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Find Drugs for Genetic Disease in Phase III Clinical Development in UNITED STATES

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            Lead Product(s): Giroctocogene fitelparvovec

            Therapeutic Area: Genetic Disease Product Name: SB-525

            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 October 07, 2020

            Details:

            AFFINE is a global Phase 3, open-label, multicenter, single arm study that will evaluate the efficacy and safety of giroctocogene fitelparvovec in patients with moderately severe to severe hemophilia A.

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            Lead Product(s): PF-06939926

            Therapeutic Area: Genetic Disease Product Name: PF-06939926

            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 October 01, 2020

            Details:

            The therapy, PF-06939926, is a recombinant adeno-associated virus serotype 9 (rAAV9) capsid carrying a shortened version of the human dystrophin gene under the control of a human muscle-specific promotor.

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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): Berotralstat

            Therapeutic Area: Genetic Disease Product Name: Orladeyo

            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 October 30, 2020

            Details:

            Under the EAMS, hereditary angioedema (HAE) patients in the UK aged 12 years and older can gain access to berotralstat for the routine prevention of recurrent attacks of HAE before the drug is granted marketing authorization by the European Commission (EC).