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[{"orgOrder":0,"company":"AMO Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"AMO Pharma Announces Initiation of Planned Pivotal Clinical Trial for Myotonic Dystrophy Following $35m Fund Raise","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"AMO Pharma"},{"orgOrder":0,"company":"Mereo BioPharma Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mereo BioPharma Announces Additional Positive Data from Phase 2b ASTEROID Study of Setrusumab in Adults with Osteogenesis Imperfecta and Provides Update on Regulatory Progress","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"New Molecular Entity","date":"January 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Mereo BioPharma Group"},{"orgOrder":0,"company":"IntraBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"IntraBio Receives FDA Fast Track Designation for Niemann-Pick Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"March 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"IntraBio"},{"orgOrder":0,"company":"Oxford Biomedica","sponsor":"Sanofi","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Sanofi Returns Rights to Gene Therapies to Oxford Biomedica","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"Oxford Biomedica"},{"orgOrder":0,"company":"ReNeuron","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"ReNeuron Announces Further Positive Data in Retinal Clinical Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"ReNeuron"},{"orgOrder":0,"company":"GSK","sponsor":"Orchard Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Orchard Licenses Gene Therapy Technology from GSK","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"GSK"},{"orgOrder":0,"company":"Mereo BioPharma Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mereo BioPharma Receives FDA Rare Pediatric Disease Designation for Setrusumab for the Treatment of Osteogenesis Imperfecta","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Mereo BioPharma Group"},{"orgOrder":0,"company":"IntraBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"IntraBio Reports Positive Data from IB1001 Multinational Clinical Trial for the Treatment of Niemann-Pick disease Type C","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"IntraBio"},{"orgOrder":0,"company":"IntraBio","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"IntraBio Reports Further Detail on Positive Data from IB1001 Multinational Clinical Trial for the Treatment of Niemann-Pick disease Type C","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"Approved","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"IntraBio"},{"orgOrder":0,"company":"Mereo BioPharma Group","sponsor":"Ultragenyx Pharmaceutical","pharmaFlowCategory":"D","amount":"$304.0 million","upfrontCash":"$50.0 million","newsHeadline":"Ultragenyx and Mereo BioPharma Announce Collaboration and License Agreement for Setrusumab in Osteogenesis Imperfecta","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Large molecule","graph2":"Mereo BioPharma Group"},{"orgOrder":0,"company":"Mereo BioPharma Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mereo BioPharma to Host Conference Call Highlighting Top-line Clinical Data from ASTRAEUS Phase 2 Study of Alvelestat","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Mereo BioPharma Group"},{"orgOrder":0,"company":"Mereo BioPharma Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Mereo BioPharma to Host Alvelestat R&D Update Call on October 31, 2022","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Mereo BioPharma Group"},{"orgOrder":0,"company":"30 Technology","sponsor":"ConvaTec","pharmaFlowCategory":"D","amount":"$218.0 million","upfrontCash":"$55.9 million","newsHeadline":"30 Technology's Clinically Validated Antimicrobial Nitric Oxide Platform Acquired by Convatec Group Plc for Several Applications","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"UNITED KINGDOM","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"30 Technology"}]

Find Drugs for Genetic Disease in Phase II Clinical Development in UNITED KINGDOM

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            Details:

            The divestment will allow 30 Technology to accelerate its ultra-broad spectrum NO-generation and rapidly advance its innovative and differentiated platform of NO-generating technologies, including RESP301, a NO-generating liquid designed to release NO in situ.

            Lead Product(s): RESP301

            Therapeutic Area: Genetic Disease Product Name: RESP301

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: ConvaTec

            Deal Size: $218.0 million Upfront Cash: $55.9 million

            Deal Type: Divestment April 19, 2023

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            Alvelestat (MPH966) is an oral drug that is being researched in people for the treatment of severe Alpha-1 antitrypsin deficiency (AATD) and Bronchiolitis Obliterans Syndrome (BOS).

            Lead Product(s): Alvelestat

            Therapeutic Area: Genetic Disease Product Name: MPH-966

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 24, 2022

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            MPH-966 (alvelestat) is an oral drug that is being researched in people with alpha-1 antitrypsin deficiency (AATD) lung disease. Lung disease is the most common impact of AATD in adults.

            Lead Product(s): Alvelestat

            Therapeutic Area: Genetic Disease Product Name: MPH966

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 06, 2022

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            Ultragenyx will lead future global development of setrusumab in both pediatric and adult patients. Mereo granted Ultragenyx an exclusive license to develop and commercialize setrusumab in the US and rest of the world, excluding Europe where Mereo retains commercial rights.

            Lead Product(s): Setrusumab

            Therapeutic Area: Genetic Disease Product Name: BPS-804

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Ultragenyx Pharmaceutical

            Deal Size: $304.0 million Upfront Cash: $50.0 million

            Deal Type: Collaboration December 17, 2020

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            IB1001 demonstrated a statistically significant and clinically meaningful change in both the primary and secondary endpoints.

            Lead Product(s): N-Acetyl-L-Leucine

            Therapeutic Area: Genetic Disease Product Name: IB1001

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 19, 2020

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            IB1001 demonstrated a statistically significant and clinically meaningful improvement in symptoms, functioning, and quality of life in both primary and topline secondary endpoints for both pediatric and adult patients with NPC.

            Lead Product(s): N-Acetyl-L-Leucine

            Therapeutic Area: Genetic Disease Product Name: IB1001

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 30, 2020

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            Details:

            In Mereo’s Phase 2b ASTEROID study, setrusumab demonstrated a dose-dependent bone building effect and a trend of reduction in fractures in addition to being safe and well tolerated in adults with OI.

            Lead Product(s): Setrusumab

            Therapeutic Area: Genetic Disease Product Name: BPS-804

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 24, 2020

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            GSK

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            Details:

            Orchard has entered into this license agreements with GSK for use of their lentiviral stable cell line technology for Orchard’s investigational hematopoietic stem cell gene therapies for Wiskott Aldrich syndrome and transfusion-dependent beta thalassemia.

            Lead Product(s): OTL-103

            Therapeutic Area: Genetic Disease Product Name: OTL-103

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

            Partner/Sponsor/Collaborator: Orchard Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement July 15, 2020

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            The Company reports that the latest data continue to demonstrate the efficacy of the therapy (hRPC stem cell therapy) , with a clinically meaningful benefit being observed at all time-points.

            Lead Product(s): Human retinal progenitor cell therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: 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 June 29, 2020

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            Oxford Biomedica has been informed by Sanofi that following completion of a company-wide portfolio review, it intends to return to OXB the rights to the following ophthalmology gene therapy programmes: SAR422459 for Stargardt disease & SAR421869 for Usher’s Syndrome type 1b.

            Lead Product(s): SAR422459

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Sanofi

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement June 09, 2020

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