[{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed First to Advance Cannabinol (CBN) into Therapeutic Clinical Trials","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"Inovio Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inovio Publishes Patient Benefit Results From A Pilot Clinical Study","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Vaccine","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Vaccine","graph2":"Inovio Pharmaceuticals"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Announces Completed Enrollment in Phase 1 Clinical Trial of INM-755 CBN","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Submits Clinical Trial Application to Evaluate INM-755 in Second Phase 1 Trial","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Announces Completion of Initial Phase 1 Clinical Trial of INM-755 CBN Cream in Healthy Subjects","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Announces Science Translational Medicine Publication of its Program in Danon Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"March 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Announces Dosing Initiation in 2nd Phase 1 Trial with INM-755","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Announces First Patient Treated in Phase 1 Trial of RP-L301 Gene Therapy for Pyruvate Kinase Deficiency","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Receives FDA Fast Track Designation for RP-L401 Gene Therapy for Infantile Malignant Osteopetrosis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rocket Pharmaceuticals Announces First Patient Treated in Higher Dose Cohort in Phase 1 Clinical Trial of RP-A501 for Danon Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Announces Completion of Subject Treatments in Second Phase 1 Clinical Trial of INM-755 CBN Cream in Healthy Subjects","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants AT-007 Pediatric Rare Disease Designation and Orphan Designation for Treatment of PMM2-CDG","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Applied Therapeutics"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Roth Capital Partners","pharmaFlowCategory":"D","amount":"$8.0 million","upfrontCash":"Undisclosed","newsHeadline":"InMed Pharmaceuticals Announces the Closing of US$8 Million Public Offering","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"Rocket Pharmaceuticals","sponsor":"California Institute for Regenerative Medicine","pharmaFlowCategory":"D","amount":"$3.7 million","upfrontCash":"Undisclosed","newsHeadline":"Rocket Pharma Gets Funding from the California Institute for Regenerative Medicine for Phase 1 Clinical Trial of RP-L401 for Infantile Malignant Osteopetrosis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Rocket Pharmaceuticals"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"D","amount":"$4.0 million","upfrontCash":"Undisclosed","newsHeadline":"InMed Announces Closing of US$4.5 Million Private Placement","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"February 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"Precigen","sponsor":"National Institutes of Health","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Precigen Receives FDA Orphan Drug Designation for PRGN-2012 AdenoVerse\u2122 Immunotherapy for Recurrent Respiratory Papillomatosis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Vaccine","productStatus":"New Molecular Entity","date":"March 2021","url1":"","url2":"","graph1":"Vaccine","graph2":"Precigen"},{"orgOrder":0,"company":"InMed Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"InMed Pharma Submits Clinical Trial Applications to Evaluate INM-755 (Cannabinol) Cream in a Phase 2 Trial for Epidermolysis Bullosa","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CANADA","productType":"Small molecule","productStatus":"Approved","date":"April 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"InMed Pharmaceuticals"},{"orgOrder":0,"company":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Saniona Initiates Phase 1 Clinical Trial of SAN711","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Saniona"},{"orgOrder":0,"company":"Beckley Psytech","sponsor":"Integrated","pharmaFlowCategory":"D","amount":"$80.0 million","upfrontCash":"Undisclosed","newsHeadline":"Beckley Psytech Completes Oversubscribed $80m (\u00a358m) Fundraise to Develop Portfolio of Psychedelic Medicine Breakthroughs","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"August 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Beckley Psytech"},{"orgOrder":0,"company":"Saniona","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Saniona Initiates Multiple Ascending Dose Stage of SAN711 Phase 1 Clinical Trial","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"DENMARK","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Saniona"},{"orgOrder":0,"company":"Xentria","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Xentria to Present Initial Findings from Preclinical Study of XTMAB-16 at ATS 2022 International Conference","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Xentria"},{"orgOrder":0,"company":"CANbridge Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"CANbridge CAN106 Phase 1 Data Presented at the European Hematology Association 2022 Congress","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"CHINA","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"CANbridge Pharmaceuticals"},{"orgOrder":0,"company":"Acer Therapeutics","sponsor":"Relief Therapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Acer Therapeutics and Relief Therapeutics Announce ACER-001 IND Submission for the Treatment of Maple Syrup Urine Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"Approved","date":"July 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Acer Therapeutics"},{"orgOrder":0,"company":"Omeros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Orphan Drug Designation to Omeros\u2019 MASP-3 Inhibitor OMS906 for Treatment of Paroxysmal Nocturnal Hemoglobinuria","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Omeros"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Relief Therapeutics Announces IRB Approval and Initiation of an Investigator Initiated Trial of Nexodyn for Epidermolysis Bullosa at Ann & Robert H. Lurie Children's Hospital of Chicago","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"September 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Relief Therapeutics"},{"orgOrder":0,"company":"Rallybio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Rallybio Announces Positive Phase 1 Single Ascending Dose Results for RLYB116, an Innovative Subcutaneously Injected Inhibitor of Complement Component 5","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Rallybio"},{"orgOrder":0,"company":"Frontera Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Frontera Therapeutics Doses First Patient in Phase 1 Clinical Trial for Gene Therapy FT-001 for the Treatment of Leber Congenital Amaurosis-2","therapeuticArea":"Rare Diseases and Disorders","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":"Frontera