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Find Drugs for Genetic Disease in Phase III Clinical Development

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            Lead Product(s): Giroctocogene fitelparvovec

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Sangamo Therapeutics

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 18, 2020

            Details:

            All five patients with severe hemophilia A who received the 3e13 vg/kg dose showed sustained factor VIII (FVIII) activity levels, with a median of 64.2% via chromogenic assay (patient-level geometric means after week 9 post-infusion).

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            Lead Product(s): rFVIIIFc-VWF-XTEN,Recombinant Antihemophilic Factor

            Therapeutic Area: Genetic Disease Product Name: BIVV001

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 29, 2020

            Details:

            Presentations include findings on hemophilia and acquired thrombotic thrombocytopenic purpura portfolio therapies and pipeline candidates.

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            Lead Product(s): Givosiran

            Therapeutic Area: Genetic Disease Product Name: ALN-AT3SC

            Highest Development Status: 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 19, 2020

            Details:

            Long-term interim results from the Phase 2 open-label extension (OLE) study reinforce fitusiran’s potential to restore hemostatic balance and to lower annualized bleed rates (ABRs) over a period up to 57 months.

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            Lead Product(s): Avalglucosidase alfa

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 16, 2020

            Details:

            Avalglucosidase alfa showed a 2.4-point improvement in percent-predicted forced vital capacity, an important measure of respiratory function in Pompe disease, compared to alglucosidase alfa (standard of care).

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            Lead Product(s): Avalglucosidase alfa

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 08, 2020

            Details:

            Sanofi will host a virtual scientific session to present data from the Phase 3 COMET trial of investigational enzyme replacement therapy (ERT) avalglucosidase alfa in patients with late-onset Pompe disease (LOPD).

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            Lead Product(s): Avalglucosidase alfa

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 10, 2020

            Details:

            In patients with late-onset Pompe disease (LOPD), 5.5 years of safety and exploratory efficacy results showed avalglucosidase alfa was well-tolerated and stabilized pulmonary and motor function.

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            Lead Product(s): Vosoritide

            Therapeutic Area: Genetic Disease Product Name: BMN 111

            Highest Development Status: Phase III Product Type: Peptide

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 23, 2020

            Details:

            The marketing application is based on the outcomes from the randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of vosoritide.

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            Lead Product(s): Odevixibat

            Therapeutic Area: Genetic Disease Product Name: A4250

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 20, 2020

            Details:

            Program expands access to odevixibat for patients with progressive familial intrahepatic cholestasis (PFIC), an ultra-rare pediatric cholestatic liver disease.

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            Lead Product(s): AT-GAA

            Therapeutic Area: Genetic Disease Product Name: AT-GAA

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Undisclosed

            Deal Size: $400.0 million Upfront Cash: Undisclosed

            Deal Type: Financing July 17, 2020

            Details:

            The strategic financing allows Amicus to deliver on its mission for patients and shareholders and places it firmly on a path to profitability and its vision to become one of the world’s leading biotechnology companies focused on rare genetic diseases.

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            Lead Product(s): Setmelanotide

            Therapeutic Area: Genetic Disease Product Name: RM-493

            Highest Development Status: Phase III Product Type: Peptide

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 16, 2020

            Details:

            Data from the study demonstrate that treatment with setmelanotide, the company’s melanocortin-4 receptor (MC4R) agonist, reduced body weight and hunger in individuals with BBS.

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