Regeneron Leads $66M Series B for Actio Biosciences’ Rare Disease Therapeutics

Actio Biosciences secured $66 million in Series B financing, co-led by Regeneron Ventures and Deerfield Management, to advance its genetically targeted therapeutic pipeline for rare and common diseases234.

The funding will support development of two lead candidates:
ABS-1230, a KCNT1 inhibitor for KCNT1-related epilepsy, and ABS-0871, a TRPV4 inhibitor for Charcot-Marie-Tooth disease type 2C (CMT2C)23.

ABS-1230 has received rare pediatric and orphan drug designations from the FDA, with a Phase 1 clinical trial set to initiate in the second half of 20252.

ABS-0871 is currently enrolling in a Phase 1 trial in healthy volunteers for CMT2C23.

Both targeted diseases are ultra-rare, affecting approximately 2,500 individuals each in the United States3.

Existing investors—including Canaan, Droia Ventures, and Euclidean Capital—also participated in this financing round23.

Sources:

2. https://www.biospace.com/press-releases/actio-biosciences-secures-66-million-series-b-financing-to-advance-genetics-driven-pipeline-of-small-molecule-therapeutics-for-rare-and-common-diseases

3. https://www.thepharmaletter.com/pharmaceutical/rare-epilepsy-actio-66-million-series-b

4. https://www.worldpharmaceuticals.net/news/actio-biosciences-secures-66m-funding-for-genetics-driven-drugs/