million in Series A Financing to Bring Groundbreaking Thrombolytic Treatment to Patients","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"NETHERLANDS","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Foresee Pharmaceuticals","sponsor":"TRPharm","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Foresee Pharmaceuticals and TRPharm Announce License and Co-Development Agreement","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Parexel Biotech","sponsor":"n-Lorem Foundation","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Parexel and n-Lorem Foundation Partner to Support Development of New Investigational Therapies for Nano-rare Patients","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Discovery"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Amryt Receives Complete Response Letter from the FDA for Oleogel-S10 NDA","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"February 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Erkim","sponsor":"Quoin Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Quoin Pharmaceuticals Extends Exclusive Distribution Agreement with ER-Kim for its Lead Asset, QRX003, for Netherton Syndrome","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"TURKEY","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"InnoCare Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InnoCare Announces Approval of Phase II Clinical Trial Using Orelabrutinib for the Treatment of NMOSD in China","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"IND Enabling","country":"CHINA","productType":"Small molecule","productStatus":"Approved","date":"March 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"IND Enabling"},{"orgOrder":0,"company":"Creyon Bio","sponsor":"DCVC Bio","pharmaFlowCategory":"D","amount":"$40.0 million","upfrontCash":"Undisclosed","newsHeadline":"Creyon Bio Launches to Engineer Safe, Effective Precision Medicines on Demand for All Patients","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Discovery"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"BridgeBio Pharma and Sentynl Therapeutics Announce Asset Purchase Agreement for BridgeBio Pharma\u2019s NULIBRY\u2122 (Fosdenopterin)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"March 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"Xentria","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Xentria to Present Initial Findings from Preclinical Study of XTMAB-16 at ATS 2022 International Conference","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I"},{"orgOrder":0,"company":"n-Lorem Foundation","sponsor":"The Conrad Prebys Foundation","pharmaFlowCategory":"D","amount":"$0.3 million","upfrontCash":"Undisclosed","newsHeadline":"The Conrad Prebys Foundation to Support the Discovery and Development of Personalized ASO Medicines for Nano-rare Patients","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Discovery"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Quoin Pharmaceuticals Announces Submission of IND for its Lead Asset, QRX003, for Netherton Syndrome","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"IND Enabling"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Marinus Pharmaceuticals Announces FDA Approval of ZTALMY\u00ae (ganaxolone) for CDKL5 Deciency Disorder","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"March 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"University of the Witwatersrand","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Cyclo Therapeutics Enters Research and Collaboration Agreement with Wits Commercial Enterprise (Pty) Ltd to Explore Potential Applications of Trappsol\u00ae Cyclo\u2122 to Expand Pipeline","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"March 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Azafaros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Azafaros Announces FDA Grant of Orphan Drug Designation for AZ-3102 in the Treatment of Niemann-Pick Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"First Patient Dosed in CAN106 Phase 1b\/2 Trial for Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in China","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Xenpozyme\u00ae (olipudase alfa), Approved in Japan, First and Only Approved Therapy Indicated to Treat Acid Sphingomyelinase Deficiency","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"FRANCE","productType":"Large molecule","productStatus":"Approved","date":"March 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Abionyx Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ABIONYX Pharma Receives FDA Orphan Drug Designation (ODD) for CER-001 for the Treatment of LCAT Deficiency Presenting as Kidney Dysfunction and\/or Ophthalmologic Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"New Analysis Finds UPLIZNA\u00ae (inebilizumab-cdon) Effective for People with Newly-Presenting Neuromyelitis Optica Spectrum Disorder (NMOSD)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Large molecule","productStatus":"Approved","date":"March 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Nobelpharma America","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Approves Nobelpharma's HYFTOR\u2122 (sirolimus topical gel) 0.2%","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"April 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA approves Novartis Vijoice\u00ae (alpelisib) as First and Only Treatment for Select Patients With PIK3CA-Related Overgrowth Spectrum (PROS)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"April 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Editas Medicine Announces Dosing of First Pediatric Patient in the BRILLIANCE Clinical Trial of EDIT-101 for LCA10","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Aeterna Zentaris","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Aeterna Zentaris Abstract Accepted for Presentation