Shots:

• The US FDA has granted orphan drug designation & rare pediatric disease designation to the company’s ABS-0871 for treating TRPV4+ Charcot-Marie-Tooth disease subtype 2C (CMT2C)
• The preclinical TRPV4 rare disease models showed significant improvements in motor function and mobility with this program compared to untreated controls
• Actio’s ABS-0871 is a small molecule that targets TRPV4 mutations and is being developed for treating CMT2C and TRPV4 related bone diseases