Therapeutics"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Benitec Biopharma Enrolls First OPMD Subject into the Clinical Development Program","therapeuticArea":"Rare Diseases and Disorders","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":"Benitec Biopharma"},{"orgOrder":0,"company":"Relief Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Relief Therapeutics Announces Enrollment of First Three Patients in Proof-of-Concept Clinical Trial of RLF-TD011 for the Treatment of Epidermolysis Bullosa","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"SWITZERLAND","productType":"Small molecule","productStatus":"Approved","date":"February 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Relief Therapeutics"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ReCode Therapeutics Announces First Participants Dosed in a Phase 1 Healthy Volunteer Clinical Trial of Novel Disease-Modifying Genetic Medicine, RCT1100 for the Treatment of Primary Ciliary Dyskinesia","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"March 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"ReCode Therapeutics"},{"orgOrder":0,"company":"BPGbio","sponsor":"Debra of America","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"BPGbio Announces Expanded Partnership with debra of America for BPM 31510 for Epidermolysis Bullosa Phase II\/III Trial","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"BPGbio"},{"orgOrder":0,"company":"Omeros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Omeros Corporation Reports Interim Data with Alternative Pathway Inhibitor OMS906 as Monotherapy in Patients with Paroxysmal Nocturnal Hemoglobinuria","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"April 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Omeros"},{"orgOrder":0,"company":"Calico Life Sciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Calico Life Sciences Announces First Participant Dosed in Phase 1b Clinical Study Evaluating ABBV-CLS-7262 for the Treatment of Vanishing White Matter Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Calico Life Sciences"},{"orgOrder":0,"company":"Protara Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Protara Therapeutics Receives Regulatory Clearance from FDA to Commence Phase 2 STARBORN-1 Trial of TARA-002 in Pediatric Patients with Lymphatic Malformations","therapeuticArea":"Rare Diseases and Disorders","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":"Protara Therapeutics"},{"orgOrder":0,"company":"TWi Biotechnology","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"TWi Biotechnology Exploring Promising Treatment for Granuloma Annulare (GA)","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"TAIWAN","productType":"Small molecule","productStatus":"Approved","date":"June 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"TWi Biotechnology"},{"orgOrder":0,"company":"NovelMed Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"NovelMed Phase I Clinical Trial Shows Inhibition of the Alternative Pathway and Preservation of the Classical Pathway A Long-Acting Anti-Properdin Monoclonal Antibody NM3086 for PNH Patients","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"NovelMed Therapeutics"},{"orgOrder":0,"company":"Azitra","sponsor":"ThinkEquity","pharmaFlowCategory":"D","amount":"$7.5 million","upfrontCash":"Undisclosed","newsHeadline":"Azitra, Inc. Announces Pricing of Initial Public Offering","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Azitra"},{"orgOrder":0,"company":"Azitra","sponsor":"ThinkEquity","pharmaFlowCategory":"D","amount":"$7.5 million","upfrontCash":"Undisclosed","newsHeadline":"Azitra, Inc. Announces Closing of Initial Public Offering","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Azitra"},{"orgOrder":0,"company":"MyoPax","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"MyoPax Receives Rare Pediatric Disease Designation from FDA for Breakthrough Regenerative Cell Product in Exstrophy-Epispadias Complex","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"GERMANY","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"MyoPax"},{"orgOrder":0,"company":"Benitec Biopharma","sponsor":"JMP Securities","pharmaFlowCategory":"D","amount":"$30.0 million","upfrontCash":"Undisclosed","newsHeadline":"Benitec Biopharma Inc. Announces Closing of $30 Million Underwritten Public Offering","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Benitec Biopharma"},{"orgOrder":0,"company":"ReCode Therapeutics","sponsor":"Bioluminescence Ventures","pharmaFlowCategory":"D","amount":"$260.0 million","upfrontCash":"Undisclosed","newsHeadline":"ReCode Therapeutics Announces Closing of Extension to Series B Financing","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"ReCode Therapeutics"}]
Find Drugs for Rare Diseases and Disorders in Phase I Clinical Development in UNITED STATES
The proceeds will advance ReCode’s proprietary Selective Organ Targeting lipid nanoparticle pipeline, including Phase 1 trial of RCT1100 for primary ciliary dyskinesia and RCT2100, its cystic fibrosis candidate.
The company intends to use the net proceeds to support the clinical development of BB-301, including the natural history lead-in study and the Phase 1b/2a BB-301 for the treatment of dysphagia associated with oculopharyngeal muscular dystrophy.
Satori-01 (human muscle stem cell) injection isolated from patient's muscle tissue and expanded ex-vivo, which is investigated for the treatment of Exstrophy-Epispadias Complex.
The company intends to use the net proceeds for clinical trials and product development of ATR-12, a proprietary strain of Staphylococcus epidermidis engineered to express therapeutic levels of LEKTI protein to treat netherton syndrome.
The Company intends to use the net proceeds for clinical trials and product development of ATR-12, a proprietary strain of Staphylococcus epidermidis engineered to express therapeutic levels of LEKTI protein to treat netherton syndrome.
Lead Product(s):
Strain of Staphylococcus Epidermidis
NM3086 is an anti-P humanized monoclonal antibody that binds properdin with high affinity. NM3086 blocks both C3b and membrane-attack complex (MAC) formation, which being invetigated for for PNH patients.
AC-1101 (tofacitinib) topical gel phase I study patients with granuloma annulare. The oral formulation of tofacitinib has already been FDA approved to treat such ailments as rheumatoid arthritis.
TARA-002 is an investigational cell-based immunopotentiator, which is being investigated for the treatment of pediatric patients with lymphatic malformations (LMs).
ABBV-CLS-7262 is an eIF2B activator which targets eIF2B, a guanine nucleotide exchange factor that is essential for protein synthesis and a key regulator of the ISR. It is being investigated for vanishing white matter disease.
OMS906 is a humanized mAb which inhibits MASP-3, the key activator of the alternative pathway of complement, it is advancing in clinical programs for paroxysmal nocturnal hemoglobinuria (PNH), complement 3 (C3) glomerulopathy and other related indications.
Market Place
Upload your Marketing & Sales content on your company Virtual Booth, click HERE
