Setara Biosciences Secures $90 Million Series A Financing to Advance Precision Medicines for Rare Genetic Diseases
Setara Biosciences, Inc., a clinical-stage biotechnology company focused on developing precision medicines for severe rare diseases using human genetics, has announced the successful completion of a $90 million Series A financing. The funding will support the advancement of the company’s clinical and preclinical development pipeline, with an emphasis on therapies designed to address the underlying genetic causes of serious diseases. The financing marks an important milestone for the newly established company as it begins operations with a portfolio of programs previously developed by Actio Biosciences.
Setara was established following the completion of Jazz Pharmaceuticals’ acquisition of Actio Biosciences (Actio), which included Actio’s lead development program, ABS-1230, an investigational treatment for KCNT1-related epilepsy. Under the transaction, Jazz acquired the rights to advance ABS-1230, while Setara was formed to continue developing Actio’s remaining pipeline and leverage its genetics-driven drug discovery platform. The new company will focus on advancing promising therapeutic candidates targeting rare genetic disorders and exploring opportunities to extend those discoveries into more prevalent diseases.
The company’s initial portfolio is led by STAR-0871, formerly known as ABS-0871, a clinical-stage small-molecule inhibitor of transient receptor potential vanilloid 4 (TRPV4). The investigational oral therapy is being developed for Charcot-Marie-Tooth disease type 2C (CMT2C), a rare inherited neuromuscular disorder, and overactive bladder, a more common condition affecting millions of people. In addition to STAR-0871, Setara plans to advance several earlier-stage discovery programs targeting genetic epilepsies and potential expansion indications, including migraine.
The $90 million Series A financing attracted participation from several existing investors, including Deerfield Management, Canaan, Droia Ventures, EcoR1, Euclidean Capital and Regeneron Ventures. Jazz Pharmaceuticals also joined the company’s shareholder base as a new investor in connection with the Actio acquisition. The participation of established biotechnology and life sciences investors provides Setara with financial support and strategic backing as it prepares to move its lead candidate into additional clinical studies and progress its broader research portfolio.
Building on Actio Biosciences’ Genetics-Driven Drug Development Model
Setara is building on the scientific foundation, drug discovery capabilities and operational experience developed at Actio Biosciences. The company’s approach centers on using insights from human genetics to identify disease-causing biological mechanisms and develop small-molecule medicines designed to address those mechanisms directly.
Human genetics can provide valuable evidence about the biological processes responsible for inherited diseases. By studying genetic variations associated with specific conditions, researchers can identify proteins, receptors and molecular pathways that contribute to disease development. These findings can help establish a strong biological rationale for potential drug targets and guide the development of therapies that are more precisely matched to the underlying causes of disease.
Setara intends to apply this strategy through what it describes as a “one to many” development model. The approach begins with rare diseases, where genetic mutations can provide relatively clear evidence of the relationship between a particular biological target and disease progression. Once a target and its role in disease biology are established, the company can investigate whether the same pathway may also be relevant to larger patient populations affected by more common conditions.
This strategy allows the company to use discoveries made in rare genetic disorders as a starting point for broader therapeutic development. Rather than limiting research to a single rare indication, Setara aims to identify additional diseases in which the same biological mechanism may contribute to symptoms or disease progression.
David Goldstein, Ph.D., co-founder, president and executive chair of Setara Biosciences, emphasized the opportunity to continue the scientific and business model established at Actio while expanding its application to diseases with significant unmet medical needs.
“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,” Goldstein said.
He also highlighted the company’s satisfaction with Actio’s progress and the opportunity for its lead epilepsy program to continue development under Jazz Pharmaceuticals. According to Goldstein, the establishment of Setara represents the next stage of the team’s efforts to develop precision medicines for rare diseases while retaining the potential to reach much larger patient populations in the future.
The transition from Actio to Setara provides continuity for several research and development programs while creating a dedicated organization to advance the remaining pipeline. The company will combine scientific expertise in human genetics with experience in medicinal chemistry, clinical development and biotechnology company management.
