IOL Chemicals IOL Chemicals

X
[{"orgOrder":0,"company":"Escape Bio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ESCAPE Bio Announces Excellent Safety and Tolerability in Phase 1 Study of Oral S1P5 Receptor Agonist ESB1609","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Escape Bio"},{"orgOrder":0,"company":"Rubius Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rubius Therapeutics Announces First Patient Dosed in Phase 1b Trial of RTX-134 for the Treatment of Patients with Phenylketonuria","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Rubius Therapeutics"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Regulus Therapeutics Reinitiates Multiple Ascending Dose Study of RGLS4326 for the Treatment of ADPKD","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Regulus Therapeutics"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FORMA Therapeutics Announces FT-4202 Granted Fast Track Designation and Rare Pediatric Disease Designation for Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Forma Therapeutics"},{"orgOrder":0,"company":"Magenta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Magenta Therapeutics Completes Dosing in Phase 1 MGTA-145 Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Magenta Therapeutics"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"NewCo","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"FORMA Therapeutics Announces Divestiture of Select Hit Discovery Capabilities","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Forma Therapeutics"},{"orgOrder":0,"company":"Inflazome","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inzomelid completes Phase I studies and shows positive results in the treatment of Cryopyrin-Associated Periodic Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"IRELAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Inflazome"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FORMA Therapeutics Receives FDA Orphan Drug Designation for FT-4202 for the Treatment of Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Forma Therapeutics"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Zafgen","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Zafgen and Chondrial Therapeutics Announce Definitive Merger Agreement","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Larimar Therapeutics"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Reneo Pharmaceuticals Initiates Clinical Trial in McArdle Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Reneo Pharmaceuticals"},{"orgOrder":0,"company":"Uniqure","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"uniQure Presents Multiple New Preclinical Data on AMT-130 at the CHDI\u2019s 15th Annual Huntington\u2019s Disease Therapeutics Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"NETHERLANDS","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Uniqure"},{"orgOrder":0,"company":"Axplora","sponsor":"Lysogene","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Novasep and Lysogene Announce their New Collaboration for Development and Production of GM1 Gangliosidosis Gene Therapy Product","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"GERMANY","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Axplora"},{"orgOrder":0,"company":"Magenta Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Magenta Therapeutics Presents Data on MGTA-145 at The Annual Meeting of American Society of Gene and Cell Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Magenta Therapeutics"},{"orgOrder":0,"company":"Expression Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Agrees to Phase 1 Trial of ET3, Gene Therapy for Hemophilia A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Expression Therapeutics"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Arcturus Therapeutics Announces First Healthy Volunteer Dosed in Phase 1 Study of ARCT-810 for Ornithine Transcarbamylase (OTC) Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Arcturus Therapeutics"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Forma Therapeutics Announces Phase I Clinical Data for Investigational Agent FT-4202 in Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Forma Therapeutics"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BridgeBio Pharma\u2019s ML Bio Solutions Dosed First Subject in Phase 1 Clinical Trial of BBP-418 For Limb Girdle Muscular Dystrophy Type 2i","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"BridgeBio Pharma"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Reneo Pharmaceuticals Completes Clinical Study in Primary Mitochondrial Myopathies and Receives FDA Orphan Drug Designation for REN001","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Reneo Pharmaceuticals"},{"orgOrder":0,"company":"Calithera Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Calithera Biosciences Initiates Phase 1b Trial of Arginase Inhibitor CB-280 for the Treatment of Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Calithera Biosciences"},{"orgOrder":0,"company":"Inflazome","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Orphan Drug Designation for Inflazome\u2019s Inzomelid for the Treatment of Cryopyrin-Associated Periodic Syndromes (CAPS)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"IRELAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Inflazome"},{"orgOrder":0,"company":"Ribomic","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"RIBOMIC Announces Dosing of First Subject in RBM-007 Phase 1 Clinical Trial for Achondroplasia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Ribomic"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Larimar Therapeutics Announces Dosing of Patients in Third Cohort of Phase 1 SAD Trial of CTI-1601 for Treatment of Friedreich\u2019s Ataxia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Larimar Therapeutics"},{"orgOrder":0,"company":"Abliva","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Abliva receives positive FDA feedback on its KL1333 development plant","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"SWEDEN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Abliva"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Larimar Therapeutics Gets Positive Opinion on Orphan Drug Designation Received from the EMA for CTI-1601","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Larimar Therapeutics"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Orphan Drug Status to Regulus Therapeutics' Treatment for Common Type of PKD","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Regulus Therapeutics"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Preclinical Characterization of Novel B2-Receptor Antagonists Published in Frontiers in Pharmacology","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"SWITZERLAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Pharvaris"},{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Enterprise Therapeutics Doses First Subjects in Phase 1 Trial for First-in-Class Cystic Fibrosis Therapy ETD002","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Enterprise Therapeutics"},{"orgOrder":0,"company":"Z Factor","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Z Factor Doses First Subject with ZF874, a Potential Disease-Modifying Treatment for Alpha-1-Antitrypsin Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Z Factor"},{"orgOrder":0,"company":"Recursion Pharma","sponsor":"Bayer AG","pharmaFlowCategory":"D","amount":"$239.0 million","upfrontCash":"Undisclosed","newsHeadline":"Recursion Pharmaceuticals Raises $239 Million And Partners With Bayer","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Recursion Pharma"},{"orgOrder":0,"company":"Corino Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Corino Therapeutics Announces Presentation of New Clinical Research Finding at the XVII International Symposium on Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Corino Therapeutics"},{"orgOrder":0,"company":"Inversago Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inversago Pharma Initiates Clinical Trials, with a Phase 1 study of INV-101, the Company\u2019s Lead Peripheral CB1 Blocker","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Inversago Pharma"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Arcturus Therapeutics Announces Completion of First Three Dose Escalation Cohorts in Phase 1 Study of ARCT-810","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Arcturus Therapeutics"},{"orgOrder":0,"company":"Calithera Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Calithera