You know that feeling when a biotech startup raises $66M, and it doesn’t just feel like a funding round, it feels like a fireworks mortar shot into the sky over San Diego? That’s Actio Biosciences, clinical-stage, precision-driven, and moving with the kind of clarity that only comes from translating the deepest layers of human genetics into something the rest of us can actually use, medicine.
Founded in 2021 by David Goldstein, Ph.D. and John McHutchison, M.D., Actio wasn’t built on vibes, it was built on decades of data, discovery, and the kind of experience that doesn’t need a hype man. Goldstein’s been decoding genomes since half of biotech was still guessing, and McHutchison’s R&D resume reads like a Gilead retrospective. Together, they didn’t set out to chase trends, they set out to solve rare genetic diseases by going straight to the biological source code.
Fast forward to today, $66 million in fresh Series B capital, co-led by Regeneron Ventures and Deerfield Management. Props also to Canaan, DROIA Ventures, and Euclidean Capital for doubling down and staying loud in the cap table. That brings Actio to $121M in total funding, and zero fluff in their pipeline.
Let’s talk targets. ABS-0871, a TRPV4 inhibitor, is now in Phase 1 trials for Charcot-Marie-Tooth disease type 2C, a rare but devastating condition. First patient dosed this March. ABS-1230 is gearing up for clinical trials in KCNT1 related epilepsy, with plans to move from bench to bedside by late 2025. Both assets are first-in-class, oral small molecules aimed at not just slowing disease but redefining what treatment looks like for patients with zero other options.
This isn’t about high-concept science fiction, it’s about platform meets purpose. Actio’s Rare Disease Target Atlas, a database of ~4,000 Mendelian disorders, is turning pure bioinformatics into real, functional therapies. This team knows how to run the game from variant to validated target, and they’re using rare diseases as a precision launchpad for more common conditions built on the same broken pathways.
Behind the scenes, Dimitrios Arkilo steps in as Chief Medical Officer, bringing a neurologist’s eye and a biotech strategist’s mind. Samuel Collins, now a consultant, helped get the ship out of port. On the board, Emil Kakkis of Ultragenyx joins McHutchison, adding even more firepower to a group that already punches above its weight class.
