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

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            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

            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.

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            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

            Details:

            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.

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            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

            Details:

            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.

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

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 March 25, 2020

            Details:

            IB1001 (N-acetyl-L-leucine) is currently being investigated for the symptomatic, and disease-modifying, neuroprotective treatment of NPC in a multinational clinical trial (IB1001-201).

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            Lead Product(s): Setrusumab

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 January 14, 2020

            Details:

            Setrusumab demonstrated a dose dependent increase in both failure load and stiffness at the radius at 12 months, achieving statistical significance in the high dose cohort.

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            Lead Product(s): Tideglusib

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 January 09, 2020

            Details:

            Trial to assess AMO-02 in treatment of congenital myotonic dystrophy to begin patient enrollment in early 2020.