Biosciences\u2019 CB-280 Arginase Inhibitor Trial in Progress Poster Presented at the North American Cystic Fibrosis 2020 Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Calithera Biosciences"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"KalVista Pharmaceuticals Provides Update on Oral Hereditary Angioedema Franchise","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"KalVista Pharmaceuticals"},{"orgOrder":0,"company":"Astellas Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"U.S. FDA Grants Fast Track Designation to Astellas Pharma's ASP0367\/MA-0211","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"JAPAN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Astellas Pharma"},{"orgOrder":0,"company":"Calithera Biosciences","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","amount":"$2.4 million","upfrontCash":"Undisclosed","newsHeadline":"Calithera Biosciences Awarded up to $2.4M from Cystic Fibrosis Foundation to Support Clinical Development of Arginase Inhibitor, CB-280","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Calithera Biosciences"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Fulcrum Therapeutics Announces Multiple Presentations During the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Fulcrum Therapeutics"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Intellia Therapeutics Doses First Patient in Landmark CRISPR\/Cas9 Clinical Trial of NTLA-2001 for the Treatment of Transthyretin Amyloidosis","therapeuticArea":"Genetic Disease","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":"Intellia Therapeutics"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Forma Therapeutics Announces FT-4202 Receives Orphan Drug Designation in Europe for Treatment of Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Forma Therapeutics"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Edgewise Therapeutics Initiates Phase 1 Clinical Trial of EDG-5506 in Becker Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Edgewise Therapeutics"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Viking Global Investors","pharmaFlowCategory":"D","amount":"$80.0 million","upfrontCash":"Undisclosed","newsHeadline":"Pharvaris Announces $80 Million Series C Financing to Advance Novel Oral Bradykinin-B2-Receptor Antagonists for the Treatment of HAE","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"SWITZERLAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Pharvaris"},{"orgOrder":0,"company":"Pharvaris","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pharvaris Presents Clinical Data at ACAAI 2020 Demonstrating Safety and Therapeutic Potential of Oral PHA121 for the Treatment of HAE","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"SWITZERLAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Pharvaris"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PTC Therapeutics Announces that PTC518 Has Entered into a Phase 1 Clinical Trial for the Huntington's Disease Program","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"PTC Therapeutics"},{"orgOrder":0,"company":"Forma Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Forma Therapeutics Presents Clinical Proof-of-Concept Data Supporting the Potential of FT-4202 to Treat Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Forma Therapeutics"},{"orgOrder":0,"company":"EspeRare","sponsor":"Pierre Fabre","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"The EspeRare Foundation and Pierre Fabre Join Forces to Develop and Market a Pioneering Treatment for XLHED","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"SWITZERLAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"EspeRare"},{"orgOrder":0,"company":"Prothena","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Prothena Reports Positive 9 Month Results from Phase 1 Long-term Extension Study of PRX004","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"IRELAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Prothena"},{"orgOrder":0,"company":"AGC Biologics","sponsor":"Pierre Fabre","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Pierre Fabre Selects AGC Biologics as CDMO to Manufacture the Orphan Drug ER-004","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"AGC Biologics"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Intellia Therapeutics Highlights Strategic Priorities and Anticipated Development Milestones for 2021","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Intellia Therapeutics"},{"orgOrder":0,"company":"Inversago Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inversago Pharma Receives Rare Pediatric Disease Designation from the FDA for INV-101 for the Treatment of Prader-Willi Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Inversago Pharma"},{"orgOrder":0,"company":"Errant Gene Therapeutics","sponsor":"Memorial Sloan Kettering Cancer Center","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Errant Gene Therapeutics Pushing Clinical Trial of Potentially Curative Treatment for Beta-Thalassemia and Eventually Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Errant Gene Therapeutics"},{"orgOrder":0,"company":"Immunoforge","sponsor":"PhaseBio Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Immunoforge's 'PF1801' Is Designated as Orphan Drug by FDA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"SOUTH KOREA","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"January 2021","url1":"","url2":"","graph1":"Undisclosed","graph2":"Immunoforge"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Crinetics Pharmaceuticals Lead ACTH Antagonist for Congenital Adrenal Hyperplasia and Cushing\u2019s Disease (CRN04894) Advances into P1 Study","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Crinetics Pharmaceuticals"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Regulus Therapeutics Announces Completion of Enrollment in First Cohort of Phase 1b Clinical Trial of RGLS4326 for Autosomal Dominant PKD","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Regulus Therapeutics"},{"orgOrder":0,"company":"Ciitizen","sponsor":"Praxis Precision Medicines","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Praxis Precision Medicines Partners With Ciitizen to Improve Patient-Guided Drug Development","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"JAPAN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Ciitizen"},{"orgOrder":0,"company":"Selecta Biosciences","sponsor":"AskBio","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Selecta Biosciences and AskBio Initiate First-in-Human Dose-Escalation Study to Evaluate ImmTOR\u2122 in Gene Therapy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Selecta Biosciences"},{"orgOrder":0,"company":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Saniona Receives U.S. FDA Orphan Drug Designation for Tesomet in Prader-Willi Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Saniona"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Intellia Therapeutics\u2019 Investigational CRISPR Treatment NTLA-2001 Receives European Union Orphan Drug Designation for ATTR Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Intellia Therapeutics"},{"orgOrder":0,"company":"Azafaros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Azafaros Enters Clinical Stage with AZ-3102, An Oral Small Molecule Being Developed for Rare Neurogenetic Disorders","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Azafaros"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Fulcrum Therapeutics Presents Published Structure of Investigational Small Molecule FTX-6058 at the ACS Spring 2021 Virtual Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Fulcrum Therapeutics"},{"orgOrder":0,"company":"Prothena","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Prothena Presents Phase 1 Study Results of PRX004 in Oral Presentation at AAN 2021","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"IRELAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Prothena"},{"orgOrder":0,"company":"Voyager Therapeutics, Inc","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Voyager Therapeutics Receives FDA Clearance of IND Application for Gene Therapy Candidate VY-HTT01 for Treatment of Huntington\u2019s Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Voyager Therapeutics, Inc"},{"orgOrder":0,"company":"Pluri","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pluristem Reports Positive Topline Phase I Results in Innovative Hematology