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TN-201

Phase 1

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

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment30
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05836259Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCMPHASE1 RECRUITING 30 — — Aug 10, 2023Aug 1, 2032Mar 19, 202610 United States
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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 — 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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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05836259primaryCompletionDate: changed
LOWMay 24, 2026NCT05836259studyFirstPostDate: changed