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Find Clinical Drug Pipelines for Rare Diseases and Disorders

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            Lead Product(s): Clemizole hydrochloride

            Therapeutic Area: Rare Diseases and Disorders

            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 27, 2020

            Details:

            This Phase 1 study successfully confirmed that EPX-100 is safe, well-tolerated, and does not show any clinically significant abnormalities among equal numbers of male and female subjects.

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            Lead Product(s): OMNI nuclease

            Therapeutic Area: Rare Diseases and Disorders

            Highest Development Status: Undisclosed Product Type: Large molecule

            Partner/Sponsor/Collaborator: AnGes

            Deal Size: $61.0 million Upfront Cash: Undisclosed

            Deal Type: Series B Financing January 15, 2020

            Details:

            The funding will accelerate novel OMNI gene editing platform into a broad therapeutic product pipeline.

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            Lead Product(s): IDE196

            Therapeutic Area: Rare Diseases and Disorders

            Highest Development Status: Preclinical Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 10, 2020

            Details:

            Preclinical studies will test IDE196, in vitro, to determine whether endothelial cells with GNAQ mutations can recover normal cell function as well as in vivo pharmacological inhibition of PKC.