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Find FDA Investigational New Drug (IND) Submissions for Genetic Disease in UNITED STATES

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            Lead Product(s): VERVE-101

            Therapeutic Area: Genetic Disease Product Name: VERVE-101

            Highest Development Status: IND Enabling Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 12, 2021

            Details:

            The new data demonstrate that in vivo base editing of the PCSK9 gene in the liver of non-human primates resulted in durable and consistent lowering of blood LDL-C and blood PCSK9 protein levels following a single course of treatment.

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            Lead Product(s): SIG-005

            Therapeutic Area: Genetic Disease Product Name: SIG-005

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 17, 2020

            Details:

            SIG-005 contains a human cell line genetically modified with a non-viral vector designed to express human a-L-iduronidase (IDUA), an enzyme which is missing or defective in patients with MPS-1.

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            Lead Product(s): AAV-based gene therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: EcoR1 Capital

            Deal Size: $115.0 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing December 16, 2020

            Details:

            Proceeds will accelerate clinical development of multiple gene therapy programs, novel gene therapy product designs, next generation technology platform and state-of-the-art AAV Vector GMP manufacturing capabilities.

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            Lead Product(s): GPH101

            Therapeutic Area: Genetic Disease Product Name: GPH101

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 14, 2020

            Details:

            GPH101 harnesses the power of CRISPR and natural DNA repair mechanisms to precisely insert the correct DNA sequence, with the aim of directly correcting the disease-causing sickle mutation and leading to the production of normal red blood cells.

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            Lead Product(s): PBGM01

            Therapeutic Area: Genetic Disease Product Name: PBGM01

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 10, 2020

            Details:

            This is the first regulatory authorization for the global PBGM01 clinical trial program, the Imagine-1 study, for the treatment of infantile GM1, a rare and often life-threatening CNS disorder with no approved disease-modifying therapies available.

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            Lead Product(s): CIP-137401

            Therapeutic Area: Genetic Disease Product Name: CIP-137401

            Highest Development Status: IND Enabling Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable December 02, 2020

            Details:

            AlloMek’s lead drug, CIP-137401, is a macrocyclic MEK kinase inhibitor with excellent pharmacokinetics and a profile optimized for potency and safety. The U.S. FDA has granted orphan-drug designation to CIP-137401, for the treatment of Neurofibromatosis type 1 (NF1).

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            Lead Product(s): GENV HEM

            Therapeutic Area: Genetic Disease Product Name: GENV HEM

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Geneventiv Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement November 02, 2020

            Details:

            GeneVentiv plans to move the therapy into the clinic mid-2022. The technology, designated as GENV HEM offers a cure for all types of hemophilia patients with or without inhibitors to their missing clotting factors.

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            Lead Product(s): PBGM01

            Therapeutic Area: Genetic Disease Product Name: PBGM01

            Highest Development Status: IND Enabling Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 13, 2020

            Details:

            The study suggests that delivery of an AAV vector optimized to express b-gal directly into the CSF restored b-gal activity in the brain may be effective in modifying and preventing the devastating effects of the genetic disease GM1.