Program, Which Is First to Study PLX-R18 in Humans","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"ISRAEL","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Pluri"},{"orgOrder":0,"company":"Praxis Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Praxis Precision Medicines Receives Orphan Drug Designation for PRAX-562 for the Treatment of SCN2A-DEE","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"SPAIN","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Praxis Pharmaceutical"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Veristat","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Larimar Reports Positive Topline Phase 1 Data Showing Dose-Dependent Increases in Frataxin Levels in Patients with Friedreich\u2019s Ataxia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Larimar Therapeutics"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"F. Hoffmann-La Roche","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sarepta Therapeutics to Share Expression and Safety Results from Study 103 (ENDEAVOR) Evaluating SRP-9001","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Sarepta Therapeutics"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Veristat","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Larimar Therapeutics Reports FDA Clinical Hold on CTI-1601 and Termination of Recently Announced Private Placement Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Larimar Therapeutics"},{"orgOrder":0,"company":"Baylor College of Medicine","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Baylor Recruiting Melas Syndrome Patients for Phase 1 Clinical Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Baylor College of Medicine"},{"orgOrder":0,"company":"Neuren Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Positive FDA Meetings Enable Neuren to Proceed with INDs for Three Phase 2 Trials","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"AUSTRALIA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Neuren Pharmaceuticals"},{"orgOrder":0,"company":"Enterprise Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Enterprise Therapeutics Doses First Subjects in Phase I Trial for Novel Cystic Fibrosis Therapy ETD001","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Enterprise Therapeutics"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Regulus to Present Additional Data from its ADPKD Program","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Regulus Therapeutics"},{"orgOrder":0,"company":"Prothena","sponsor":"Novo Nordisk","pharmaFlowCategory":"D","amount":"$1,200.0 million","upfrontCash":"$100.0 million","newsHeadline":"Prothena and Novo Nordisk Announce Acquisition Agreement for Prothena\u2019s ATTR Amyloidosis Programme","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"IRELAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Prothena"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Intellia and Regeneron Announce Landmark Clinical Data Showing Deep Reduction in Disease-Causing Protein After Single Infusion of NTLA-2001","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Intellia Therapeutics"},{"orgOrder":0,"company":"Synlogic","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Synlogic Announces Initiation of Phase 1 Study of SYNB1934, a Next-Generation Strain for the Treatment of Phenylketonuria (PKU)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Synlogic"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Crinetics Pharmaceuticals\u2019 CRN04894, Shows Pharmacologic Proof-of-Concept with Dose-Dependent Cortisol Suppression","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Crinetics Pharmaceuticals"},{"orgOrder":0,"company":"Aldeyra Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Aldeyra Therapeutics Receives Orphan Drug Designation from the U.S. Food and Drug Administration for ADX-2191 to Treat Retinitis Pigmentosa","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"August 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Aldeyra Therapeutics"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Edgewise Receives U.S. FDA Fast Track Designation for EDG-5506 for the Treatment of Individuals with Becker Muscular Dystrophy (BMD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Edgewise Therapeutics"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Announces FDA Lifts Clinical Hold on Danon Disease Trial of RP-A501","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"Inversago Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inversago Pharma Initiates a Phase 1 Clinical Trial on INV-202, a Next Generation Peripherally-acting CB1 Blocker","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Inversago Pharma"},{"orgOrder":0,"company":"Inversago Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inversago Pharma Begins a Phase 1 Clinical Trial with INV-202, A New Generation Peripheral Action CB1 Blocker","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Inversago Pharma"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Presents Clinical Data from RP-A501 Trial in Danon Disease at the HFSA Annual Scientific Meeting 2021","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"Sage Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Sage Therapeutics Receives Fast Track Designation for SAGE-718 for the Treatment of Huntington\u2019s Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Sage Therapeutics"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Crinetics Pharmaceuticals\u2019 Oral SST5 Agonist CRN04777 Demonstrated Pharmacologic Proof-of-Concept with Strong Dose-dependent Suppression of Insulin Secretion in Phase 1 Single Ascending Dose Study","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Crinetics Pharmaceuticals"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PTC Therapeutics to Host Conference Call to Discuss Results of PTC518 Phase 1 Study for Huntington's Disease Program","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"PTC Therapeutics"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Krystal Biotech Receives Approval from the Human Research Ethics Committee in Australia for Phase 1 Trial of KB407 for Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Krystal Biotech"},{"orgOrder":0,"company":"Inhibrx","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"INBRX-101 Shows Favorable Safety Profile in Patients with Alpha-1 Antitrypsin Deficiency and Demonstrates the Potential to Achieve Normal Alpha-1 Antitrypsin Levels with Monthly Dosing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2021","url1":"","url2":"","graph1":"Large molecule","graph2":"Inhibrx"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Homology Medicines Announces World\u2019s First Gene Editing Clinical Trial for PKU","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Homology Medicines"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Homology Medicines Initiates Clinical Trial for HMI-203, a One-Time Investigational Gene Therapy Candidate for Adults with MPS II (Hunter Syndrome)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Homology Medicines"},{"orgOrder":0,"company":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Saniona Completes Submission of Manufacturing Data for Tesomet Capsules To U.S. FDA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Saniona"},{"orgOrder":0,"company":"Calithera Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Calithera Biosciences to Present First Clinical Data from Development Program for Arginase Inhibitor CB-280 at NACFC 2021","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Calithera Biosciences"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Intellia Therapeutics Receives U.S. FDA Orphan Drug Designation for NTLA-2001, an Investigational CRISPR Therapy for the Treatment of Transthyretin (ATTR) Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Intellia Therapeutics"},{"orgOrder":0,"company":"Nova Mentis Life Science","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Nova Mentis Earns Psilocybin Orphan Drug Designation in Europe for Fragile X Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Nova Mentis Life Science"},{"orgOrder":0,"company":"Recursion Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Recursion is Granted Orphan Drug Designation for REC-4881 for the Potential Treatment of Familial Adenomatous Polyposis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Recursion Pharma"},{"orgOrder":0,"company":"Calithera Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Calithera