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            Lead Product(s): Gene targeted chimeras

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Product Type: Small molecule

            Partner/Sponsor/Collaborator: Renaissance Capital

            Deal Size: $228.0 million Upfront Cash: Undisclosed

            Deal Type: Public Offering March 22, 2021

            Details:

            Design Therapeutics is developing novel small-molecule therapies, called gene targeted chimeras (GeneTACs), that are designed to be disease-modifying and target the underlying cause of inherited nucleotide repeat expansion diseases.

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            Lead Product(s): Undisclosed

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Discovery Product Type: Small molecule

            Partner/Sponsor/Collaborator: Global Blood Therapeutics

            Deal Size: $353.0 million Upfront Cash: Undisclosed

            Deal Type: Licensing Agreement March 16, 2021

            Details:

            These mechanisms of the two licensed novel small moleculeare are distinct and potentially complementary to that of Oxbryta tablets, a novel hemoglobin S polymerization inhibitor approved in the United States for the treatment of SCD in patients ages 12 years and older.