Find Drugs for Genetic Disease in Phase I Clinical Development

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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): ZF874

            Therapeutic Area: Genetic Disease Product Name: ZF874

            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 August 19, 2020

            Details:

            First human volunteer was dosed with ZF874, company's novel treatment for alpha-1-antitrypsin deficiency (AATD). ZF874 is a novel compound that acts as a molecular ‘patch’ for the faulty protein, allowing it to fold correctly.

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            Lead Product(s): ETD002

            Therapeutic Area: Genetic Disease Product Name: ETD002

            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 August 17, 2020

            Details:

            ETD002. The first-in-man safety study is being conducted in healthy participants with ETD002, a TMEM16A potentiator aimed at treating all people with cystic fibrosis (CF).

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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): PHA121

            Therapeutic Area: Genetic Disease Product Name: PHA121

            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 July 30, 2020

            Details:

            The data set found in the publication demonstrates the preclinical profile of the new class of molecules relative to icatibant, an injectable B2-receptor antagonist used as the leading therapy for on-demand treatment of HAE.

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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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            Lead Product(s): RBM-007

            Therapeutic Area: Genetic Disease Product Name: RBM-007

            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 15, 2020

            Details:

            RBM-007 is a novel nucleic acid medicine (oligonucleotide-based aptamer) developed in-house at RIBOMIC’s research facilities in Tokyo.

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