Presents Interim Data from Phase 1b Trial of Arginase Inhibitor CB-280 in Cystic Fibrosis at NACFC 2021","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Calithera Biosciences"},{"orgOrder":0,"company":"Centessa Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Centessa Pharmaceuticals Demonstrates Proof-of-Mechanism from First Three PiMZ Subjects Dosed in Part B of Phase 1 Study Evaluating ZF874","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Centessa Pharmaceuticals"},{"orgOrder":0,"company":"Nova Mentis Life Science","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"U.S. FDA Approves Nova Mentis Orphan Drug Application","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Nova Mentis Life Science"},{"orgOrder":0,"company":"RepliCel","sponsor":"Innovacell Biotechnologie","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"RepliCel\u2019s Clinical Advisory Team Delivers Skin Rejuvenation Clinical Study Synopsis to its Japanese Regulatory and Clinical Study Management Team","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"RepliCel"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Announces Positive Updates from Phase 1 Clinical Trial of RP-A501 in Danon Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Intellia Therapeutics Announces Expansion of Ongoing Phase 1 Study of NTLA-2001 to Include Adults with Transthyretin Amyloidosis with Cardiomyopathy (ATTR-CM)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Intellia Therapeutics"},{"orgOrder":0,"company":"Zydus Lifesciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Zydus to Begin Phase II (a) Clinical Trial of ZYIL1, a Novel Oral NLRP3 Inflammasome Inhibitor in Patients with CryopyrinAssociated Periodic Syndrome (CAPS) in Australia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"INDIA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Zydus Lifesciences"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GBT Presents Positive Results from Phase 1 Study of GBT021601 in Patients with Sickle Cell Disease and Healthy Volunteers at ASH Annual Meeting and Exposition","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Global Blood Therapeutics"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Edgewise Therapeutics to Report Phase 1b Topline Results of EDG-5506 in Individuals with Becker Muscular Dystrophy (BMD) on January 5, 2022, and Initiates ARCH Follow-On Open Label BMD Study","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Edgewise Therapeutics"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Crinetics Pharmaceuticals to Present Corporate and Clinical Update at 40th Annual J.P. Morgan Healthcare Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Crinetics Pharmaceuticals"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Edgewise Therapeutics Announces Positive Topline Results From the EDG-5506 Phase 1b Clinical Trial in Adults With Becker Muscular Dystrophy (BMD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Edgewise Therapeutics"},{"orgOrder":0,"company":"EdiGene","sponsor":"Haihe Laboratory","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"EdiGene Enters Strategic R&D Collaboration with Haihe Laboratory of Cell Ecosystem to Develop Hematopoietic Stem Cell Regenerative Therapies and Platform Technologies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"CHINA","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"EdiGene"},{"orgOrder":0,"company":"Viking Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Viking Therapeutics Announces Clinical Hold on Phase 1b Trial of VK0214 in Patients with X-ALD","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Viking Therapeutics"},{"orgOrder":0,"company":"Azafaros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Azafaros Receives FDA Orphan Drug Designation for AZ-3102 in GM2 Gangliosidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Azafaros"},{"orgOrder":0,"company":"Azafaros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Azafaros Presents Positive Clinical and Preclinical Data Supporting Development of Lead Compound AZ-3102 in Lysosomal Storage Disorders at the 18th Annual WORLDSymposium\u2122 Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Azafaros"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Veristat","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Larimar Therapeutics Provides Update on CTI-1601 Clinical Program","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Larimar Therapeutics"},{"orgOrder":0,"company":"Bristol Myers Squibb","sponsor":"Biohaven Pharmaceuticals","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Biohaven Licenses Taldefgrobep Alfa, a Phase 3-Ready Anti-Myostatin Adnectin for Spinal Muscular Atrophy (SMA), From Bristol Myers Squibb","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Bristol Myers Squibb"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Intellia and Regeneron Announce Updated Phase 1 Data Demonstrating a Single Dose of NTLA-2001, Investigational Crispr Therapy for Transthyretin (ATTR) Amyloidosis, Resulted in Rapid, Deep and Sustained Reduction in Disease-causing Protein","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Intellia Therapeutics"},{"orgOrder":0,"company":"Inhibrx","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inhibrx Receives FDA Orphan-Drug Designation for INBRX-101 for the Treatment of Alpha-1 Antitrypsin Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Inhibrx"},{"orgOrder":0,"company":"Recursion Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Recursion Provides Updated Guidance on Clinical Trial Starts","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Recursion Pharma"},{"orgOrder":0,"company":"Aldeyra Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Aldeyra Therapeutics Initiates Phase 2 Clinical Trial of ADX-2191 for the Treatment of Retinitis Pigmentosa","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"March 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Aldeyra Therapeutics"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Muscular Dystrophy Association","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Edgewise Therapeutics to Present on EDG-5506 for Becker and Duchenne Muscular Dystrophy (BMD, DMD) at the 2022 Annual MDA Clinical and Scientific Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Edgewise Therapeutics"},{"orgOrder":0,"company":"Rejuvenate Biomed","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rejuvenate Biomed Started a Groundbreaking Clinical Trial and Expands for Future Growth","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"BELGIUM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Rejuvenate Biomed"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Crinetics Pharmaceuticals Reports Positive Top-Line Results from CRN04777 Phase 1 Study Multiple-Ascending Dose Cohorts","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Crinetics Pharmaceuticals"},{"orgOrder":0,"company":"Xortx Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"XORTX Files IND with US FDA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Xortx Therapeutics"},{"orgOrder":0,"company":"Bond Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Bond Biosciences Announces First Dosing of Sentinel Patients in a Phase Ia\/b Clinical Trial for a Novel Therapy to Treat Patients with Hemochromatosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Bond Biosciences"},{"orgOrder":0,"company":"Dystrogen Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Dystrogen Therapeutics Investigational Chimeric Cell Therapy DT-DEC01 for the Treatment of Duchene Muscular Dystrophy Shows Safety and Functional Improvements","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Dystrogen Therapeutics"},{"orgOrder":0,"company":"Design Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Design Therapeutics Completes Dosing in First Patient Cohort of Phase 1 Trial of DT-216 GeneTAC Molecule for the Treatment of Friedreich Ataxia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Design Therapeutics"},{"orgOrder":0,"company":"Chinook Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Chinook Therapeutics Announces Initiation of Phase 1 Healthy Volunteer Trial of CHK-336, a First-in-Class LDHA Inhibitor to Treat Hyperoxalurias","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Chinook Therapeutics"},{"orgOrder":0,"company":"Nabriva Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Nabriva