at 13th International Congress on Autoimmunity","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Peptide","graph2":"Preclinical"},{"orgOrder":0,"company":"Marinus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Marinus Pharmaceuticals Announces Publication in The Lancet Neurology of ZTALMY\u00ae (ganaxolone) Phase 3 Marigold Trial Results","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"April 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PTC Therapeutics Provides Update on EMA Regulatory Review Timeline For Its AADC Deficiency Gene Therapy Program","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II"},{"orgOrder":0,"company":"Octantbio","sponsor":"Catalio Capital Management","pharmaFlowCategory":"D","amount":"$80.0 million","upfrontCash":"Undisclosed","newsHeadline":"Octant Raises $80 Million Series B Round to Support Expansion of Next-Generation Drug Discovery Platform, Announces Collaboration with Bristol Myers Squibb and Key Appointments","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Discovery"},{"orgOrder":0,"company":"Savara","sponsor":"Silicon Valley Bank","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Savara Announces Debt Refinancing of $26.5M Credit Facility With Silicon Valley Bank","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"April 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Modalis Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Modalis Therapeutics to Present Data Supporting of Development of Transformative Epigenetic Modulating Medicines for the Treatment of a Type of Muscular Dystrophy and the Other Genetic Disorders at the ASGCT Annual Meeting","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CHMP Adopts Positive Opinion for Filsuvez for the Treatment of Dystrophic and Junctional EB","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"April 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"University of L\u2019Aquila","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"InMed Expands Patent Portfolio with Novel Cannabinoid Analogs and Advances Collaboration Agreement with Leading Cannabinoid Research Expert","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"April 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Quoin Pharmaceuticals Announces FDA Clearance of IND for its Lead Asset, QRX003, for Netherton Syndrome","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"IND Enabling"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Taysha Gene Therapies Announces Oral Presentations at the Upcoming 2022 IRSF Rett Syndrome Scientific Symposium and the ASCEND National Summit","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ProQR to Present at the Retinal Cell and Gene Therapy Innovation Summit and the Association for Research in Vision and Ophthalmology (ARVO) 2022","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"NETHERLANDS","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Anavex Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Anavex Life Sciences Announces Presentations at the IRSF Rett Syndrome Scientific Meeting","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Horizon Therapeutics plc Receives European Commission (EC) Approval of UPLIZNA\u00ae (inebilizumab) for the Treatment of Adults With Neuromyelitis Optica Spectrum Disorder (NMOSD)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Large molecule","productStatus":"Approved","date":"May 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ReCode Therapeutics to Present Preclinical Data Using Proprietary SORT LNP Delivery Platform From Its mRNA-based Therapeutic Programs at the ATS 2022 International Conference in San Francisco","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultomiris Met Primary Endpoint in CHAMPION-NMOSD Phase III Trial in Adults With Neuromyelitis Optica Spectrum Disorder","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"Approved","date":"May 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Protara Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Protara Therapeutics Presents Results from Retrospective Analysis of Randomized and Open-Label Studies Evaluating the Safety and Efficacy of OK-432 in Patients with Lymphatic Malformations","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Orphazyme","sponsor":"KemPharm","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"$12.8 million","newsHeadline":"KemPharm Announces Strategic Acquisition of Arimoclomol from Orphazyme, Expanding its Rare CNS Diseases Pipeline","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"ASC Therapeutics","sponsor":"UMass Chan Medical School","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ASC Therapeutics, U Mass Medical School, and the Clinic for Special Children Announce Podium Presentation of Safety and Efficacy in Murine and Bovine Models for Novel Gene Therapy in Maple Syrup Urine Disease at the 25th ASGCT Meeting","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Affinia Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Affinia Therapeutics Presents New Data at American Society of Gene and Cell Therapy Annual Meeting","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Capsida","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Capsida Biotherapeutics Presents Data Demonstrating Unique Industry-Leading Technology and Capabilities With Potential to Treat Both Rare and Common Diseases at the Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery Platform","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Discovery Platform"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Editas Medicine Presents Data on SLEEK Gene Editing Technology at the American Society of Gene and Cell Therapy Annual Meeting","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CHMP Recommends Approval of Xenpozyme\u00ae (Olipudase Alfa), the First and Only