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

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            Lead Product(s): CB-280

            Therapeutic Area: Genetic Disease Product Name: CB-280

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 07, 2020

            Details:

            The randomized, double-blind, placebo-controlled, multiple ascending dose-escalation study is exploring CB-280 versus placebo in adults with cystic fibrosis and chronic infection with Pseudomonas aeruginosa who are stable on cystic fibrosis medications.

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            Lead Product(s): ARCT-810

            Therapeutic Area: Genetic Disease Product Name: LUNAR-OTC

            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 October 05, 2020

            Details:

            All subjects in cohorts up to 0.3 mg/kg have completed dosing and all study visits. Based on the available preliminary study data, ARCT-810 has been well tolerated at these doses, which are all within the anticipated therapeutic range.

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            Lead Product(s): Gavorestat

            Therapeutic Area: Genetic Disease Product Name: AT-007

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable September 24, 2020

            Details:

            AT-007, a novel CNS penetrant Aldose Reductase inhibitor (recently given the INN scientific name gavorestat) has demonstrated biological activity in a validated model of PMM2-CDG.

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            Lead Product(s): REC-994

            Therapeutic Area: Genetic Disease Product Name: REC-994

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Bayer HealthCare

            Deal Size: $239.0 million Upfront Cash: Undisclosed

            Deal Type: Series D Financing September 09, 2020

            Details:

            Proceeds from the financing will support the clinical development of Recursion’s therapeutic pipeline which is the broadest and deepest pipeline to date of any company operating within the space of digitally-powered drug discovery.

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            Lead Product(s): RP-A501

            Therapeutic Area: Genetic Disease Product Name: RP-A501

            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 September 02, 2020

            Details:

            Rocket Pharmaceuticals has treated the first patient in the higher dose cohort in its open-label, Phase 1 clinical trial of RP-A501, the Company’s adeno-associated viral vector (AAV)-based gene therapy for the treatment of Danon Disease.

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            Lead Product(s): RP-L401

            Therapeutic Area: Genetic Disease Product Name: RP-L401

            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 August 27, 2020

            Details:

            U.S.FDA has granted Fast Track designation to RP-L401, the Company’s lentiviral vector (LVV)-based gene therapy for the treatment of Infantile Malignant Osteopetrosis. The Phase 1 clinical trial of RP-L401 will enroll two pediatric patients, one month of age or older.

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            Lead Product(s): RGLS4326

            Therapeutic Area: Genetic Disease Product Name: RGLS4326

            Highest Development Status: Phase I Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 30, 2020

            Details:

            Regulus Therapeutics announced FDA has granted the company orphan drug designation for RGLS4326, an investigational therapy being studied to treat autosomal dominant polycystic kidney disease (ADPKD).

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            Lead Product(s): CTI-1601

            Therapeutic Area: Genetic Disease Product Name: CTI-1601

            Highest Development Status: Phase I Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 28, 2020

            Details:

            CTI-1601 is a recombinant fusion protein intended to deliver human frataxin into the mitochondria of patients with FA who are unable to produce enough of this essential protein.

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            Lead Product(s): 4D-110

            Therapeutic Area: Genetic Disease Product Name: 4D-110

            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 27, 2020

            Details:

            First patient has been dosed in the Phase 1 clinical trial of 4D-110, a Roche-licensed product candidate, delivered by a single intravitreal injection for Choroideremia.

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            Lead Product(s): CTI-1601

            Therapeutic Area: Genetic Disease Product Name: CTI-1601

            Highest Development Status: Phase I Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 20, 2020

            Details:

            The double-blind, placebo-controlled trial is evaluating the safety, tolerability and pharmacokinetics of single ascending doses of subcutaneously administered CTI-1601 in patients over age 18 with FA.

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