Gene therapy breakthrough? first human test of TN-201 for heart muscle disease begins

NCT ID NCT05836259

Recruiting now Disease control Sponsor: Tenaya Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests a new gene therapy called TN-201 in 30 adults with hypertrophic cardiomyopathy caused by a specific genetic mutation (MYBPC3). The therapy is given as a one-time IV infusion and aims to correct the underlying genetic defect. The main goals are to check safety and find the right dose, while also looking at how it affects heart function and symptoms.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: TN-201 (a gene therapy given as a one-time IV infusion)
What this could lead to: If successful, this could lead to a one-time treatment that addresses the root genetic cause of hypertrophic cardiomyopathy, potentially reducing symptoms and improving heart function.
What could go wrong: This is a very early (first-in-human) trial with only 30 participants, so safety and effectiveness are not yet known. Gene therapies can have serious side effects, and it may not work for everyone.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

familial hypertrophic cardiomyopathy hypertrophic cardiomyopathy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HYPERTROPHIC CARDIOMYOPATHY

Contacts and locations

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Study contacts

Contact

Email: •••••@•••••

Locations

Brigham and Women's Hospital
WITHDRAWN

Boston, Massachusetts, 02115, United States
Cleveland Clinic
RECRUITING

Cleveland, Ohio, 44195, United States
Emory University
WITHDRAWN

Atlanta, Georgia, 30322, United States
Hospital of the University of Pennsylvania
WITHDRAWN

Philadelphia, Pennsylvania, 19104, United States
Houston Methodist Hospital
RECRUITING

Houston, Texas, 77030, United States
Mayo Clinic
RECRUITING

Rochester, Minnesota, 55905, United States
Oregon Health & Science University
WITHDRAWN

Portland, Oregon, 97239, United States
The Christ Hospital Physicians - The Ohio Heart and Vascular Center
WITHDRAWN

Cincinnati, Ohio, 45219, United States
UC San Diego Altman Clinical and Translational Research Institute - Center for Clinical Research
RECRUITING

La Jolla, California, 92093, United States
University of California San Francisco
RECRUITING

San Francisco, California, 94117, United States

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