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TN-201

Phase 1

Hypertrophic Cardiomyopathy | Gene therapy | Cardiovascular |Tenaya Therapeutics, Inc.|Last Updated: Mar 19, 2026

Target and mechanism

Molecular targetMYBPC3
Target classGene
ModalityGene therapy

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDDMC
Total Trials1
Total Enrollment30

FDA Designations

FAST_TRACKORPHAN_DRUGRARE_PEDIATRIC_DISEASE

Clinical trial landscape

TN-201 · 1 trial · 1 indication

Phase 1 1
NCT05836259Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCMHypertrophic Cardiomyopathy
RECRUITING30 Analytics
PHASE1RECRUITING
Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM
Hypertrophic Cardiomyopathy
NCT0583625930
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Study Endpoints

Primary Endpoints

Number and severity of Adverse Events over the course of the study.
5 Years
Number of Serious Adverse Events related to study drug.
5 Years

Secondary Endpoints

Change from baseline to Week 52 in Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS).
52 Weeks
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Cohort 1EXPERIMENTALDose for Cohort 1 will be 3E13 vg/kg
Cohort 2EXPERIMENTALDose for Cohort 2 will be 6E13 vg/kg

Interventions

NameTypeDescription
TN-201GENETICTN-201 is a recombinant adeno-associated virus serotype 9 (AAV9) containing Myosin Binding Protein C (MYBPC3) transgene. It is a single (one-time) intravenous dose.
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Eligibility Criteria

Age Range18 Years to 75 Years
SexALL
Healthy VolunteersNo
Study Sites10

Inclusion Criteria: * MYBPC3 mutation * Hypertrophic Cardiomyopathy (obstructive and nonobstructive) * Left Ventricular Ejection Fraction ≥45% * NYHA Functional Class II or III symptoms * NT-proBNP ≥160pg/ml Exclusion Criteria: * High AAV9 neutralizing antibody titer

Countries:United States
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Frequently asked questions about TN-201

What is TN-201 used for?

TN-201 is an investigational gene therapy being developed for hypertrophic cardiomyopathy, a condition in which the heart muscle becomes thickened. It is specifically being studied in adults with symptomatic MYBPC3 mutation-associated hypertrophic cardiomyopathy.

What does TN-201 target?

TN-201 targets the MYBPC3 gene, which provides instructions for making a protein important for normal heart muscle function. Mutations in this gene are a common cause of hypertrophic cardiomyopathy. The therapy is designed to address the underlying genetic cause of the condition.

Who makes TN-201?

TN-201 is being developed by Tenaya Therapeutics, Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol TNYA. The company is focused on discovering and developing therapies for cardiovascular diseases.

What phase is TN-201 in?

TN-201 is currently in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities and is still being studied for safety and tolerability in clinical trials.

What clinical trials is TN-201 in?

TN-201 is being studied in a Phase 1 clinical trial with the identifier NCT05836259. This is a multi-center, open-label, single-ascending dose study evaluating the safety and tolerability of TN-201 in adults with symptomatic MYBPC3 mutation-associated hypertrophic cardiomyopathy. The trial is recruiting participants in the United States.

Is TN-201 the same as MYBPC3 gene therapy?

TN-201 is a gene therapy that targets the MYBPC3 gene, but it is not referred to by that name. It is specifically designed to address mutations in the MYBPC3 gene that cause hypertrophic cardiomyopathy. The drug is known only as TN-201.