STAR-0871 Leads Setara’s Clinical Development Pipeline
STAR-0871 is the lead investigational candidate in Setara’s development portfolio. It is a potential first-in-class oral small-molecule inhibitor of TRPV4, a protein that functions as an ion channel and plays a role in several physiological processes.
The company is initially pursuing two clinical development opportunities for STAR-0871: CMT2C and overactive bladder. Although these conditions differ substantially in prevalence and clinical presentation, both provide potential opportunities to investigate the therapeutic benefits of selectively inhibiting TRPV4.
The first development focus is CMT2C, a rare inherited neuromuscular disease associated with gain-of-function mutations in the TRPV4 gene. These mutations can lead to excessive activation of the TRPV4 ion channel, disrupting normal biological processes and contributing to neuromuscular dysfunction.
CMT2C affects both motor and sensory functions. Individuals living with the condition may experience progressive muscle weakness, impaired movement and other neurological complications that can interfere with everyday activities. The disease is estimated to affect approximately 2,500 individuals in the United States, highlighting the relatively small but medically important population that could benefit from a targeted treatment.
Currently, treatment options for CMT2C remain limited, and there is a significant need for therapies capable of addressing the biological mechanisms responsible for the condition. Because the disease is associated with mutations that increase TRPV4 activity, selectively inhibiting the channel represents a potential therapeutic strategy.
STAR-0871 has been designed to inhibit TRPV4 activity and directly target the biological mechanism associated with CMT2C. By focusing on the underlying genetic cause rather than solely addressing symptoms, the investigational therapy could offer a targeted approach to treating the disease.
The company’s development strategy is based on the potential for selective TRPV4 inhibition to provide meaningful therapeutic benefits in patients whose disease is driven by abnormal activation of this ion channel. Further clinical evaluation will be necessary to establish the candidate’s safety, tolerability and potential effectiveness in the intended patient population.
Expanding Development Into Overactive Bladder
In addition to its rare disease focus, Setara is evaluating STAR-0871 for overactive bladder, a common condition that affects tens of millions of people across the United States.
Overactive bladder is characterized by urinary urgency and increased frequency of urination, sometimes accompanied by urge urinary incontinence. The condition can significantly affect quality of life, sleep patterns, social activities and daily functioning. Although several treatment approaches are currently available, existing therapies do not provide adequate symptom control for all patients.
Some individuals experience limited benefits from available medications, while others may discontinue treatment because of tolerability concerns or insufficient symptom improvement. Consequently, there remains a need for new therapeutic options that could provide improved outcomes for people living with the condition.
TRPV4 is involved in several biological processes beyond those associated with inherited neuromuscular disorders. Its activity in relevant physiological pathways provides a scientific rationale for exploring the potential of STAR-0871 in overactive bladder.
Setara believes that selectively inhibiting TRPV4 could offer a new approach to treating this condition. The company’s development strategy will therefore evaluate whether the same molecular target being investigated in CMT2C could also provide therapeutic benefits in a significantly larger patient population.
The overactive bladder program represents an important component of Setara’s “one to many” strategy. By initially establishing the therapeutic relevance of TRPV4 inhibition in a genetically defined rare disease, the company aims to explore the broader potential of the target in more prevalent conditions.
Clinical Development Plans and FDA Designations
Setara has already completed the healthy volunteer portion of a Phase 1 clinical trial evaluating STAR-0871. This represents an important development milestone as the company prepares to expand clinical testing into patient populations.
Following completion of the initial healthy volunteer evaluation, Setara plans to advance STAR-0871 into a Phase 1b clinical study in patients with CMT2C. The planned study, known as THERON, is expected to begin in 2027.
The Phase 1b program will represent an important step in evaluating the investigational therapy in individuals affected by the genetic disorder. Moving into patient studies will allow the company to further investigate the candidate’s clinical profile and assess its potential as a targeted treatment for CMT2C.
STAR-0871 has received three important designations from the US Food and Drug Administration (FDA): Rare Pediatric Disease Designation, Orphan Drug Designation and Fast Track Designation.
