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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: 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): Eflornithine HCl,Sulindac

            Therapeutic Area: Genetic Disease Product Name: CPP-1X/sul

            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 June 29, 2020

            Details:

            The clinical development of CPP-1X/sul was designed to establish this fixed dose combination product as a potential pharmaco-preventive drug treatment specifically for FAP patients.

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            Lead Product(s): Etranacogene dezaparvovec

            Therapeutic Area: Genetic Disease Product Name: AMT-061

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

            Partner/Sponsor/Collaborator: CSL Behring

            Deal Size: Undisclosed Upfront Cash: $450.0 million

            Deal Type: Licensing Agreement June 24, 2020

            Details:

            The AMT-061 program, currently in Phase 3 clinical trials, could be one of the first gene therapies to provide potentially long-term benefits to patients with hemophilia B.

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            Lead Product(s): Diazoxide

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Guggenheim Securities

            Deal Size: $50.0 million Upfront Cash: Undisclosed

            Deal Type: Public Offering June 24, 2020

            Details:

            Soleno currently intends to use the net proceeds from the offering to fund its current research and development efforts primarily focused on advancing its lead candidate, Diazoxide Choline Controlled-Release tablets for the treatment of Prader-Willi Syndrome.

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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 June 24, 2020

            Details:

            Healthy obese people treated with the weekly formulation of setmelanotide achieved comparable weight loss to those treated with the daily formulation, and both weekly and daily formulations of setmelanotide were observed to be safe and well-tolerated.

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            Lead Product(s): Gaboxadol

            Therapeutic Area: Genetic Disease Product Name: OV101

            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 June 19, 2020

            Details:

            OV101 is believed to be the only delta (δ)-selective GABAA receptor agonist in development and is currently being evaluated in the Company’s pivotal Phase 3 NEPTUNE trial in Angelman syndrome, with topline results expected in the fourth quarter of 2020.

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            Lead Product(s): Nedosiran

            Therapeutic Area: Genetic Disease Product Name: DCR-PHXC

            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 18, 2020

            Details:

            The U.S. Food and Drug Administration has granted rare pediatric disease designation for Dicerna's nedosiran, an investigational RNAi therapy being developed as a once-monthly treatment for primary hyperoxaluria (PH).

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            Lead Product(s): Eflornithine HCl,Sulindac

            Therapeutic Area: Genetic Disease Product Name: CPP-1X/sul

            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 June 18, 2020

            Details:

            Cancer Prevention Pharmaceuticals plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration this month seeking accelerated approval for CPP-IX/sul for the same indication.

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            Lead Product(s): Valoctocogene Roxaparvovec

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 17, 2020

            Details:

            BioMarin Pharmaceutical reports additional data from its previously reported four-year update of an open-label Phase 1/2 study of valoctocogene roxaparvovec. valoctocogene roxaparvovec resulted in 96% mean reduction in Factor VIII usage.

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            Lead Product(s): Daratumumab,Cyclophosphamide,Dexamethasone

            Therapeutic Area: Genetic Disease Product Name: Darzalex

            Highest Development Status: Phase III Product Type: Large molecule

            Partner/Sponsor/Collaborator: Janssen Pharmaceutical

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 13, 2020

            Details:

            The Phase III ANDROMEDA (AMY3001) study of subcutaneous daratumumab utilizing ENHANZE® in combination with cyclophosphamide, bortezomib and dexamethasone for patients with newly diagnosed light-chain amyloidosis met its primary endpoint.

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