What rare diseases does Tenaya Therapeutics focus on?

Tenaya Therapeutics has active programs across 2 rare diseases: Arrhythmogenic Cardiomyopathy, Familial Hypertrophic Cardiomyopathy. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.

Is Tenaya Therapeutics focused exclusively on rare diseases?

Yes. Tenaya Therapeutics is a rare disease specialist headquartered in South San Francisco, United States, founded in 2016. The company focuses primarily on developing treatments for rare and orphan diseases, with programs targeting Arrhythmogenic Cardiomyopathy, Familial Hypertrophic Cardiomyopathy.

How can I find Tenaya Therapeutics clinical trials for rare diseases?

The clinical trials section on this page shows active and recruiting rare disease trials sponsored by Tenaya Therapeutics, 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 a Tenaya Therapeutics 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 Tenaya Therapeutics have?

None that we track as of November 2026. No Tenaya Therapeutics 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 Tenaya Therapeutics is recruiting for now, and the FDA calendar lists any application once a decision date is public.

When was Tenaya Therapeutics founded and where is it based?

Tenaya Therapeutics was founded in 2016 and is headquartered in South San Francisco, United States. The company has been active for 10 years. It trades publicly under the ticker symbol TNYA. As a rare disease specialist, the company dedicates its resources to developing therapies for patients with rare and orphan conditions.

Tenaya Therapeutics

Rare disease drugmaker · based in South San Francisco, United States · founded 2016 · ticker TNYA · tenayatherapeutics.com

Tenaya Therapeutics works on Arrhythmogenic Cardiomyopathy and Familial Hypertrophic Cardiomyopathy, 2 of the rare diseases we track. It has 4 active clinical trials, and 3 are looking for volunteers now.

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.

Tenaya Therapeutics pipeline

Each row lists the diseases Tenaya Therapeutics 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.

Familial Hypertrophic Cardiomyopathy recruiting

Phase 1

First studies in people, mostly checking safety.

Arrhythmogenic Cardiomyopathy recruiting

Other studies

Studies with no phase listed, such as registries and natural history studies.

Arrhythmogenic Cardiomyopathy recruiting · Cardiomyopathy

Tenaya Therapeutics clinical trials

These are Tenaya Therapeutics 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.

3 trials looking for volunteers now

Looking for volunteersPhase 1/2No 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

Tests TN-201

For Hypertrophic Cardiomyopathy

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

View full study

Looking for volunteersPhase 1Hasn'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

Tests TN-401

For Arrhythmogenic Right Ventricular Cardiomyopathy

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

View full study

Looking for volunteersHasn'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)

For Arrhythmogenic Right Ventricular Cardiomyopathy

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

View full study

1 trial underway, not taking new volunteers

Underway, not taking new volunteersRecently updatedNCT05112237

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

For 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

View full study

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More about Tenaya Therapeutics

As a rare disease specialist, Tenaya Therapeutics has active clinical trial programs and drug development efforts across 2 rare diseases, including Arrhythmogenic Cardiomyopathy, Familial Hypertrophic 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 active and recruiting rare disease 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.

Where Tenaya Therapeutics trials take place

The trials above run at 67 study sites in 8 countries. Tap a country to see its states, cities and hospitals.

United States

46▼

United Kingdom

7▼

France

4▼

Spain

3▼

Germany

2▼

Italy

2▼

Canada

2▼

Sweden

1▼

Rare diseases Tenaya Therapeutics works on

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

Arrhythmogenic Cardiomyopathy

Arrhythmogenic cardiomyopathy is a rare genetic heart disease characterized by replacement of cardiac myocardium with fibrofatty tissue, particularly affecting the right ventricle.

How common: 1 in 2,000 to 5,000 people

Familial Hypertrophic Cardiomyopathy

Familial hypertrophic cardiomyopathy is a genetic heart muscle disease characterized by inappropriate left ventricular hypertrophy and diastolic dysfunction.

How common: 1 in 500 people; 50% of first-degree relatives of affected individuals inherit the mutation

Where to get support

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

NIH GARD - Arrhythmogenic Cardiomyopathy
Arrhythmogenic Cardiomyopathy and Familial Hypertrophic Cardiomyopathy
Arrhythmogenic Cardiomyopathy Association
Arrhythmogenic Cardiomyopathy
Hypertrophic Cardiomyopathy Association
Familial Hypertrophic Cardiomyopathy

Tenaya Therapeutics

Tenaya Therapeutics pipeline

Tenaya Therapeutics clinical trials

3 trials looking for volunteers now

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

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

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)

1 trial underway, not taking new volunteers

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

More about Tenaya Therapeutics

Where Tenaya Therapeutics trials take place

Rare diseases Tenaya Therapeutics works on

Where to get support

Other companies working on the same diseases

Questions about Tenaya Therapeutics