CSBio CSBio

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Applicable","newsHeadline":"SparingVision Reports Positive Initial Safety Data from the first cohort treated in its PRODYGY Phase I\/II Gene Therapy Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"August 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"SparingVision"},{"orgOrder":0,"company":"Ocugen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ocugen Announces Positive Clinical Study Update from the Phase 1\/2 Trial of OCU400, a Modifier Gene Therapy Product Candidate, for the Treatment of Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular 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FDA Orphan Drug Designation for DYNE-101 for the Treatment of Myotonic Dystrophy Type 1","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Dyne Therapeutics"},{"orgOrder":0,"company":"Nexcella","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"U.S. Food and Drug Administration Approves Orphan Drug Designation for Nexcella NXC-201 as a Treatment for Amyloid Light Chain (AL) Amyloidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Nexcella"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Taysha Gene Therapies Announces Second Patient Dosed with TSHA-102 in the REVEAL Phase 1\/2 Adult Trial for the Treatment of Rett Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","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":"AM-Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AM-Pharma Announces Updated Clinical Development Strategy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"NETHERLANDS","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"AM-Pharma"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"JCR Pharmaceuticals Announces 52-Week Interim Data from its Global Phase I\/II Study of JR-171 in Individuals with Mucopolysaccharidosis Type I (MPS I)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"JCR Pharmaceuticals"},{"orgOrder":0,"company":"Resilience","sponsor":"BridgeBio Pharma","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"BridgeBio and Resilience Announce Strategic Multi-Year Partnership to Advance BBP-631, BBP-812 and Future Gene Therapy Treatments","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Resilience"},{"orgOrder":0,"company":"Freeline Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Freeline Reports Positive Initial Clinical Data from First Cohort of Phase 1\/2 GALILEO-1 Trial of FLT201, Its Novel Gene Therapy Candidate, in Gaucher Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Freeline Therapeutics"},{"orgOrder":0,"company":"SwanBio Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"SwanBio Advances to Higher-Dose Cohort in First-in-Human Study of Gene Therapy for Adrenomyeloneuropathy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"SwanBio Therapeutics"},{"orgOrder":0,"company":"AM-Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AM-Pharma Announces Positive Clinical Data From Phase 1b Study Evaluating Ilofotase Alfa in Hypophosphatasia Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"NETHERLANDS","productType":"Large molecule","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"AM-Pharma"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Intellia Therapeutics Receives Priority Medicines (PRIME) Designation from the European Medicines Agency for NTLA-2002, an Investigational In Vivo CRISPR Genome Editing Treatment for Hereditary Angioedema","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Intellia Therapeutics"},{"orgOrder":0,"company":"Lexeo Therapeutics","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","amount":"$100.0 million","upfrontCash":"Undisclosed","newsHeadline":"Lexeo Therapeutics Announces Pricing of Initial Public Offering","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Lexeo Therapeutics"},{"orgOrder":0,"company":"Medicinova","sponsor":"Sanofi","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"MediciNova's Collaborator Initiates Clinical Development of a Gene Therapy Product for the Treatment of Phenylketonuria","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Medicinova"},{"orgOrder":0,"company":"Solid Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Solid Biosciences Announces IND Clearance by FDA for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Solid Biosciences"},{"orgOrder":0,"company":"Intellia Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Intellia Therapeutics Receives European Union Orphan Drug Designation for NTLA-2002, an Investigational In Vivo CRISPR Genome Editing Treatment for Hereditary Angioedema","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"November 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Intellia Therapeutics"},{"orgOrder":0,"company":"Ionis Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ionis Shares Positive Clinical Update from Ongoing Trial of ION582 for Angelman syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Ionis Pharmaceuticals"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","amount":"$100.0 million","upfrontCash":"Undisclosed","newsHeadline":"Wave Life Sciences Prices $100 Million Public Offering of Ordinary Shares","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Wave Life Sciences"},{"orgOrder":0,"company":"JCR Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"JCR Pharmaceuticals Announces First Patient Dosed in a Global Phase I\/II Clinical Trial of JR-441 for the Treatment of MPS IIIA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"JAPAN","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"JCR Pharmaceuticals"},{"orgOrder":0,"company":"4D Molecular Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"4DMT Gains Alignment with FDA on Plan to Lift Clinical Hold on Phase 1\/2 INGLAXA Clinical Trial for 4D-310 for Fabry Disease Cardiomyopathy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"October 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"4D Molecular Therapeutics"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"J.P. 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Therapeutics"},{"orgOrder":0,"company":"Ultragenyx Pharmaceutical","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Ultragenyx Announces Completion of Enrollment in Global Phase 1\/2 Trial of GTX-102 in Pediatric Patients with Angelman Syndrome","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Large molecule","graph2":"Ultragenyx Pharmaceutical"},{"orgOrder":0,"company":"Belief BioMed","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Belief BioMed Successfully Completed Dosing of First Subject in the Registrational Clinical Trial for Hemophilia A","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase 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Announces Dosing Completion of Subjects with Stargardt in Cohort 1 of Phase 1\/2 Clinical Trial Evaluating the Safety and Efficacy of OCU410ST","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"U.S.A","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"February 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Ocugen"}]

