Japan

Ciitizen

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JAPAN Japan

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Praxis Precision Medicines Partners With Ciitizen to Improve Patient-Guided Drug Development

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).

Astellas Pharma

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U.S. FDA Grants Fast Track Designation to Astellas Pharma's ASP0367/MA-0211

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.