Treatment for ASMD","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"FRANCE","productType":"Large molecule","productStatus":"Approved","date":"May 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PTC Therapeutics Receives Positive CHMP Opinion for Upstaza\u2122 for the Treatment of AADC Deficiency","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"EMA Accepts Regulatory Submission for Olipudase Alfa, the First Potential Therapy for ASMD","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"FRANCE","productType":"Large molecule","productStatus":"Approved","date":"December 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"DepYmed","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"DepYmed Receives FDA Orphan Drug Designation for Clinical Candidate DPM-1001 for the Treatment of Wilson Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"IND Enabling"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Pharmaceuticals Announces Commencement of Phase 2 Clinical Trial Investigating Cannabinol (CBN), a Rare Cannabinoid, in the Treatment of Epidermolysis Bullosa","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"September 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CANbridge CAN106 Phase 1 Data Presented at the European Hematology Association 2022 Congress","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I"},{"orgOrder":0,"company":"Hong Kong Winhealth Pharma Group","sponsor":"Quoin Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Quoin Pharmaceuticals Announces Licensing and Distribution Agreement with Leading Rare Disease Company for Greater China","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"IND Enabling","country":"HONG KONG","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"IND Enabling"},{"orgOrder":0,"company":"Cresence","sponsor":"Bioasis","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Bioasis Acquires Phase 2 Ready Rare Orphan EGF Assets","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"IND Enabling","country":"NORWAY","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"IND Enabling"},{"orgOrder":0,"company":"ResVita Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ResVita Bio Receives Rare Pediatric Disease Designation for RVB-001 as a Treatment for Netherton Syndrome","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Undisclosed","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Undisclosed"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Venthera","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BridgeBio Pharma Affiliate Venthera Presents Preliminary Results from Clinical Trial of VT30 (BBP-681) for Venous, Lymphatic, and Venolymphatic Malformations","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Aeterna Zentaris","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Aeterna Zentaris Achieves Proof-of-Concept for the Treatment of NMOSD with AIM Biologicals Program","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Savara","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Savara Awarded Innovation Passport in United Kingdom (UK) for Molgramostim Nebulizer Solution (Molgramostim), a Novel Investigational Inhaled Biologic","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Zenyaku Kogyo","sponsor":"Chugai Pharmaceutical","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Anti-CD20 Monoclonal Antibody Rituxan Approved for the Prevention of Recurrence of Neuromyelitis Optica Spectrum Disorder","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"JAPAN","productType":"Large molecule","productStatus":"Approved","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Generian Pharmaceuticals","sponsor":"Mitobridge","pharmaFlowCategory":"D","amount":"$180.0 million","upfrontCash":"Undisclosed","newsHeadline":"Generian and Astellas Enter into Collaboration and Exclusive License Agreement to Discover and Develop Novel Small Molecules For Undruggable Targets","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery Platform","country":"U.S.A","productType":"Small molecule","productStatus":"Undisclosed","date":"June 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Discovery Platform"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Leaps by Bayer","pharmaFlowCategory":"D","amount":"$200.0 million","upfrontCash":"Undisclosed","newsHeadline":"ReCode Therapeutics to Expand Next-Generation Delivery Platform and Diversify Genetic Medicines Pipeline with Oversubscribed Series B Financing Totaling $200M","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Cyclo Therapeutics to Present at the 2022 NPC Patient and Family Conference Hosted by the Australian NPC Disease Foundation","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"European Commission Approves Filsuvez\u00ae for the treatment of Dystrophic and Junctional EB","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"June 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Xenpozyme\u00ae (olipudase alfa) Approved by European Commission as First and Only treatment for ASMD","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"FRANCE","productType":"Large molecule","productStatus":"Approved","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Cyclerion Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Cyclerion Therapeutics Provides Additional Positive CY6463 MELAS Clinical Data at a Webinar Hosted by United Mitochondrial Disease Foundation","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Quoin Pharmaceuticals Announces Opening of First Clinical Site for Netherton Syndrome Clinical Study","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"IND Enabling"},{"orgOrder":0,"company":"InflaRx","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InflaRx Receives FDA Fast Track Designation for Treatment of Ulcerative Pyoderma