Find Drugs for Genetic Disease in Phase I/ Phase II Clinical Development

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            Response to COVID-19 pandemic anticipated to cause interruptions in certain clinical trial operations; updated interim data readout timing to be provided at later date.

            Lead Product(s): MRT5005

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 26, 2020

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            TMF Summit 2024

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            First inhaled mRNA therapeutic remains on track to report results from its ongoing multiple-ascending dose (MAD) portion of Phase 1/2 clinical trial in the third quarter of 2020.

            Lead Product(s): MRT5005

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 26, 2020

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            OCU410ST (AAV5-hRORA) is a modifier gene therapy candidate, which utilizes an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. It is being evaluated for the treatment of patients with Stargardt Disease.

            Lead Product(s): AAV5-hRORA

            Therapeutic Area: Genetic Disease Product Name: OCU410ST

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 22, 2024

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            Kresladi (marnetegragene autotemcel) is an investigational gene therapy that acts as a vector to deliver a functional copy of the ITGB2 gene. It is being developed for the treatment of severe leukocyte adhesion deficiency-I (LAD-I).

            Lead Product(s): Marnetegragene Autotemcel

            Therapeutic Area: Genetic Disease Product Name: Kresladi

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 13, 2024

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            The Company intends to use the proceeds to fund the ongoing clinical development of DYNE-101, an experimental treatment for Duchenne muscular dystrophy and DYNE-251, including the acceleration of activities related to expedited regulatory pathways for DYNE-101 and DYNE-251.

            Lead Product(s): DYNE-101

            Therapeutic Area: Genetic Disease Product Name: DYNE-101

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Morgan Stanley

            Deal Size: $345.1 million Upfront Cash: Undisclosed

            Deal Type: Public Offering January 11, 2024

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            The Company intends to use the proceeds to fund the ongoing clinical development of DYNE-101, an experimental treatment for Duchenne muscular dystrophy and DYNE-251, including the acceleration of activities related to expedited regulatory pathways for DYNE-101 and DYNE-251.

            Lead Product(s): DYNE-101

            Therapeutic Area: Genetic Disease Product Name: DYNE-101

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Morgan Stanley

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Public Offering January 03, 2024

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            GTX-102 is an investigational antisense oligonucleotide which is designed to target and inhibit expression of UBE3A antisense transcript (UBE3A-AS). It is under phase 1/2 clinical development for the treatment of pediatric patients with Angelman Syndrome.

            Lead Product(s): GTX-102

            Therapeutic Area: Genetic Disease Product Name: GTX-102

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 03, 2024

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            BBM-H803 is an AAV-based gene therapy. It is administered intravenously to deliver the coagulation factor Ⅷ gene into the body of patients with hemophilia A,

            Lead Product(s): BBM-H803

            Therapeutic Area: Genetic Disease Product Name: BBM-H803

            Highest Development Status: Phase I/ Phase II Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 03, 2024

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            DYNE-101 is an investigational therapeutic which consists of an antisense oligonucleotide conjugated to a fragment antibody (Fab) that binds to the transferrin receptor 1 (TfR1). It is being evaluated in the Phase 1/2 global clinical trial for people living with DM1.

            Lead Product(s): DYNE-101

            Therapeutic Area: Genetic Disease Product Name: DYNE-101

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 03, 2024

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            Details:

            The net proceeds from this offering will be used to advance RNA editing pipeline of programs, including WVE-006 and to accelerate the development of Wave's INHBE program into the clinical.

            Lead Product(s): WVE-006

            Therapeutic Area: Genetic Disease Product Name: WVE-006

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: J.P. Morgan

            Deal Size: $100.0 million Upfront Cash: Undisclosed

            Deal Type: Public Offering December 07, 2023

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