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

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            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

            Details:

            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.

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            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

            Details:

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

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            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

            Details:

            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.

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            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

            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.

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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.