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Find Drugs for Genetic Disease in Phase I/ Phase II Clinical Development in UNITED STATES

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            Lead Product(s): VTX-801

            Therapeutic Area: Genetic Disease Product Name: VTX-801

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

            Partner/Sponsor/Collaborator: Vivet Therapeutics

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Agreement September 23, 2020

            Details:

            Under the terms of the agreement, Pfizer will provide clinical supply for a Phase 1/2 clinical trial evaluating Vivet’s proprietary, investigational gene therapy, VTX-801, for the potential treatment of Wilson disease, a rare and potentially life-threatening liver disorder.

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            Lead Product(s): AXO-AAV-GM1

            Therapeutic Area: Genetic Disease Product Name: AXO-AAV-GM1

            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 October 09, 2020

            Details:

            AXO-AAV-GM1 is the first gene therapy to enter clinical trials for GM1 gangliosidosis and the company believes that it has the potential to provide meaningful clinical benefit to both Type I and Type II patients.

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            Lead Product(s): SP-420

            Therapeutic Area: Genetic Disease Product Name: SP-420

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 06, 2020

            Details:

            The PK data confirmed that the doses of SP-420 planned for the company's upcoming Phase 1-2 trial in patients with transfusional iron overload (TIO) should provide sufficient drug exposure for the desired therapeutic effect.

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            Lead Product(s): Homocystinase

            Therapeutic Area: Genetic Disease Product Name: ACN00177

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

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 05, 2020

            Details:

            Company will give a poster presentation on the development of ACN00177, a novel engineered human enzyme therapy being investigated for the treatment of Homocystinuria, at the American Society of Human Genetics (ASHG) Virtual Meeting 2020.

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            Lead Product(s): BMN 307

            Therapeutic Area: Genetic Disease Product Name: BMN 307

            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 October 02, 2020

            Details:

            Both the FDA and EMA have granted BMN 307 Orphan Drug Designation. BMN 307 will be evaluated to determine safety and whether a single dose of treatment can restore natural Phe metabolism, increase plasma Phe levels, and enable a normalization of diet in patients with PKU.

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            Lead Product(s): AGLE-102

            Therapeutic Area: Genetic Disease Product Name: AGLE-102

            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 October 01, 2020

            Details:

            AGLE-102 is an extracellular vesicle ("EV") therapy that delivers proteins, genetic material and regenerative healing factors to diseased and damaged tissue. AGLE-102 will be evaluated in DEB patients in a phase 1/2a trial initiating in 2021.

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            Lead Product(s): SGT-001

            Therapeutic Area: Genetic Disease Product Name: SGT-001

            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 October 01, 2020

            Details:

            Updated safety and effectiveness data for all patients SGT-001 dosed to date, modifications to the trial’s protocol, and improvements in the therapy’s manufacturing process allowed the restart of the trial which was put on hold by the FDA in May, 2020.

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            Lead Product(s): LB-001

            Therapeutic Area: Genetic Disease Product Name: LB-001

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

            Partner/Sponsor/Collaborator: Jefferies

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Public Offering September 30, 2020

            Details:

            LogicBio intends to use the net proceeds from the offering to support clinical development of LB-001, to progress the development of its GeneRide and Next Generation Capsid platforms, to expand its pipeline of product candidates into other indications.

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            Lead Product(s): RGX-121

            Therapeutic Area: Genetic Disease Product Name: RGX-121

            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 September 30, 2020

            Details:

            RGX-121 is an investigational one-time gene therapy, designed to use the AAV9 vector to deliver the gene that encodes the iduronate-2-sulfatase (I2S) enzyme directly to the central nervous system (CNS) through intracisternal administration.