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Find Clinical Drug Pipeline Developments & Deals by Rocket Pharmaceuticals

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            Lead Product(s): RP-L301

            Therapeutic Area: Genetic Disease Product Name: RP-L301

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 13, 2020

            Details:

            First patient has received investigational therapy in the open-label, Phase 1 clinical trial of RP-L301, the Company’s lentiviral vector (LVV)-based gene therapy for the treatment of Pyruvate Kinase Deficiency (PKD), a rare monogenic red blood cell disorder.

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            Lead Product(s): RP-L201

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase I/ 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 May 12, 2020

            Details:

            Two oral presentations highlight updates from the company’s Phase 1/2 study of RP-L201 for the treatment of severe LAD-I and the Phase 1/2 study of RP-L102 “Process A” for the treatment of FA.

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            Lead Product(s): RP-A501

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 19, 2020

            Details:

            Researchers evaluated the efficacy of a single intravenous (IV) infusion of recombinant adeno-associated serotype nine capsid containing the human LAMP2B transgene in LAMP2-deficient mice.

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            Lead Product(s): RP-L102

            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 February 24, 2020

            Details:

            The review provides the FA community with an additional resource to better understand the phenomenon and further supports Rocket’s approach of treating patients with RP-L102.

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            Lead Product(s): RP-L201

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase I/ Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Lonza Group

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Expanded Partnership January 30, 2020

            Details:

            The agreement covers development and manufacturing of RP-L201, a phase 1/2 candidate for the treatment of Leukocyte Adhesion Deficiency-I (LAD-I).