These regulatory designations reflect the company’s efforts to advance a potential treatment for a rare disease population with significant unmet medical needs. Each designation serves a different purpose within the FDA’s regulatory framework and may provide benefits intended to support the development and review of qualifying therapies.
Rare Pediatric Disease Designation is intended to encourage the development of treatments for serious or life-threatening diseases that primarily affect children and meet the FDA’s eligibility criteria. Orphan Drug Designation supports the development of medicines for rare diseases and may provide certain development incentives. Fast Track Designation is designed to facilitate the development and expedite the review of therapies intended to address serious conditions with unmet medical needs.
While these designations do not establish that STAR-0871 is safe or effective, they provide regulatory recognition of the potential importance of advancing treatment options for the targeted disease population.
Alongside its CMT2C development program, Setara plans to initiate a Phase 2a clinical study evaluating STAR-0871 in patients with overactive bladder. This parallel development strategy will enable the company to explore the candidate’s potential across two distinct indications.
The planned Phase 2a study will provide an opportunity to investigate the therapeutic potential of TRPV4 inhibition in a broader patient population. Together, the CMT2C and overactive bladder programs form the foundation of STAR-0871’s clinical development strategy.
Broader Discovery Pipeline Targets Genetic Epilepsies and Migraine
Beyond STAR-0871, Setara intends to advance a portfolio of early-stage discovery programs focused on genetic epilepsies and additional potential indications.
Genetic epilepsies represent an important area of unmet medical need, particularly for patients whose seizures are associated with specific inherited genetic abnormalities. These disorders can be difficult to manage, and existing treatment options may not adequately address the underlying molecular mechanisms responsible for disease.
Setara plans to use its genetics-driven research capabilities to identify therapeutic targets and develop potential small-molecule treatments for these conditions. The company’s experience at Actio provides a foundation for continuing research into genetically defined neurological disorders.
The establishment of Setara also follows the transfer of Actio’s lead KCNT1-related epilepsy program, ABS-1230, to Jazz Pharmaceuticals as part of the acquisition. Jazz will have the opportunity to continue advancing that investigational program, while Setara focuses on the remaining research and development portfolio.
Migraine is another potential expansion indication included in the company’s discovery strategy. By investigating additional conditions associated with relevant biological pathways, Setara aims to identify opportunities to expand the clinical and commercial potential of its research platform.
The company’s broader pipeline strategy reflects its intention to develop medicines based on validated genetic and biological insights rather than pursuing targets without a clear connection to disease mechanisms.
Experienced Leadership Team to Guide Setara’s Growth
Setara will be led by David Goldstein, Ph.D., an expert in human genetics and co-founder of Actio Biosciences. Goldstein will serve as president and executive chair of Setara while maintaining his position at Jazz Pharmaceuticals at least through the acceptance of the new drug application submission for ABS-1230 in KCNT1-related epilepsy.
His leadership is expected to provide continuity between Actio’s scientific foundation and Setara’s future development activities. Goldstein brings experience in human genetics and precision medicine, areas that are central to the company’s drug discovery and development strategy.
Several other members of Actio’s leadership team will also transition to Setara, supporting the company’s efforts to maintain its scientific capabilities and operational expertise.
The leadership team includes:
- David Goldstein, Ph.D. – Co-founder, President and Executive Chair: Responsible for leading the company’s overall strategy and development.
- David Breckenridge, Ph.D. – Co-founder and Chief Scientific Officer: Responsible for guiding scientific research and drug discovery activities.
- Pete Condon – Chief Business Officer: Responsible for business development and strategic corporate initiatives.
- Cayce Denton – Chief Financial Officer: Responsible for financial management and corporate finance activities.
The leadership structure combines expertise in human genetics, scientific research, business development and financial management. This experience will be important as Setara advances its clinical programs and manages the financial and operational requirements associated with biotechnology development.
Board of Directors Brings Extensive Biotechnology and Investment Experience
Setara has also established a board of directors comprising leaders with experience in pharmaceutical research, biotechnology company development, clinical medicine, venture investment and scientific innovation.
