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

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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): 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 July 13, 2020

            Details:

            The study will evaluate the safety and optimal dose range of CB-280 when added onto existing therapies for CF patients, including CFTR modulators.

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            Lead Product(s): RP-L301

            Therapeutic Area: Genetic Disease Product Name: RP-L301

            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 13, 2020

            Details:

            First patient has received investigational therapy in the open-label, Phase 1 clinical trial of RP-L301, the Company’s lentiviral vector (LVV)-based gene therapy for the treatment of Pyruvate Kinase Deficiency (PKD), a rare monogenic red blood cell disorder.

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            Lead Product(s): REN001

            Therapeutic Area: Genetic Disease Product Name: REN001

            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 June 23, 2020

            Details:

            U.S. Food and Drug Administration (FDA) Office of Orphan Products Development has granted Orphan Drug Designation to the company’s lead drug candidate, REN001, for the treatment of primary mitochondrial myopathies (PMM).

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            Lead Product(s): Ribitol

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 June 11, 2020

            Details:

            The Phase 1 clinical trial is designed to assess safety, tolerability, pharmacokinetics and food effect of BBP-418 in healthy volunteers.

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            Lead Product(s): FT-4202

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 June 09, 2020

            Details:

            The data, presented at the Virtual Edition of the 25th European Hematology Association Annual Congress, demonstrate a favorable tolerability profile and favorable pharmacokinetic/pharmacodynamic (PK/PD) effects of FT-4202 in patients with SCD.

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            Lead Product(s): ARCT-810

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 June 05, 2020

            Details:

            Arcturus Therapeutics has dosed the first healthy volunteer in a Phase 1 study with ARCT-810, the Company’s messenger RNA (mRNA)-based therapeutic candidate for Ornithine Transcarbamylase (OTC) deficiency.

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            Lead Product(s): CTI-1601

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase I Product Type: Large molecule

            Partner/Sponsor/Collaborator: Zafgen

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Merger May 29, 2020

            Details:

            The merger creates clinical-stage company focused on the development of novel protein replacement therapies for rare diseases Chondrial Therapeutics’ lead asset, CTI-1601, currently being evaluated in Phase 1 clinical trial for Friedreich’s ataxia.

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            Lead Product(s): ET3

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 May 26, 2020

            Details:

            ET3 consists of autologous mobilized peripheral blood stem and progenitor cells transduced with a recombinant lentiviral vector, encoding a bioengineered coagulation factor VIII transgene designed for high‑level expression at low vector copy number.

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            Lead Product(s): FT-4202

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 April 15, 2020

            Details:

            FT-4202 is a potent activator of pyruvate kinase-R that, in preclinical studies, demonstrated the potential to beneficially impact both anemia and vaso-occlusive crises for people living with SCD.

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