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            Lead Product(s): PRAX-562

            Therapeutic Area: Genetic Disease Product Name: PRAX-562

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Praxis Precision Medicines

            Deal Size: Undisclosed Upfront Cash: Undisclosed

            Deal Type: Partnership February 16, 2021

            Details:

            Praxis will utilize the Ciitizen platform to provide deeper insights across disease populations through the analysis of real-world data, initially to inform the development of PRAX-222 and PRAX-562 in SCN2A developmental and epileptic encephalopathy (SCN2A-DEE).

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            Lead Product(s): ASP0367

            Therapeutic Area: Genetic Disease Product Name: MA-0211

            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 October 20, 2020

            Details:

            Preclinical data and results from Astellas’ Phase I healthy volunteer study collectively suggest that ASP0367 could improve exercise intolerance and fatigue in PMM patients by increasing the number and enhancing the function of mitochondria in patient’s cells.

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            Lead Product(s): RBM-007

            Therapeutic Area: Genetic Disease Product Name: RBM-007

            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 July 15, 2020

            Details:

            RBM-007 is a novel nucleic acid medicine (oligonucleotide-based aptamer) developed in-house at RIBOMIC’s research facilities in Tokyo.