Therapeutics Announces First Patient Enrolled in Phase 1 Trial of XENLETA (lefamulin) in Adult Patients with Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"April 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Nabriva Therapeutics"},{"orgOrder":0,"company":"Ribomic","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"RIBOMIC Announces Completion of IND Submission for an Observational Study for Continuous Phase 2 Trial of RBM-007 for Treatment of Achondroplasia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Ribomic"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"European Medicines Agency Grants PRIME Designation to BioCryst's ALK-2 Inhibitor, BCX9250, for Treatment of Fibrodysplasia Ossificans Progressiva","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Homology Medicines Announces Upcoming Presentation on Optimized In Vivo Gene Editing Candidate HMI-103 with First Details of Unique Mechanism of Action at ASGCT Annual Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Homology Medicines"},{"orgOrder":0,"company":"Stealth Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Stealth BioTherapeutics Receives Orphan Drug Designation from FDA for Elamipretide for Treatment of Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Peptide","graph2":"Stealth Biotherapeutics"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Fulcrum Therapeutics to Present Initial Data from Phase 1b Trial of FTX-6058 in Adults Living with Sickle Cell Disease at the European Hematology Association (EHA) Hybrid Congress in Vienna, Austria","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Fulcrum Therapeutics"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GBT Announces Five Data Presentations on Sickle Cell Disease at Upcoming EHA2022 Congress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Global Blood Therapeutics"},{"orgOrder":0,"company":"PepGen","sponsor":"BofA Securities","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"PepGen Announces Pricing of Initial Public Offering","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Peptide","graph2":"PepGen"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Presents Positive Clinical Data from Danon Disease, Fanconi Anemia and Pyruvate Kinase Deficiency Programs at the 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"Inhibrx","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"INBRX-101 Shows Favorable Safety Profile in Patients with Alpha-1 Antitrypsin Deficiency and Demonstrates Potential to Achieve Normal Functional Alpha-1 Antitrypsin Levels with Monthly Dosing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Inhibrx"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Homology Medicines Presents Details of Optimized HMI-103 Nuclease-Free Gene Editing Candidate Featuring Integrated Liver-Specific Promoter to Maximize Long-Term Expression","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Homology Medicines"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Larimar Therapeutics Announces Upcoming Poster Presentations at the 4th Pan American Parkinson's Disease and Movement Disorders Congress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Larimar Therapeutics"},{"orgOrder":0,"company":"Liminal BioSciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Liminal BioSciences Announces First Subject Dosed in Phase 1a Single Ascending Dose Clinical Trial of Fezagepras","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Liminal BioSciences"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Crinetics Reports Top-Line CRN04894 MAD Results from Phase 1 Study","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Crinetics Pharmaceuticals"},{"orgOrder":0,"company":"National Institutes of Health","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rare Eye Disease Gene Therapy Lacks Efficacy in NIH-funded Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"National Institutes of Health"},{"orgOrder":0,"company":"Global Blood Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GBT's Inclacumab and GBT601 Receive U.S. FDA Orphan Drug and Rare Pediatric Disease Designations for the Treatment of Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Global Blood Therapeutics"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Intellia Therapeutics to Present Updated Interim Clinical Data from Ongoing Phase 1 Study of NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis at the International Liver Congress\u2122 2022","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Intellia Therapeutics"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Fulcrum Therapeutics Announces Proof-of-Concept for FTX-6058 in Sickle Cell Disease Based on Initial Data from the Ongoing Phase 1b Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Fulcrum Therapeutics"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Larimar Therapeutics Announces Upcoming Scientific Conference Presentations","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Larimar Therapeutics"},{"orgOrder":0,"company":"Piramal Pharma Solutions","sponsor":"Epirium","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Piramal Pharma Solutions to Collaborate with Epirium Bio on Exclusive Integrated Development & Manufacturing Program for Orphan Drugs","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"August 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Piramal Pharma Solutions"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Fast Track Designation for BioCryst\u2019s ALK-2 Inhibitor, BCX9250","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Regulus Therapeutics Announces First Subject Dosed in Phase 1 Single-Ascending Dose (SAD) Clinical Trial of RGLS8429 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Regulus Therapeutics"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Regulus Therapeutics Announces Receipt of FDA Orphan Drug Designation for RGLS8429 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Regulus Therapeutics"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BridgeBio Pharma Announces Early Positive Data for BBP-812, its Investigational AAV9 Gene Therapy for Canavan Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"BridgeBio Pharma"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Regeneron Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Intellia and Regeneron Present Updated Interim Data from Phase 1 Study of CRISPR-based NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis Demonstrating that Deep Serum TTR Reductions Remained Durable After a Single Dose","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Intellia Therapeutics"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BridgeBio Pharma Announces Positive Phase 1 Data and Phase 2\/3 Trial Design for BBP-711, a Potentially Best-In-Class GO Inhibitor for Primary Hyperoxaluria Type 1 (PH1) and Recurrent Kidney Stone Formers","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"BridgeBio Pharma"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Larimar Therapeutics Announces Oral Presentation at the Upcoming Gordon Research Conference on Mitochondria and Chloroplasts","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Larimar Therapeutics"},{"orgOrder":0,"company":"Viking Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Viking Therapeutics Announces FDA Has Lifted Clinical Hold on Phase 1b Trial of VK0214 in Patients with X-ALD","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Viking Therapeutics"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Larimar Therapeutics Announces Presentation at the Upcoming Gordon Research Conference on Neurobiology of Brain Disorders","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Larimar Therapeutics"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Krystal Biotech Receives FDA Acceptance of KB407 IND Application for Cystic Fibrosis Clinical Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Krystal Biotech"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Astria Therapeutics Announces Initiation of Phase 1a Trial of STAR-0215, a Monoclonal Antibody Inhibitor of Plasma Kallikrein for Treatment of Hereditary Angioedema","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Astria Therapeutics"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Additional Analyses of CRN04894 Preclinical and Phase 1 Results to be Presented at the 10th International Congress of Neuroendocrinology","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Crinetics