What rare diseases does Actio Biosciences focus on?

Actio Biosciences has active programs across 1 rare disease: KCNT1-Related Epilepsy. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.

Is Actio Biosciences focused exclusively on rare diseases?

Yes. Actio Biosciences is a rare disease specialist headquartered in San Francisco, United States. The company focuses primarily on developing treatments for rare and orphan diseases, with programs targeting KCNT1-Related Epilepsy. It operates as part of Jazz Pharmaceuticals.

How can I find Actio Biosciences clinical trials for rare diseases?

The clinical trials section on this page shows active and recruiting rare disease trials sponsored by Actio Biosciences, pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.

How do I enroll in an Actio Biosciences clinical trial?

To explore enrollment, find a recruiting trial in the clinical trials list on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.

What FDA-approved rare disease drugs does Actio Biosciences have?

None that we track as of November 2026. No Actio Biosciences label in the FDA's openFDA database matches a rare disease on Trial Friend, which usually means the company has no approved product yet or its approved drugs are for common conditions. In development: the trials list on this page shows what Actio Biosciences is recruiting for now, and the FDA calendar lists any application once a decision date is public.

Actio Biosciences

Rare disease drugmaker · part of Jazz Pharmaceuticals · based in San Francisco, United States · actiobiosciences.com

Actio Biosciences works on KCNT1-Related Epilepsy, one of the rare diseases we track. It has 2 active clinical trials, and all of them are looking for volunteers now.

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.

Actio Biosciences pipeline

Each row lists the diseases Actio Biosciences is studying at that stage, from the ones closest to approval down to the earliest work.

Phase 2

Mid-size studies that check whether a drug works and at what dose.

KCNT1-Related Epilepsy recruiting

Phase 1

First studies in people, mostly checking safety.

KCNT1-Related Epilepsy recruiting

Actio Biosciences clinical trials

These are Actio Biosciences trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.

2 trials looking for volunteers now

Looking for volunteersPhase 2Recently 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

Tests ABS-1230, Placebo

For KCNT1-Related Epilepsy

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 Years2 locations

View full study

Looking for volunteersPhase 1Updated a few months agoNCT07156201

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

Tests ABS-1230, Placebo, Omeprazole, Voriconazole

For KCNT1-Related Epilepsy

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

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More about 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 active and recruiting rare disease 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, a subsidiary of Jazz Pharmaceuticals. 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.

Where Actio Biosciences trials take place

The trials above run at 3 study sites in 2 countries. Tap a country to see its states, cities and hospitals.

United States

2▼

Australia

1▼

Rare diseases Actio Biosciences works on

Each disease page lists every open trial, not only Actio Biosciences's, plus approved treatments.

KCNT1-Related Epilepsy

KCNT1-related epilepsy is a group of rare genetic epilepsies caused by changes in the KCNT1 gene, which makes a potassium channel that helps control how easily brain cells fire.

How common: No population prevalence has been established

Where to get support

Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Actio Biosciences's own site is actiobiosciences.com, and our help paying for treatment page lists drugmaker programs and charity funds.

GeneReviews - KCNT1-Related Epilepsy
KCNT1-Related Epilepsy
KCNT1 Epilepsy Foundation
KCNT1-Related Epilepsy