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

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            Lead Product(s): jCell

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Santen Pharmaceutical

            Deal Size: $252.0 million Upfront Cash: $50.0 million

            Deal Type: Licensing Agreement May 08, 2020

            Details:

            Under the agreement, Santen Pharmaceutical will develop and commercialize its first-in-class, investigational therapy, jCell, outside the U.S., in regions including Europe, Asia and Japan.

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            Lead Product(s): Gaboxadol

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 07, 2020

            Details:

            OV101 produced statistically significant reductions in behavioral and functional symptoms in individuals with Fragile X syndrome and met the study’s primary objective of safety and tolerability.

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            Lead Product(s): Sepiapterin

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: PTC Therapeutics

            Deal Size: $526.0 million Upfront Cash: $10.0 million

            Deal Type: Acquisition May 06, 2020

            Details:

            Censa Pharmaceuticals acquisition diversifies and strengthens PTC 's portfolio of rare disorders by adding the lead orphan metabolic disease drug, CNSA-001.

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            Lead Product(s): Trehalose API

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 30, 2020

            Details:

            SLS-005 was previously granted Orphan Drug Designation from the FDA and EMA for Spinocerebellar Ataxia Type 3 and Oculopharyngeal Muscular Dystrophy as well as Fast Track designation for OPMD.

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            Lead Product(s): MS1819

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 29, 2020

            Details:

            The Phase 2 study is designed to investigate the safety, tolerability and efficacy of MS1819 using 2.2 and 4.4 gram doses in enteric capsules head-to-head against the current standard of care.

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            Lead Product(s): Lonafarnib

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 27, 2020

            Details:

            European Medicines Agency has completed its validation of the Marketing Authorization Application for lonafarnib to treat Hutchinson-Gilford Progeria Syndrome and Progeroid Laminopathies.

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            Lead Product(s): Dalcinonacog alfa

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 21, 2020

            Details:

            The open-label Phase 2b study was designed to evaluate the ability of DalcA to maintain steady state protective FIX levels above 12% in six individuals with severe hemophilia B.

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            Lead Product(s): Olinciguat

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 10, 2020

            Details:

            Closed enrollment for olinciguat Phase 2 STRONG SCD study for sickle cell disease; topline data readout expected Q3 2020.

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            Lead Product(s): KVD900

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 07, 2020

            Details:

            The Phase 2 trial for KVD900 as an on-demand oral therapy for HAE attacks remains active, but the company anticipate a delay in timing of data due to the unprecedented worldwide impact of COVID-19.

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            Lead Product(s): Isotretinoin

            Therapeutic Area: Genetic Disease

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: U.S. Food & Drug Administration

            Deal Size: $1.5 million Upfront Cash: $0.5 million

            Deal Type: Funding April 06, 2020

            Details:

            Funds support Phase 2b CONTROL Study of TMB-001 in congenital ichthyosis

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