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[{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Unveils New Strategic Plan","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"Freeline Therapeutics","sponsor":"Novo Holdings","pharmaFlowCategory":"D","amount":"$120.0 million","upfrontCash":"Undisclosed","newsHeadline":"Freeline Closes Major $120M Funding Round","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Freeline Therapeutics"},{"orgOrder":0,"company":"Freeline Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Freeline Announces Acceptance of Late Breaking Abstract at the International Society on Thrombosis and Haemostasis (ISTH) 2020 Meeting","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Freeline Therapeutics"},{"orgOrder":0,"company":"Freeline Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"D","amount":"$100.0 million","upfrontCash":"Undisclosed","newsHeadline":"Phase 1\/2 Gene Therapy Biotech Freeline Therapeutics Files for a $100 million US IPO","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Freeline Therapeutics"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"FDA Grants Rare Pediatric Disease Designation to AMO Pharma for AMO-02 for Treatment of Congenital Myotonic Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"November 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"AMO Pharma"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Orchard Therapeutics Announces New England Journal of Medicine Publication of HSC Gene Therapy Data for ADA-SCID","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"May 2021","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Orchard Therapeutics"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AMO Pharma Announces Completion of Enrollment in REACH-CDM Study in Congenital Myotonic Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"AMO Pharma"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AMO Pharma Announces MHRA Confirmation of CDM1-RS as Approvable Primary Outcome Measure in Myotonic Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"AMO Pharma"},{"orgOrder":0,"company":"AMO Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AMO Pharma Announces Completion of Treatment of Last Patient in Pivotal REACH-CDM Clinical Trial in Myotonic Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"AMO Pharma"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Syncona Limited","pharmaFlowCategory":"D","amount":"$120.0 million","upfrontCash":"Undisclosed","newsHeadline":"Beacon Therapeutics Launches with \u00a396 Million ($120 million) to Develop a New Generation of Gene Therapies for Retinal Diseases Resulting in Blindness","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II\/ Phase III","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Beacon Therapeutics"}]

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            Details:

            The financing will fund Beacon's lead asset, AGTC-501, a gene therapy program in Phase II trials for the treatment of X-linked retinitis pigmentosa. AGTC-501 expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP.

            Lead Product(s): Recombinant Adeno-associated Virus Vector

            Therapeutic Area: Genetic Disease Product Name: AGTC-501

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

            Partner/Sponsor/Collaborator: Syncona Limited

            Deal Size: $120.0 million Upfront Cash: Undisclosed

            Deal Type: Financing June 12, 2023

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            AMO-02 (tideglusib), has a dual mechanism disrupting the pathogenic RNA repeat in CDM1 and inhibiting excess levels of the kinase GSK3β. It is in development for the treatment of congenital myotonic dystrophy.

            Lead Product(s): Tideglusib

            Therapeutic Area: Genetic Disease Product Name: AMO-02

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 25, 2023

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            AMO-02 (tideglusib), has a dual mechanism disrupting the pathogenic RNA repeat in CDM1 and inhibiting excess levels of the kinase GSK3β. It is in development for the treatment of congenital myotonic dystrophy.

            Lead Product(s): Tideglusib

            Therapeutic Area: Genetic Disease Product Name: AMO-02

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 27, 2023

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            AMO-02 (tideglusib) is in development for the treatment of congenital myotonic dystrophy and has potential for use in adult-onset myotonic dystrophy, additional CNS, neuromuscular and other orphan indications.

            Lead Product(s): Tideglusib

            Therapeutic Area: Genetic Disease Product Name: AMO-02

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 05, 2022

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            Details:

            The investigational gene therapy products, including OTL-101 resulted in overall survival of 100% through the end of follow-up. At one year, event-free survival was 97% in U.S. study patients and 100% in UK study patients.

            Lead Product(s): OTL-101

            Therapeutic Area: Genetic Disease Product Name: OTL-101

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 11, 2021

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            Details:

            Designation qualifies AMO-02 for fast track review and AMO Pharma to receive a priority review voucher pending marketing approval. AMO-02, AMO-01 and AMO-04 are investigational medicines that have not yet been approved for the treatment of patients anywhere in the world.

            Lead Product(s): Tideglusib

            Therapeutic Area: Genetic Disease Product Name: AMO-02

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable November 09, 2020

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            Details:

            The company's lead candidate, FLT180a, an investigational gene therapy medicinal product candidate for the treatment of hemophilia B, is currently in a Phase 1/2 trial and has dosed 10 patients as of June 15, 2020.

            Lead Product(s): FLT180a

            Therapeutic Area: Genetic Disease Product Name: FLT180a

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

            Partner/Sponsor/Collaborator: Undisclosed

            Deal Size: $100.0 million Upfront Cash: Undisclosed

            Deal Type: Public Offering July 17, 2020

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            Details:

            Freeline expects to use the proceeds from the financing to bring its lead program in Haemophilia B into a pivotal trial, as well as to continue its Phase 1/2 clinical program for Fabry Disease and further progress its pipeline programs for Gaucher Disease and Haemophilia A.

            Lead Product(s): FLT180a

            Therapeutic Area: Genetic Disease Product Name: FLT180a

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

            Partner/Sponsor/Collaborator: Novo Holdings

            Deal Size: $120.0 million Upfront Cash: Undisclosed

            Deal Type: Series C Financing June 30, 2020

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            Details:

            Updated data will be presented in an oral presentation on the efficacy and safety of AAV gene therapy FL180a in the B-AMAZE study in severe haemophilia B patients.

            Lead Product(s): FLT180a

            Therapeutic Area: Genetic Disease Product Name: FLT180a

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 29, 2020

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            Details:

            Orchard Therapeutics to establish OTL-200, OTL-103, OTL-203 and OTL-201 programs as top near-term priorities and reduce investment in OTL-101and OTL-300.

            Lead Product(s): OTL-101

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 07, 2020

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