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

A collaboration with SCORR Marketing

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            Lead Product(s): FT-4202

            Therapeutic Area: Genetic Disease

            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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            Lead Product(s): FT-4202

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: NewCo

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Divestment March 19, 2020

            Details:

            Transaction supports FORMA’s business model transition from early research focus to integrated biopharma company with research, development and commercial capacity.

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            Lead Product(s): RP-A501

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase I Product Type: Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 19, 2020

            Details:

            Researchers evaluated the efficacy of a single intravenous (IV) infusion of recombinant adeno-associated serotype nine capsid containing the human LAMP2B transgene in LAMP2-deficient mice.

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            Lead Product(s): FT-4202

            Therapeutic Area: Genetic Disease

            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 February 20, 2020

            Details:

            FORMA is currently enrolling patients with SCD in a Phase 1 study to evaluate the safety and pharmacokinetics/pharmacodynamics (PK/PD) of FT-4202.

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            Lead Product(s): REN001

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase I Product Type: Gene therapy

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 18, 2020

            Details:

            The company is currently recruiting participants into a Phase 1b clinical trial in the United Kingdom and Spain.

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            Lead Product(s): RGLS4326

            Therapeutic Area: Genetic Disease

            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 February 13, 2020

            Details:

            Regulus is planning to initiate a Phase 1b dosing study in patients with ADPKD in 2020 to evaluate RGLS4326 for safety, pharmacokinetics, and biomarkers of pharmacodynamic activity.

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            Lead Product(s): RTX-134

            Therapeutic Area: Genetic Disease

            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 January 30, 2020

            Details:

            The trial is designed to evaluate the safety and pharmacokinetics of RTX-134 in adult patients with PKU.

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            Lead Product(s): ESB1609

            Therapeutic Area: Genetic Disease

            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 January 06, 2020

            Details:

            ESB1609 demonstrated durable CSF pharmacokinetics and excellent safety & tolerability in a single ascending dose Phase 1 Study.