Actio Biosciences

Actio Biosciences works on 1 rare disease tracked on Trial Friend, including KCNT1-Related Epilepsy, with 3 recruiting clinical trials.

Actio Biosciences develops precision medicines for rare genetic disorders by translating genetic insights into small molecule treatments. The company focuses on rare genetic epilepsies and other neurological disorders, with its lead program ABS-1230 targeting KCNT1-associated seizure disorders.

Type
Rare Disease Specialist
Headquarters
San Francisco, United States
3
Active Rare Disease Trials
1
Rare Diseases in Portfolio

Focus areas at Actio Biosciences

As a rare disease specialist, Actio Biosciences has active clinical trial programs and drug development efforts across 1 rare diseases, including KCNT1-Related Epilepsy. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.

The clinical trials section below shows all active and recruiting studies sponsored by Actio Biosciences, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites. The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.

Actio Biosciences is headquartered in San Francisco, United States. The company dedicates its research and development resources exclusively to rare and orphan diseases, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.

Actio Biosciences Drug Pipeline

Actio Biosciences has 3 active clinical trials across 3 development stages, with 3 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.

Note: This pipeline includes all of Actio Biosciences's active interventional trials, not only those targeting rare diseases. We show the full pipeline because a company's broader research activity, therapeutic expertise, and development infrastructure directly shape its ability to advance rare disease programs. A strong overall pipeline often signals deeper clinical operations, faster enrollment capabilities, and greater commitment to bringing new treatments to patients.

Understand Actio Biosciences's pipeline
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1
Early Phase / Phase 11 trial
1
Phase 21 trial
1
Other1 trial
TRPV4 Gene Mutation
Recruiting

Actio Biosciences Clinical Trials (3)

Active and recruiting clinical trials sponsored by Actio Biosciences, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.

Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.

Ask about Actio Biosciences's trials
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RECRUITINGPHASE2Recently updatedNCT07600736

A Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Clinical Activity of ABS-1230 in Pediatric and Young Adult Participants With KCNT1-related Epilepsy

Intervention: ABS-1230, Placebo

This trial will evaluate the safety, tolerability, pharmacokinetics, and clinical activity of ABS-1230 compared with placebo in participants with KCNT1-related epilepsy

Ages 1 Month - 21 Years1 location
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RECRUITINGPHASE1Recently updatedNCT07156201

A Study to Investigate the Safety, Tolerability, and Pharmacokinetics of ABS-1230 Given Orally Compared With Placebo in Healthy Participants Aged 18 to 55 Years

Intervention: ABS-1230, Placebo, Omeprazole, Voriconazole

This first in human trial will evaluate the safety, tolerability, and pharmacokinetics of single ascending doses, multiple ascending doses, and fed and fasted doses of ABS-1230 given orally compared with placebo in adult healthy participants.

Ages 18 Years - 55 Years1 location
View full study
RECRUITINGNo updates in a whileNCT05600764

The Natural History of TRPV4 Neuropathy

TRPV4 Gene Mutation

The goal of this research is to document the natural history of neuropathy in patients with a confirmed genetic mutation in the TRPV4 gene. The investigators are searching for patients willing to participate in a 6-year long study to document the symptoms of TRPV4-associated disease and their progression over time. Participation requires annual study visits at Johns Hopkins for adult and juvenile participants.

Ages 3 Years - 80 Years1 location
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FDA-Approved Drugs

No FDA-approved drugs found for this company at this time.

Actio Biosciences Trial Locations

Actio Biosciences clinical trials are running at 3 sites in 2 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.

United States
2▼
Australia
1▼

Rare Disease Focus Areas (1)

Diseases targeted by Actio Biosciences's clinical trial and drug development programs

Patient Resources

Organizations and resources related to Actio Biosciences's rare disease focus areas

Frequently Asked Questions About Actio Biosciences

Common questions about Actio Biosciences's rare disease programs, clinical trials, and treatments.