Gangrenosum","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"GERMANY","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Amryt Presents New Analyses from the EASE Phase 3 Trial in Epidermolysis Bullosa at SPD 2022","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"July 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Kyowa Kirin","pharmaFlowCategory":"D","amount":"$500.0 million","upfrontCash":"Undisclosed","newsHeadline":"Ultragenyx Announces Sale of a Portion of Future North American Royalties on Crysvita (burosumab) for $500 Million to OMERS Capital Markets","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"July 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Endo Ventures Limited","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Endo Announces Agreement for Paladin Labs to Develop, Commercialize and Supply Quoin Pharmaceuticals' QRX003 in Canada","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"IND Enabling"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Upstaza\u2122 Granted Marketing Authorization by European Commission as First Disease-Modifying Treatment for AADC Deficiency","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Approved","date":"July 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Approved"},{"orgOrder":0,"company":"Strm.Bio","sponsor":"National Center for Advancing Translational Sciences","pharmaFlowCategory":"D","amount":"$2.1 million","upfrontCash":"Undisclosed","newsHeadline":"STRM.BIO Receives $2.1 Million SBIR Grant to Advance Extracellular Vesicle Technology for Non-Viral, In Vivo Delivery of Gene Therapies","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Nobelpharma America","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Nobelpharma America Debuts New Product, HYFTOR\u2122 (sirolimus topical gel) 0.2%, at the 2022 World TSC Conference","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"July 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Announces Update on Phase 2 Clinical Trial Investigating INM-755 Cannabinol Cream for Epidermolysis Bullosa","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"July 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BridgeBio Pharma and Sentynl Therapeutics Receive Positive CHMP Opinion for NULIBRY (fosdenopterin) for the Treatment of MoCD Type A","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"July 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"Kira Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Kira Pharmaceuticals Receives FDA Orphan Drug Designation for KP104, a Bifunctional Antibody Fusion Protein, for the Treatment of Paroxysmal Nocturnal Hemoglobinuria","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Relief Therapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Acer Therapeutics and Relief Therapeutics Announce ACER-001 IND Submission for the Treatment of Maple Syrup Urine Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"July 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Quoin Pharmaceuticals Announces Receipt of Constructive Scientific Advice from EMA for QRX003 Development in Europe","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"IND Enabling"},{"orgOrder":0,"company":"Omeros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Orphan Drug Designation to Omeros\u2019 MASP-3 Inhibitor OMS906 for Treatment of Paroxysmal Nocturnal Hemoglobinuria","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I"},{"orgOrder":0,"company":"Epigenic Therapeutics","sponsor":"Morningside Venture Capital","pharmaFlowCategory":"D","amount":"$20.0 million","upfrontCash":"Undisclosed","newsHeadline":"Epigenic Therapeutics Raises $20 Million in Series Angel and Pre-A Funding to Advance Next Generation Gene Editing Therapy","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Preclinical"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pharming Group Receives Accelerated Assessment in Europe for leniolisib for the Treatment of Rare Immunodeficiency, APDS","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"ImmunAbs","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ImmunAbs Inc. Announces US FDA Approval for a Phase 1 Clinical Trial","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"IND Enabling","country":"SOUTH KOREA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"IND Enabling"},{"orgOrder":0,"company":"Resilience","sponsor":"Mayo Clinic","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Resilience Announces Collaboration to Manufacture and Deliver Biotherapeutics for Rare and Complex Conditions","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"Undisclosed","date":"August 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Agios Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Agios Announces Publication of Phase 2 Data in The Lancet Demonstrating Safety and Efficacy of PYRUKYND\u00ae (mitapivat) in Non-transfusion-dependent \u03b1- and \u03b2-Thalassemia","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"August 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Acer Therapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Relief Therapeutics and Acer Therapeutics Announce that the European Commission Has Granted Orphan Drug Designation for ACER-001 in Maple Syrup Urine Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"August 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Chugai Pharmaceutical","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Chugai Gets Priority Review for PNH Med Crovalimab in China","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"IASO Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"World's First CAR-T for NMOSD Treatment, IASO Biotherapeutics' Equecabtagene Autoleucel, Receives IND Approval by NMPA","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"IND Enabling","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"IND