Pharmaceuticals"},{"orgOrder":0,"company":"Nabriva Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Nabriva Therapeutics Completes Patient Enrollment in Phase 1 Trial of XENLETA\u00ae (lefamulin) in Adult Patients with Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"IRELAND","productType":"Large molecule","productStatus":"Approved","date":"August 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Nabriva Therapeutics"},{"orgOrder":0,"company":"Vaderis Therapeutics","sponsor":"Medicxi","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vaderis Therapeutics AG Emerges from Stealth and Announces Initiation of Clinical Proof-of-Concept Trial in HHT","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"SWITZERLAND","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Vaderis Therapeutics"},{"orgOrder":0,"company":"VectorBuilder","sponsor":"Lantu Biopharma","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"VectorBuilder Supports First Gene Therapy Trial for Menkes Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"VectorBuilder"},{"orgOrder":0,"company":"Synlogic","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Synlogic Granted FDA Fast Track Designation for SYNB1353 for the Treatment of Homocystinuria (HCU)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Synlogic"},{"orgOrder":0,"company":"Maze Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Maze Therapeutics Announces FDA Orphan Drug Designation Granted to MZE001 for the Treatment of Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Maze Therapeutics"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Guggenheim Securities","pharmaFlowCategory":"D","amount":"$70.0 million","upfrontCash":"Undisclosed","newsHeadline":"Larimar Therapeutics Announces $70 Million Underwritten Offering","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Larimar Therapeutics"},{"orgOrder":0,"company":"Acasti Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Acasti Pharma Announces Initiation of Pharmacokinetic Study for GTX-102, the Company\u2019s Drug Candidate for the Treatment of Ataxia Telangiectasia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Acasti Pharma"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Larimar Therapeutics Announces FDA Clearance to Initiate the 25 mg Cohort of a Phase 2 Dose Exploration Trial of CTI-1601 in Friedreich\u2019s Ataxia Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Larimar Therapeutics"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Guggenheim Securities","pharmaFlowCategory":"D","amount":"$80.5 million","upfrontCash":"Undisclosed","newsHeadline":"Larimar Therapeutics Announces Closing of Underwritten Offering and Full Exercise of Option to Purchase Additional Shares","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Larimar Therapeutics"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Astria Therapeutics to Host Virtual R&D Day: Update on STAR-0215 and Its Clinical Development","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Astria Therapeutics"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals to Present Updated Data from Phase 1 Danon Disease Trial for RP-A501 at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Intellia and Regeneron Announce Initial Data from the Cardiomyopathy Arm of Ongoing Phase 1 Study of NTLA-2001, an Investigational CRISPR Therapy for the Treatment of Transthyretin (ATTR) Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Intellia Therapeutics"},{"orgOrder":0,"company":"Dystrogen Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Dystrogen Therapeutics Investigational Chimeric Cell Therapy DT-DEC01 for Duchenne Muscular Dystrophy Demonstrates Clinically Significant Functional and Biomarker Improvements","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Dystrogen Therapeutics"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Announces Positive Updates from Phase 1 Clinical Trial for RP-A501 in Danon Disease at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"PepGen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PepGen Reports Positive Data from Phase 1 Trial of PGN-EDO51 for the Treatment of Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Peptide","graph2":"PepGen"},{"orgOrder":0,"company":"Poxel","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Poxel Announces PXL770 Granted Orphan Drug Designation from the U.S. FDA for Autosomal-Dominant Polycystic Kidney Disease (ADPKD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"FRANCE","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Poxel"},{"orgOrder":0,"company":"Inhibrx","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inhibrx To Host Webcast to Announce Regulatory Pathway for INBRX-101, its Optimized Recombinant Human AAT-Fc Protein for the Treatment of Alpha-1 Antitrypsin Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Inhibrx"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Homology Medicines to Participate at Upcoming Conferences","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Homology Medicines"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Innovation Passport Granted for CRN04777 for the Treatment of Congenital Hyperinsulinism","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Crinetics Pharmaceuticals"},{"orgOrder":0,"company":"Attralus","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Attralus Announces First Subject Enrolled in Phase 1 Clinical Trial of AT-02, Its Lead Pan-Amyloid Removal Therapeutic Candidate","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Attralus"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vertex Advances Program Targeting Alpha-1 Antitrypsin Deficiency","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Vertex Pharmaceuticals"},{"orgOrder":0,"company":"Inhibrx","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inhibrx Announces Opportunity for Accelerated Approval Pathway on Functional AAT Serum Levels for INBRX-101 in AATD and Announces Bronchoalveolar Lavage Fluid Detection Results from the Phase 1 Study","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Inhibrx"},{"orgOrder":0,"company":"PepGen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PepGen Presents Data from its Duchenne Muscular Dystrophy Program at World Muscle Society Congress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Peptide","graph2":"PepGen"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Homology Medicines Presents on Design of pheEDIT Trial Evaluating One-Time Nuclease-Free Gene Editing Candidate HMI-103 for PKU at American Society of Human Genetics Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Homology Medicines"},{"orgOrder":0,"company":"KalVista Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"KalVista Pharmaceuticals Announces Positive Phase 1 Data for Orally Disintegrating Tablet Formulation of Sebetralstat for Use in Hereditary Angioedema","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"KalVista Pharmaceuticals"},{"orgOrder":0,"company":"Verve Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Verve Therapeutics Provides Regulatory Update on VERVE-101 Investigational New Drug Application and Reports Third Quarter 2022 Financial Results","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Verve Therapeutics"},{"orgOrder":0,"company":"Jnana Therapeutics","sponsor":"Bain Capital Life Sciences","pharmaFlowCategory":"D","amount":"$107.0 million","upfrontCash":"Undisclosed","newsHeadline":"Jnana Therapeutics Raises $107 Million to Advance Lead PKU Program and Progress Therapeutics Pipeline Discovered by the RAPID Platform","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Jnana Therapeutics"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Crinetics Pharmaceuticals Provides Update on CRN04777 Program","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Crinetics Pharmaceuticals"},{"orgOrder":0,"company":"Nabriva Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Nabriva Therapeutics Announces Positive Topline Results from Phase 1 Trial of XENLETA\u00c2\u00ae (lefamulin) in Adult Patients with Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"IRELAND","productType":"Small molecule","productStatus":"Approved","date":"November 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Nabriva Therapeutics"},{"orgOrder":0,"company":"Design Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Design Therapeutics Reports Positive Data from