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

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            Lead Product(s): SEL-399

            Therapeutic Area: Genetic Disease Product Name: SEL-399

            Highest Development Status: Phase I Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: AskBio

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 17, 2021

            Details:

            The trial aims to determine the optimal dose of ImmTOR to mitigate the formation of antibodies to AAV8 capsids used in gene therapies.

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            Lead Product(s): RGLS4326

            Therapeutic Area: Genetic Disease Product Name: RGLS4326

            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 10, 2021

            Details:

            The study is designed to evaluate the safety, pharmacokinetics, and changes in levels of polycystin 1 (PC1) and polycystin 2 (PC2) in patients with ADPKD administered RGLS4326 every other week for a total of four doses.

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            Lead Product(s): CRN04894

            Therapeutic Area: Genetic Disease Product Name: CRN04894

            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 04, 2021

            Details:

            This study will assess the safety and tolerability of single and multiple doses of CRN04894 and will measure the effect of CRN04894 on suppression of cortisol, cortisol precursors, and adrenal androgens following exogenous ACTH stimulation.

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            Lead Product(s): Autologous CD34+ cells,TNS9.3.55

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase I Product Type: Cell and Gene therapy

            Partner/Sponsor/Collaborator: Memorial Sloan Kettering Cancer Center

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 11, 2021

            Details:

            The EHA abstract, submitted by Simona Raso, reports that 2 out of 3 Thalassemia patients treated with EGT’s autologous CD34+ cells have sustained dramatic reduction in blood transfusions after 8 and 5 years, respectively.