Enabling"},{"orgOrder":0,"company":"ProQR Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ProQR to Focus Exclusively on Axiomer RNA-editing Technology and Partner Ophthalmology Programs","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"NETHERLANDS","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Amgen Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Amgen Announces Positive Top-line Results From Phase 3 Study of ABP959, Biosimilar Candidate to Soliris\u00ae (Eculizumab)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Biosimilar","date":"August 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Kira Pharmaceuticals","sponsor":"Perelman School of Medicine","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Kira Pharmaceuticals to Present New Preclinical Data on Lead Asset KP104 at the 2022 European Meeting on Complement in Human Disease Conference","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Sanofi","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Xenpozyme TM (Olipudase Alfa-rpcp) Approved by FDA as First Disease-specific Treatment for ASMD (Non-CNS Manifestations)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"FRANCE","productType":"Large molecule","productStatus":"Approved","date":"August 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Nobelpharma America","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Hyftor\u2122 (Sirolimus Topical Gel) 0.2%, the First FDA-approved Topical Treatment for Facial Angiofibroma Associated With Tuberous Sclerosis, is Now Available in the US","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"August 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"Savara","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Savara Presented Data at the European Respiratory Society (ERS) International Congress 2022","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"September 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"JMP Securities","pharmaFlowCategory":"D","amount":"$18.0 million","upfrontCash":"Undisclosed","newsHeadline":"Benitec Biopharma Inc. Announces Pricing of $18 Million Underwritten Public Offering","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"IND Enabling"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"H.C. Wainwright & Co.","pharmaFlowCategory":"D","amount":"$6.0 million","upfrontCash":"Undisclosed","newsHeadline":"InMed Pharmaceuticals Announces Closing of $6 Million Private Placement Priced at a Premium to Market","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Nacuity Pharmaceuticals","sponsor":"Arctic Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Nacuity Pharmaceuticals and Arctic Therapeutics Enter Licensing Agreement and Strategic Partnership to Develop NPI-001","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"KBio","sponsor":"ZabBio","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"KBio Announces Strategic Partnership with ZabBio to Discover and Develop Antibody Therapeutics","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Discovery"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Baymedica","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Pharmaceuticals Provides Update on its Core Research and Development Programs and BayMedica Business","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"JMP Securities","pharmaFlowCategory":"D","amount":"$18.0 million","upfrontCash":"Undisclosed","newsHeadline":"Benitec Biopharma Inc. Announces Closing of $18 Million Underwritten Public Offering","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"IND Enabling","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"IND Enabling"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Sentynl Therapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BridgeBio Pharma and Sentynl Therapeutics Receive Marketing Authorization in the EU for NULIBRY\u00ae (fosdenopterin) for the Treatment of MoCD Type A","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Danicopan (ALXN2040) Add-On to Ultomiris or Soliris Met Primary Endpoint In Alpha Phase III Trial For Patients With Paroxysmal Nocturnal Haemoglobinuria Who Experience Clinically Significant Extravascular Haemolysis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Danicopan (ALXN2040) Add-On to ULTOMIRIS\u00ae (ravulizumab-cwvz) or SOLIRIS\u00ae (eculizumab) Met Primary Endpoint in ALPHA Phase III Trial for Patients with Paroxysmal Nocturnal Hemoglobinuria Who Experience Clinically Significant Extravascular Hemolysis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Relief Therapeutics Announces IRB Approval and Initiation of an Investigator Initiated Trial of Nexodyn for Epidermolysis Bullosa at Ann & Robert H. Lurie Children's Hospital of Chicago","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase I"},{"orgOrder":0,"company":"KBio","sponsor":"Leaf Expression Systems","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"KBio Announces Strategic Collaboration with Leaf Expression Systems to Enhance Development and Production of Novel Therapeutics","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Alexion, AstraZeneca Rare Disease to Acquire LogicBio\u00ae Therapeutics to Accelerate Growth in Genomic Medicine","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Discovery"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Atsena Therapeutics Announces Positive Results from Phase I\/II Clinical Trial of ATSN-101 for the Treatment of GUCY2D-Associated Leber Congenital Amaurosis (LCA1)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Saol therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Saol Therapeutics Announces Enrollment Achievement in Pivotal Phase 3 Trial for Rare Mitochondrial Disease - Pyruvate Dehydrogenase Complex Deficiency (PDCD)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"WuXi Biologics","sponsor":"Toregem BioPharma","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"WuXi