Setara Biosciences, Inc. (Setara), a clinical-stage biotechnology company leveraging genetics to develop precision medicines for severe rare diseases, today announced the completion of a $90 million Series A financing. Setara was formed following the closing of Jazz Pharmaceuticals’ acquisition of Actio Biosciences (Actio) for its lead program ABS-1230, an investigational therapy for KCNT1-related epilepsy. Setara intends to advance Actio’s remaining pipeline, led by the clinical-stage small molecule TRPV4 inhibitor, STAR-0871 (formerly ABS-0871), for the treatment of CMT2C and overactive bladder, along with a portfolio of earlier-stage discovery programs. Current investors Deerfield Management, Canaan, Droia Ventures, EcoR1, Euclidean Capital, and Regeneron Ventures participated in the financing, along with new shareholder Jazz Pharmaceuticals as part of the Actio acquisition.
Setara brings together an experienced team and a proven genetics-driven approach to design and development of small molecule precision medicines that address the underlying causes of disease. Building on an approach pioneered, refined, and successfully scaled at Actio, Setara is working to advance a pipeline of precision medicines with the potential to transform the lives of patients. Setara’s “one to many” strategy starts with rare diseases, where human genetics can provide a clear understanding of disease biology, and then also applies those insights to identify opportunities to treat broader patient populations with more prevalent conditions.
“With Setara, we have the opportunity to build on a model that has already demonstrated its potential and apply it to severe, rare diseases with significant unmet need today,” said David Goldstein, Ph.D., co-founder, president and executive chair of Setara. “We are incredibly proud of what we built at Actio and that ABS-1230 for KCNT1-related epilepsy now has the opportunity to advance as part of Jazz. Setara allows us to take the next step in our journey, to carry forward a pipeline of promising programs for rare diseases, with the potential to ultimately reach much broader patient populations.”
Lead Program Targets TRPV4: Launching Two Clinical Trials in Patients
Setara’s pipeline is led by STAR-0871, a potential first-in-class oral small molecule TRPV4 inhibitor for the treatment of TRPV4-related neuromuscular disease, including CMT2C and overactive bladder. CMT2C is a rare inherited disorder that affects motor and sensory functions with a U.S. prevalence of approximately 2,500 individuals. Overactive bladder affects tens of millions of patients in the U.S., and current treatments have only limited efficacy.
CMT2C is caused by gain-of-function mutations in the TRPV4 gene that result in excessive activation of the TRPV4 ion channel. By selectively inhibiting TRPV4, STAR-0871 is designed to directly address the underlying genetic cause of the disease. Given the role of TRPV4 in other biological processes, Setara believes that STAR-0871 may also have therapeutic potential in more prevalent conditions, such as overactive bladder.
Setara has completed the healthy volunteer portion of a Phase 1 clinical trial of STAR-0871. The company plans to expand into a Phase 1b study (THERON) in patients with CMT2C in 2027, and in this context, STAR-0871 was granted Rare Pediatric Disease, Orphan Drug and Fast Track Designations from the U.S. FDA. In parallel, Setara also plans to initiate a Phase 2a study to evaluate STAR-0871 in overactive bladder patients.
In addition to its lead program, Setara is working to advance a portfolio of early-stage discovery programs focused on genetic epilepsies and expansion indications, including migraine.
Leadership and Board of Directors
Setara will be led by Actio founder and human genetics expert David Goldstein, Ph.D., who serves as the company’s president and executive chair while maintaining his position at Jazz at least through the acceptance of the ABS-1230 new drug application submission for KCNT1-related epilepsy. Several members of Actio’s leadership team will also transition to Setara, bringing experience in genetics-driven drug discovery, clinical development, and company building. The full Setara leadership team and board include the following:
Leadership Team
- David Goldstein, Ph.D., co-founder, president and executive chair
- David Breckenridge, Ph.D., co-founder, chief scientific officer
- Pete Condon, chief business officer
- Cayce Denton, chief financial officer
Board of Directors
- David Goldstein, Ph.D., executive chair, co-founder, and president, Setara
- Ben Cravatt, Ph.D., professor and Norton B. Gilula Chair of Chemical Biology in the Department of Chemistry, The Scripps Research Institute
- Jason Fuller, Ph.D., partner, Deerfield Management
- George Golumbeski, Ph.D., partner, Droia Ventures
- Emil Kakkis, M.D., Ph.D., CEO, president and director, Ultragenyx
- Nina Kjellson, general partner, Canaan
- Wheaton Little, Ph.D., chief scientific officer, neuroscience, Jazz Pharmaceuticals
- John McHutchison, AO, M.D., co-founder, Actio Biosciences
- Basheer Zada, principal, EcoR1
“We believe that Actio’s acquisition by Jazz validates the genetics-driven approach to drug discovery and development that underpins Setara,” said Jason Fuller, Ph.D., Setara board member and Partner at Deerfield Management. “Setara brings together experienced operators, a compelling foundation in human genetics, and a promising pipeline grounded in strong biological rationale. We’re excited to support David and the team as they advance Setara’s lead program and pursue the development of new medicines for patients with major unmet medical needs.”
About Setara Biosciences
Setara Biosciences, Inc. is a clinical-stage biotechnology company pushing the boundaries of how scientific insights can translate genetic data into novel precision medicines. The company applies deep expertise in Mendelian genetics, precision drug discovery, and data science to identify targets whose biology underlies rare diseases with high unmet need and may also hold promise for the treatment of more prevalent indications.
Setara is focused on developing small molecule therapies that directly target the underlying cause of disease by correcting the pathogenic overactivity of disease-causing proteins, regardless of an individual’s specific mutation. By developing potentially life-changing medicines for rare diseases, Setara aims to generate deeper insights into target biology that can inform opportunities in more prevalent indications with shared underlying biology. Our mission is to bring meaningful new therapeutics from “One to Many.”
For more information, please visit setarabiosciences.com and follow the company on LinkedIn and X.
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