Setara Biosciences Launches With $90 Million Series A and Clinical-Stage Rare Disease Pipeline

Jazz Pharmaceuticals’ acquisition of Actio Biosciences, centered on an epilepsy drug candidate, included plans to spin off Actio’s rare disease assets into a standalone company. That new venture, Setara Biosciences, officially launched on Friday with $90 million in funding to advance a lead therapy through early-stage clinical development.

Actio built its discovery engine on precision medicine, leveraging a human genetics platform to pinpoint disease targets and understand their biological roles. This strategy produced candidates across multiple indications. Setara, headquartered in San Diego, is now advancing a lead program with potential applications in both a rare disease and a more prevalent condition.

Setara’s lead asset, STAR-0871 (previously ABS-0871), targets Charcot-Marie-Tooth disease type 2C, a rare neuromuscular disorder with no FDA-approved treatments. The small-molecule compound inhibits TRPV4, an ion channel protein expressed broadly across the body that regulates multiple physiological processes.

In Charcot-Marie-Tooth disease, TRPV4 gene mutations cause ion channel overactivity that drives peripheral nerve damage. Setara’s drug is designed to selectively inhibit TRPV4, targeting the root cause of the disorder. Because TRPV4 also helps detect bladder fullness, the company is simultaneously exploring the compound for overactive bladder—a condition affecting millions of patients despite limited effective treatment options.

A Phase 1 trial of the TRPV4 inhibitor was already in progress when the Jazz-Actio acquisition was announced in August. Setara reports that the healthy volunteer cohort has now been completed. The study is slated to expand into a Phase 1b trial enrolling Charcot-Marie-Tooth patients next year. Concurrently, the company plans a separate Phase 2a trial in overactive bladder. Setara’s portfolio also includes earlier-stage Actio assets targeting epilepsy and migraine, though the specific targets remain undisclosed.

The most advanced program from Actio’s research is ABS-1230, a small-molecule inhibitor of KCNT1 ion channels developed for KCNT1-related epilepsy, a rare seizure disorder that typically resists standard antiseizure medications. No FDA-approved therapies exist for this condition. Under Actio, ABS-1230 advanced into the Phase 2 segment of a potentially pivotal Phase 1b/2a trial. When the Jazz acquisition closed in September, the deal included $820 million upfront, with up to $500 million more contingent on milestone achievements.

Setara’s Series A financing drew support from existing Actio backers, including Deerfield Management, Canaan, Droia Ventures, EcoR1, Euclidean Capital, and Regeneron Ventures. Jazz Pharmaceuticals also participated as a new investor. David Goldstein, who previously served as Actio’s CEO, now leads Setara as president and executive chair.

“Setara enables us to advance the next phase of our mission—moving a pipeline of promising rare-disease programs forward with the potential to reach significantly broader patient populations,” Goldstein said in a prepared statement.

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