Biologics and Toregem BioPharma Sign MOU for Development of Anti-USAG-1 Antibody","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Horizon Therapeutics plc Announces New UPLIZNA\u00ae (inebilizumab-cdon) Data in Neuromyelitis Optica Spectrum Disorder (NMOSD) to be Presented at ECTRIMS 2022","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Large molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alexion Aims To Advance NMOSD Treatment Landscape With Exceptional ULTOMIRIS\u00ae (ravulizumab-cwvz) Efficacy Data at ECTRIMS 2022","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alexion Aims To Advance NMOSD Treatment Landscape With Exceptional ULTOMIRIS (ravulizumab-cwvz) Efficacy Data At ECTRIMS 2022","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Cyclo Therapeutics Announces Publication of Phase 1 Data for Trappsol\u00ae Cyclo\u2122 for the Treatment of Niemann-Pick Disease Type C1 (NPC1)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Avilar Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Avilar Therapeutics to Present New In Vivo Proof-of-Concept Data for ATAC Extracellular Protein Degraders at Upcoming Drug Discovery Conferences","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Quoin Pharmaceuticals Announces Plans to Initiate a Second Clinical Trial in Netherton Syndrome Patients","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"LEXEO Therapeutics Receives Orphan Drug Designation for LX1004 from European Commission","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Alexion Aims To Advance NMOSD Treatment Landscape With Exceptional Ultomiris Efficacy Data At ECTRIMS 2022","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Goldman Sachs & Co","pharmaFlowCategory":"D","amount":"$28.0 million","upfrontCash":"Undisclosed","newsHeadline":"Taysha Announces Pricing of Public Offering of Common Stock","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase II"},{"orgOrder":0,"company":"Amryt Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"The British Journal of Dermatology Publishes Results from Amryt\u2019s EASE Phase 3 Trial in Epidermolysis Bullosa","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Novartis Investigational Oral Monotherapy Iptacopan Demonstrates Clinically Meaningful Superiority Over Anti-C5 Treatment in Phase III APPLY-PNH Study","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"New Analysis of UPLIZNA\u00ae (inebilizumab-cdon) Phase 3 Trial Data Demonstrates Importance of Reducing Plasmablasts to Help Prevent Neuromyelitis Optica Spectrum Disorder (NMOSD) Attacks","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Large molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultomiris Showed Zero Relapses In Adults With Neuromyelitis Optica Spectrum Disorder (NMOSD) With Median Treatment Duration Of 73 Weeks","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"Approved","date":"October 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Avilar Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Avilar Therapeutics to Present In Vivo Non-Human Primate Data on ASGPR-Targeting Chimeras (ATACs) at the Keystone Symposium on Targeted Protein Degradation","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Rallybio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rallybio Announces Positive Phase 1 Single Ascending Dose Results for RLYB116, an Innovative Subcutaneously Injected Inhibitor of Complement Component 5","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase I"},{"orgOrder":0,"company":"AstraZeneca","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AstraZeneca Expands Global Footprint In Rare Disease With Availability Of First Alexion Rare Disease Therapy For Patients In China","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"Approved","date":"November 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"LogicBio Therapeutics","sponsor":"Alexion Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Alexion, AstraZeneca Rare Disease, Completes Acquisition of LogicBio\u00ae Therapeutics","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Discovery"},{"orgOrder":0,"company":"Editas Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Editas Medicine Announces Clinical Data Demonstrating Proof of Concept of EDIT-101 from Phase 1\/2 BRILLIANCE Trial","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"IntraBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"IntraBio Reaches Target Enrollment for IB1001 NPC Pivotal Clinical Trial","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pharming Group Announces Presentation Of New Leniolisib Data At 2022 ASH Annual Meeting","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"AbCellera","sponsor":"Rallybio","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"AbCellera and Rallybio Announce Strategic Alliance to Discover, Develop, and Commercialize Novel Antibody-Based Therapeutics for Rare Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery Platform","country":"CANADA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Discovery Platform"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Piper Sandler","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"X4 Pharmaceuticals Announces Proposed Public Offering","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Altavant Sciences","sponsor":"Enzyvant Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Enzyvant Announces Merger with Altavant","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"Approved","date":"December 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Approved"},{"orgOrder":0,"company":"X4 Pharmaceuticals","sponsor":"Piper Sandler","pharmaFlowCategory":"D","amount":"$65.0 million","upfrontCash":"Undisclosed","newsHeadline":"X4 Pharmaceuticals Prices $65.0 Million Public