Actio Biosciences has received orphan drug designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the FDA for its lead candidate ABS-0871.
ABS-0871 is a TRPV4 inhibitor indicated for the treatment of TRPV4+ Charcot-Marie-Tooth disease subtype 2C (CMT2C), a rare genetic pediatric disorder. The company has partnered with the CMTA-STAR research program, contributing to the development of novel therapeutics for rare genetic diseases.
Actio Biosciences is a precision biotechnology company focused on developing new therapeutics for rare and common genetic diseases. Its platform includes the Rare Disease Target Atlas proprietary target database and expertise in bioinformatics, biology, and chemistry. Its lead program is currently in clinical development for the treatment of rare diseases caused by TRPV4 mutations, including Charcot-Marie-Tooth disease type 2C (CMT2C) and bone diseases such as metabotropic dysplasia.
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