Single-Ascending Dose Trial of DT-216 for the Treatment of Friedreich Ataxia and Portfolio Progress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Design Therapeutics"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Astria Therapeutics Announces Positive Preliminary Results from the Phase 1a Clinical Trial of STAR-0215 in Healthy Subjects","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Astria Therapeutics"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Provides Update on Anticipated Registration Path for RP-A501 in Danon Disease Following End-of-Phase 1 FDA Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"Acasti Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Acasti Announces Preliminary Topline Results Met All Outcome Measures in the Pharmacokinetic Bridging Study for GTX-102, the Company\u2019s Drug Candidate for the Treatment of Ataxia Telangiectasia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"December 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Acasti Pharma"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Homology Medicines Provides Update on pheEDIT and juMPStart Clinical Trials and Announces Expected 2023 Milestones, Including Initial Data Read-Outs from Both Programs","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Homology Medicines"},{"orgOrder":0,"company":"Mission Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mission Therapeutics Successfully Completes First Clinical Assessment for Lead DUB Program, MTX652","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Mission Therapeutics"},{"orgOrder":0,"company":"Nexcella","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Immix Biopharma Subsidiary Nexcella, Inc. Announces Additional Positive NXC-201 Clinical Data Demonstrating 100% Complete Responses in Relapsed\/Refractory AL Amyloidosis Patients, Duration of Response Not Yet Reached","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Nexcella"},{"orgOrder":0,"company":"BioCryst Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BioCryst Reports Initial Clinical Data with Oral Factor D Inhibitor BCX10013 Supporting Development as a Once-daily Treatment for Complement-mediated Diseases","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"BioCryst Pharmaceuticals"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Expands Cardiac Gene Therapy Portfolio with Addition of RP-A601 for PKP2-ACM and Announces Positive Updated Phase 1 Data for RP-A501 in Danon Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"ADARx Pharmaceuticals","sponsor":"Ascenta Capital","pharmaFlowCategory":"D","amount":"$46.0 million","upfrontCash":"Undisclosed","newsHeadline":"ADARx Pharmaceuticals Raises $46 Million as Lead Product Enters the Clinic","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"ADARx Pharmaceuticals"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Krystal Biotech Receives European Commission Orphan Designation for KB407 for the Treatment of Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Krystal Biotech"},{"orgOrder":0,"company":"ADARx Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ADARx Pharmaceuticals Announces Dosing of First Cohort in Phase 1 Clinical Study of ADX-324","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"ADARx Pharmaceuticals"},{"orgOrder":0,"company":"Elixirgen Therapeutics","sponsor":"Hitachi Global Life Solutions","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Elixirgen Therapeutics Enters Memorandum of Understanding with Hitachi Global Life Solutions for the Development of Micro GMP Suites for Cell Therapy Administration","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Elixirgen Therapeutics"},{"orgOrder":0,"company":"Immusoft Corporation","sponsor":"California Institute for Regenerative Medicine","pharmaFlowCategory":"D","amount":"$8.0 million","upfrontCash":"Undisclosed","newsHeadline":"Immusoft Awarded $8M in Funding from the California Institute for Regenerative Medicine (CIRM) for MPS I Clinical Program","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Immusoft Corporation"},{"orgOrder":0,"company":"Reneo Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Reneo Pharmaceuticals Receives Fast Track Designation from the FDA for Mavodelpar (REN001) in a Genotype of Long-Chain Fatty Acid Oxidation Disorder (LC-FAOD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Reneo Pharmaceuticals"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Jefferies","pharmaFlowCategory":"D","amount":"$100.0 million","upfrontCash":"Undisclosed","newsHeadline":"Astria Therapeutics Announces Pricing of $100 Million Underwritten Offering of Common Stock","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Astria Therapeutics"},{"orgOrder":0,"company":"KGK Science","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Wellbeing Subsidiary KGK Science Receives Exemption Under Section 56 of the Controlled Drugs and Substances Act to Begin Psilocybin Clinical Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"February 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"KGK Science"},{"orgOrder":0,"company":"Accelagen","sponsor":"Kiora Pharmaceuticals","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Groundbreaking Clinical Trial Underway to Improve Vision in Individuals with Advanced Retinitis Pigmentosa (RP)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"AUSTRALIA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Accelagen"},{"orgOrder":0,"company":"Verve Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Verve Therapeutics Announces VERVE-101 Awarded Innovation Passport by the UK MHRA for the Treatment of Heterozygous Familial Hypercholesterolemia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Verve Therapeutics"},{"orgOrder":0,"company":"Maze Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Maze Therapeutics Announces Completion of Phase 1 First-in-Human Trial Evaluating MZE001 as a Potential Oral Treatment for Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Maze Therapeutics"},{"orgOrder":0,"company":"Fulcrum Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Fulcrum Therapeutics Announces Clinical Hold on FTX-6058 in Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Fulcrum Therapeutics"},{"orgOrder":0,"company":"Maze Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Maze Therapeutics Announces Positive Phase 1 Results from First-in-Human Trial Evaluating MZE001 as a Potential Oral Treatment for Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Maze Therapeutics"},{"orgOrder":0,"company":"BioLineRx","sponsor":"Washington University School of Medicine","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"BioLineRx Announces Clinical Trial Collaboration with Washington University School of Medicine to Evaluate Motixafortide for CD34+ Hematopoietic Stem Cell Mobilization for Gene Therapies in Sickle Cell Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"ISRAEL","productType":"Peptide","productStatus":"New Molecular Entity","date":"March 2023","url1":"","url2":"","graph1":"Peptide","graph2":"BioLineRx"},{"orgOrder":0,"company":"Mabwell","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mabwell Launched First In Human Clinical Trial of Its Iron Homeostasis Regulating Macromolecular Drug 9MW3011","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Mabwell"},{"orgOrder":0,"company":"Maze Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Maze Therapeutics Announces New Clinical Data Supporting MZE001 as a Potential Treatment for Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Maze Therapeutics"},{"orgOrder":0,"company":"GRIN Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"GRIN Therapeutics Announces First Patient Dosed in Phase 1B Clinical Trial with Radiprodil for Treatment of GRIN-related Disorders","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"GRIN Therapeutics"},{"orgOrder":0,"company":"Neurelis","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Neurelis Announces First Healthy Subjects Dosed in Investigational Study of NRL-1049, a RHO Kinase (rock) Inhibitor with Potential to Treat Cerebral Cavernous Malformations","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Neurelis"},{"orgOrder":0,"company":"Vega Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vega Therapeutics Initiates Clinical Trial Program for VGA039, a First\u2011in\u2011Class Antibody Therapy for von Willebrand Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Vega