Offering","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Kira Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Kira Pharmaceuticals Announces First Cohort of Patients Dosed in Phase 2 Study of KP104 in Paroxysmal Nocturnal Hemoglobinuria (PNH)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase II"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Opus Genetics Receives FDA Clearance of IND Application for OPGx-001, a Gene Therapy Candidate Intended for the Treatment of Rare Inherited Retinal Disease LCA5","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Phase I\/ Phase II"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Brazilian Regulatory Authority Grants Approval for Waylivra\u2122 for Familial Partial Lipodystrophy","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Large molecule","productStatus":"Approved","date":"December 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pharming Announces Publication Of Data From Phase 3 Study Of Leniolisib In Patients With APDS in ASH's Blood","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Genomic Biopharma","sponsor":"POIC e dintorni","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Genomic Biopharma and Patient Advocacy Organization POIC e Dintorni APS Partner To Offer Whole Genome Sequencing To Bring Better Treatments And Cures To Those Living With Gastrointestinal Diseases","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"ITALY","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Discovery"},{"orgOrder":0,"company":"IntraBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"IntraBio Completes Recruitment for IB1001 NPC Pivotal Clinical Trial","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Apellis Pharmaceuticals","sponsor":"Swedish Orphan Biovitrum AB","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sobi Receives Approval from Health Canada for Empaveli\u2122 (pegcetacoplan) for the Treatment of Certain Patients with Paroxysmal Nocturnal Hemoglobinuria","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Peptide","productStatus":"Approved","date":"December 2022","url1":"","url2":"","graph1":"Peptide","graph2":"Approved"},{"orgOrder":0,"company":"NovelMed Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Anti-Properdin Antibody (NM3086) Reduced Hemolysis, LDH, and Free Hemoglobin in an Animal Model of Paroxysmal Nocturnal Hemoglobinuria (PNH) -- a Rare Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Preclinical"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Quoin Pharmaceuticals Announces Dosing of First Patient in Clinical Trial in Netherton Syndrome","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Novartis Presents Pivotal Phase III APPLY-PNH Data at ASH Demonstrating Investigational Oral Monotherapy Iptacopan Superiority Over Anti-C5","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"F. Hoffmann-La Roche","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Roche\u2019s Subcutaneous Crovalimab Given Every Four Weeks Achieves Disease Control In People With PNH, a Life-threatening Blood Condition","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Novartis Pharmaceuticals Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Novartis Investigational Iptacopan Provides Clinically Meaningful Increases In Hemoglobin Levels In Complement-inhibitor-na\u00efve Patients With PNH","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"SWITZERLAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Pharming","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pharming Announces Positive Interim Analysis Data from Open-Label Extension Study of Leniolisib in Presentation at The 64th American Society of Hematology (ASH) Annual Meeting and Exposition English","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Phase III"},{"orgOrder":0,"company":"ANI Pharmaceuticals Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ANI Pharmaceuticals Announces the FDA Approval and Launch of Levocarnitine Tablets USP","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"December 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Approved"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"UPLIZNA\u00ae (inebilizumab-cdon) Approved in Brazil for the Treatment of Neuromyelitis Optica Spectrum Disorder (NMOSD)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Large molecule","productStatus":"Approved","date":"December 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Ethris","sponsor":"Cipla","pharmaFlowCategory":"D","amount":"$16.0 million","upfrontCash":"Undisclosed","newsHeadline":"Cipla EU to Invest EUR 15 Million In Ethris, Initiating A Strategic Collaboration","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Discovery","country":"GERMANY","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Discovery"},{"orgOrder":0,"company":"Cyclo Therapeutics","sponsor":"H.C. Wainwright & Co.","pharmaFlowCategory":"D","amount":"$4.2 million","upfrontCash":"Undisclosed","newsHeadline":"Cyclo Therapeutics Announces $4.2 Million Registered Direct Offering Priced At-The-Market Under Nasdaq Rules","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Phase III"},{"orgOrder":0,"company":"Horizon Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Analysis from the Phase 3 N-MOmentum Study Demonstrates the Effectiveness of UPLIZNA\u00ae (inebilizumab-cdon) Among Neuromyelitis Optica Spectrum Disorder Patients with Genetic Variations","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Approved","country":"IRELAND","productType":"Large molecule","productStatus":"Approved","date":"May 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Approved"},{"orgOrder":0,"company":"Medable","sponsor":"Every Cure","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Medable Partners with Every Cure to Accelerate Discovery of Treatments for Rare Diseases","therapeuticArea":"Rare Diseases and Disorders","highest
Market Place