Tenaya Therapeutics

Tenaya Therapeutics works on 3 rare diseases tracked on Trial Friend, including Arrhythmogenic Cardiomyopathy, Familial Hypertrophic Cardiomyopathy, Peripartum Cardiomyopathy, with 4 recruiting clinical trials.

Tenaya Therapeutics is a clinical-stage biotechnology company developing gene therapies and small molecule treatments for rare genetic heart diseases. Lead programs include TN-201 for MYBPC3-associated hypertrophic cardiomyopathy and TN-401 for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC), which dosed its first patient in the RIDGE-1 Phase 1b/2 trial in November 2024.

Type
Rare Disease Specialist
Ticker
TNYA
Headquarters
South San Francisco, United States
Founded
2016
4
Active Rare Disease Trials
3
Rare Diseases in Portfolio
10
Years Active

Focus areas at Tenaya Therapeutics

As a rare disease specialist, Tenaya Therapeutics has active clinical trial programs and drug development efforts across 3 rare diseases, including Arrhythmogenic Cardiomyopathy, Familial Hypertrophic Cardiomyopathy, Peripartum Cardiomyopathy. 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 Tenaya Therapeutics, 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.

Tenaya Therapeutics is headquartered in South San Francisco, United States, founded in 2016, publicly traded under the ticker symbol TNYA. 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.

Tenaya Therapeutics Drug Pipeline

Tenaya Therapeutics has 4 active clinical trials across 3 development stages, with 4 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 Tenaya Therapeutics'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 Tenaya Therapeutics's pipeline
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1
Early Phase / Phase 11 trial
1
Phase 21 trial
2
Other2 trials
Cardiomyopathy
Recruiting

Tenaya Therapeutics Clinical Trials (4)

Active and recruiting clinical trials sponsored by Tenaya Therapeutics, 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 Tenaya Therapeutics's trials
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ACTIVE NOT RECRUITINGRecently updatedNCT05112237

Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy

Cardiomyopathy

The objective of this study is to collect information on patients with cardiomyopathy (CM) due to mutations in the MYBPC3 gene, to evaluate their disease course, burden of illness, risk factors for this disease, and the quality of life (QoL). This study will also collect information on treatments, procedures and outcome in infants and children up to 18 yrs who have this mutation.

Ages 0 Years - 18 Years29 locations
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RECRUITINGPHASE1, PHASE2No updates in a whileNCT05836259

Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM

Intervention: TN-201

This is a first-in-human, non-randomized, open-label study designed to evaluate the safety, tolerability, and pharmacodynamics (PD) of TN-201 in adult patients with symptomatic hypertrophic cardiomyopathy (HCM) caused by mutations in the MYBPC3 gene.

Ages 18 Years - 75 Years10 locations
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RECRUITINGPHASE1Hasn't posted an update in over a yearNCT06228924

Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC

Intervention: TN-401

This first-in-human study is designed to evaluate the safety, and preliminary efficacy (PD) of TN-401 gene therapy in adult patients with symptomatic PKP2 mutation-associated ARVC.

Ages 18 Years - 65 Years7 locations
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RECRUITINGHasn't posted an update in over a yearNCT06311708

Non-interventional Study of Seroprevalence of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) and the Progression of Disease in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)

This is a multicenter, non-interventional study to observe the natural progression of the disease and to study the prevalence of pre-existing antibodies to AAV9 used for gene therapy in a population of patients with PKP2 gene-associated ARVC. Participation from all patients is encouraged regardless of interest in or eligibility for gene therapy.

Ages 14 Years - 65 Years21 locations
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FDA-Approved Drugs

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

Tenaya Therapeutics Trial Locations

Tenaya Therapeutics clinical trials are running at 67 sites in 8 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
46▼
United Kingdom
7▼
France
4▼
Spain
3▼
Canada
2▼
Germany
2▼
Italy
2▼
Sweden
1▼

Rare Disease Focus Areas (3)

Diseases targeted by Tenaya Therapeutics's clinical trial and drug development programs

Patient Resources

Organizations and resources related to Tenaya Therapeutics's rare disease focus areas

Frequently Asked Questions About Tenaya Therapeutics

Common questions about Tenaya Therapeutics's rare disease programs, clinical trials, and treatments.