The board includes:
- David Goldstein, Ph.D., executive chair, co-founder and president of Setara Biosciences.
- Ben Cravatt, Ph.D., professor and Norton B. Gilula Chair of Chemical Biology in the Department of Chemistry at The Scripps Research Institute.
- Jason Fuller, Ph.D., partner at Deerfield Management.
- George Golumbeski, Ph.D., partner at Droia Ventures.
- Emil Kakkis, MD, Ph.D., CEO, president and director of Ultragenyx.
- Nina Kjellson, general partner at Canaan.
- Wheaton Little, Ph.D., chief scientific officer, neuroscience, at Jazz Pharmaceuticals.
- John McHutchison, AO, MD, co-founder of Actio Biosciences.
- Basheer Zada, principal at EcoR1.
The combination of scientific leaders, biotechnology executives and experienced investors is expected to provide strategic guidance as the company develops its pipeline and evaluates additional opportunities.
The board’s expertise spans chemical biology, neuroscience, rare disease drug development, pharmaceutical investment and biotechnology operations. This multidisciplinary experience could help Setara navigate the challenges of clinical development, regulatory engagement, financing and future business development.
Investors Express Confidence in Setara’s Development Strategy
The successful completion of the $90 million Series A financing represents an important endorsement of Setara’s scientific approach and development opportunities.
Jason Fuller, Ph.D., Setara board member and partner at Deerfield Management, said the acquisition of Actio by Jazz Pharmaceuticals provides validation of the genetics-driven drug discovery and development model that forms the foundation of Setara.
“We believe that Actio’s acquisition by Jazz validates the genetics-driven approach to drug discovery and development that underpins Setara,” Fuller said.
He highlighted the combination of experienced biotechnology operators, a strong human genetics foundation and a pipeline supported by biological rationale as important factors behind the company’s potential.
Fuller also expressed support for Goldstein and the Setara team as they work to advance STAR-0871 and pursue additional therapeutic development opportunities for patients with significant unmet medical needs.
The participation of existing Actio investors, together with Jazz Pharmaceuticals, provides continuity as Setara begins its independent operations. The funding is expected to support the company’s clinical development priorities and ongoing discovery activities.
Advancing the Next Generation of Precision Medicines
With $90 million in Series A financing, an experienced leadership team and a clinical-stage lead candidate, Setara Biosciences is positioning itself to build a biotechnology company focused on genetically informed precision medicines.
The company’s immediate priorities include advancing STAR-0871 into planned patient studies for CMT2C and overactive bladder, while continuing to develop its early-stage programs targeting genetic epilepsies and other potential indications.
The planned Phase 1b THERON study in CMT2C in 2027 and the Phase 2a overactive bladder study represent important upcoming milestones. These programs will help determine whether selective TRPV4 inhibition can deliver meaningful clinical benefits across rare and more prevalent conditions.
Setara’s broader ambition is to establish a sustainable drug development model that begins with the genetic understanding of rare diseases and uses those insights to identify opportunities in larger patient populations. By combining human genetics, targeted drug discovery and clinical development, the company aims to address the biological causes of diseases that have historically lacked effective treatment options.
The formation of Setara following Jazz Pharmaceuticals’ acquisition of Actio’s ABS-1230 program also demonstrates how a biotechnology pipeline can continue progressing through a focused organizational structure. With its remaining programs now under Setara’s direction, the company is preparing to build on Actio’s scientific achievements and pursue new opportunities in precision medicine.
As clinical development advances, STAR-0871 will be a central measure of the company’s ability to translate genetic discoveries into potential treatments. Its development across CMT2C and overactive bladder will also test the company’s strategy of extending discoveries from rare genetic conditions into broader therapeutic applications.
Through its new financing, experienced scientific leadership and genetics-based research platform, Setara aims to advance a pipeline of innovative small-molecule medicines and bring new treatment possibilities to patients living with serious diseases and significant 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.”