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

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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.

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            Lead Product(s): VY-HTT01

            Therapeutic Area: Genetic Disease Product Name: VY-HTT01

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

            Partner/Sponsor/Collaborator: Sanofi

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 12, 2020

            Details:

            Voyager submitted the IND for VY-HTT01 in September 2020 and has been notified that the IND was placed on clinical hold pending the resolution of certain chemistry, manufacturing and controls (CMC) matters.

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            Lead Product(s): FTX-6058

            Therapeutic Area: Genetic Disease Product Name: FTX-6058

            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 October 05, 2020

            Details:

            This Phase 1 trial will evaluate the safety, tolerability and pharmacokinetics of FTX-6058 and will be comprised of four parts. Part A will be a randomized, double-blind, placebo-controlled, single ascending dose (SAD) study in up to six cohorts.

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            Lead Product(s): NTLA-2001

            Therapeutic Area: Genetic Disease Product Name: NTLA-2001

            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 September 29, 2020

            Details:

            In Vivo gene knockout and insertion data to be presented at OTS Annual Meeting highlight modularity of Intellia’s platform and potential for variety of single-course therapies, with company’s first systemic treatment (NTLA-2001) expected to enter the clinic by year-end.

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            Lead Product(s): FTX-6058

            Therapeutic Area: Genetic Disease Product Name: FTX-6058

            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 September 25, 2020

            Details:

            Fulcrum has identified FTX-6058, a highly potent small molecule inhibitor of Embryonic Ectoderm Development (EED) capable of inducing robust HbF protein expression in cell and murine models.

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            Lead Product(s): Gene therapy

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

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

            Partner/Sponsor/Collaborator: Versant Ventures

            Deal Size: $45.0 million Upfront Cash: Undisclosed

            Deal Type: Series A Financing September 16, 2020

            Details:

            Graphite Bio is focused on correcting defective genes by high-efficiency site-specific integration of new genetic sequences. Graphite Bio’s gene editing platform includes several complementary technologies that enable targeted and permanent DNA integration.

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            Lead Product(s): ABO-202

            Therapeutic Area: Genetic Disease Product Name: ABO-202

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

            Partner/Sponsor/Collaborator: Taysha Gene Therapies

            Deal Size: $63.0 million Upfront Cash: $7.0 million

            Deal Type: Licensing Agreement September 11, 2020

            Details:

            The agreements grant Taysha worldwide exclusive rights to intellectual property developed by scientists at the University of North Carolina at Chapel Hill (UNC) and Abeona, and Abeona know-how relating to the research, development, and manufacture of ABO-202.

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            Lead Product(s): TSHA-101

            Therapeutic Area: Genetic Disease Product Name: TSHA-101

            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 August 27, 2020

            Details:

            Taysha Gene Therapies has received Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. FDA for TSHA-101, an AAV9-based gene therapy in development for GM2 Gangliosidosis.

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            Lead Product(s): EDIT-301

            Therapeutic Area: Genetic Disease Product Name: EDIT-301

            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 August 24, 2020

            Details:

            U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for EDIT-301, an experimental, autologous cell medicine, being developed as a potentially best-in-class, durable medicine for sickle cell disease.

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            Lead Product(s): AB-G023

            Therapeutic Area: Genetic Disease Product Name: AB-G023

            Highest Development Status: IND Enabling Product Type: Peptide

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable August 20, 2020

            Details:

            US Food and Drug Administration Offices of Pediatric Therapeutics and Orphan Products Development granted a rare pediatric disease designation to AmideBio’s glucagon analog (AB‐G023) for the treatment of congenital hyperinsulinism (CHI).

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