Therapeutics"},{"orgOrder":0,"company":"Chinook Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Chinook Therapeutics Announces Voluntary Pause in Dosing of CHK-336 in Ongoing Phase 1 Clinical Trial in Healthy Volunteers","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Chinook Therapeutics"},{"orgOrder":0,"company":"Inhibrx","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inhibrx Initiates a Registration-Enabling Trial of INBRX-101 in AATD and Announces Lift of Partial Clinical Hold on INBRX-109 DR5 Agonist Trials","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Inhibrx"},{"orgOrder":0,"company":"Maze Therapeutics","sponsor":"Sanofi","pharmaFlowCategory":"D","amount":"$750.0 million","upfrontCash":"$150.0 million","newsHeadline":"Maze Therapeutics Announces Exclusive Worldwide License Agreement with Sanofi for MZE001, an Oral Substrate Reduction Therapy for the Treatment of Pompe Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Maze Therapeutics"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Regulus Therapeutics Advances to Cohort 2 of Phase 1b Multiple-Ascending Dose (MAD) Clinical Trial of RGLS8429 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Regulus Therapeutics"},{"orgOrder":0,"company":"PepGen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PepGen Announces Clearance by Health Canada of CTA for PGN-EDO51 to Begin the Phase 2 Clinical Trial, CONNECT1-EDO51, for the Treatment of Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Peptide","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Peptide","graph2":"PepGen"},{"orgOrder":0,"company":"Freeline Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Freeline Granted ILAP Designation in United Kingdom by MHRA for FLT201, an Investigational Gene Therapy for the Treatment of Gaucher Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Freeline Therapeutics"},{"orgOrder":0,"company":"Vega Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Vega Therapeutics Receives FDA Orphan Drug Designation for VGA039 for the Treatment of von Willebrand Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Vega Therapeutics"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RP-L301 Gene Therapy for Pyruvate Kinase Deficiency (PKD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"ConSynance Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ConSynance Therapeutics Reports Positive Phase 1 Results for CSTI-500 in Prader-Willi Syndrome Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"ConSynance Therapeutics"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Regulus Therapeutics Announces First Patient Dosed in Second Cohort of Phase 1b Multiple-Ascending Dose (MAD) Clinical Trial of RGLS8429 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Regulus Therapeutics"},{"orgOrder":0,"company":"Evotec","sponsor":"Bayer AG","pharmaFlowCategory":"D","amount":"$351.3 million","upfrontCash":"Undisclosed","newsHeadline":"Evotec Receives Milestone Payment for First Patient Dosed in Phase I Study of Bayer Kidney Disease Programme","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"GERMANY","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Evotec"},{"orgOrder":0,"company":"Krystal Biotech","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Krystal Biotech Announces First Patient Dosed in Phase 1 Clinical Trial of KB407 for the Treatment of Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Krystal Biotech"},{"orgOrder":0,"company":"Cystetic Medicines","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"cystetic Medicines Initiates Phase 1 Clinical Trial of a Molecular Prosthetic for Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Cystetic Medicines"},{"orgOrder":0,"company":"Bond Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Bond Biosciences Announces Positive Phase Ia\/b Pharmacodynamic and Safety Data for BBI-001, a Novel, Non-Absorbed, Oral Therapy for Iron Overload","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Bond Biosciences"},{"orgOrder":0,"company":"Homology Medicines","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Homology Medicines Announces Encouraging Initial Data from First Dose Level in the pheEDIT Trial Evaluating Gene Editing Candidate HMI-103 in Adults with Classical PKU","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Homology Medicines"},{"orgOrder":0,"company":"ADARx Pharmaceuticals","sponsor":"Bain Capital Life Sciences","pharmaFlowCategory":"D","amount":"$200.0 million","upfrontCash":"Undisclosed","newsHeadline":"ADARx Pharmaceuticals, a Leader in Next Generation RNA Therapeutics, Announces Oversubscribed $200 Million Series C Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"August 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"ADARx Pharmaceuticals"},{"orgOrder":0,"company":"Design Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Design Therapeutics Reports Initial Results from Phase 1 Multiple-Ascending Dose Study of DT-216 for the Treatment of Friedrich Ataxia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Design Therapeutics"},{"orgOrder":0,"company":"Peak Bio","sponsor":"White Lion Capital","pharmaFlowCategory":"D","amount":"$100.0 million","upfrontCash":"Undisclosed","newsHeadline":"Peak Bio, Inc. Announces Amendment and Continued Access to Equity Line of up to $100 Million USD via an Accelerated Common Stock Purchase Transaction with White Lion Capital","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Peak Bio"},{"orgOrder":0,"company":"Star Therapeutics","sponsor":"Sofinnova Investments","pharmaFlowCategory":"D","amount":"$90.0 million","upfrontCash":"Undisclosed","newsHeadline":"Star Therapeutics Raises $90 Million Financing to Accelerate its Pipeline of Novel Antibody Therapies and Growth of its Portfolio Companies","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Star Therapeutics"},{"orgOrder":0,"company":"SpliSense","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"SpliSense Successfully Completed Phase 1 Study of SPL84, RNA-Based Therapy, for the Treatment of Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"ISRAEL","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"SpliSense"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","amount":"$175.0 million","upfrontCash":"Undisclosed","newsHeadline":"Rocket Pharmaceuticals Announces Pricing of Public Offering of Common Stock","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Reaches FDA Alignment on Pivotal Phase 2 Trial Design for RP-A501 in Danon Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Regulus Therapeutics Announces Completion of Enrollment in Second Cohort of Phase 1b Multiple-Ascending Dose (MAD) Clinical Trial of RGLS8429 for the Treatment of Patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Regulus Therapeutics"},{"orgOrder":0,"company":"Atamyo Therapeutics","sponsor":"Bpifrance","pharmaFlowCategory":"D","amount":"$8.6 million","upfrontCash":"Undisclosed","newsHeadline":"ATA-200, Atamyo Therapeutics\u2019 Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type 2C\/R5, Reaches Key Milestones with the Filing of a Clinical Trial Application in Europe and a Non-Dilutive Financing from France 2030 Program","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Atamyo Therapeutics"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Taysha Gene Therapies Provides Update on TSHA-120 Program in Giant Axonal Neuropathy (GAN)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Taysha Gene Therapies"},{"orgOrder":0,"company":"Regulus Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Regulus Therapeutics Announces Positive Topline Data from the First Cohort of Patients in its Phase 1b Multiple-Ascending Dose (MAD) Clinical Trial of RGLS8429 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Regulus Therapeutics"},{"orgOrder":0,"company":"Entrada Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Entrada Therapeutics Announces First Participant Dosed in its Phase 1 Clinical Trial of ENTR-601-44 for the Potential Treatment of Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Entrada Therapeutics"},{"orgOrder":0,"company":"Arcturus Therapeutics","sponsor":"Cystic Fibrosis Foundation","pharmaFlowCategory":"D","amount":"$25.0 million","upfrontCash":"Undisclosed","newsHeadline":"Arcturus Therapeutics and Cystic Fibrosis Foundation Extend Agreement to Advance ARCT-032, an Investigational Messenger RNA (mRNA) Therapeutic to Treat Cystic Fibrosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Enti