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TN-401

Phase 1

Arrhythmogenic Right Ventricular Cardiomyopathy | Gene therapy | Cardiovascular |Tenaya Therapeutics, Inc.|Last Updated: Feb 6, 2025

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDDMC
Total Trials1
Total Enrollment15

FDA Designations

No designations recorded

Clinical trial landscape

TN-401 · 1 trial · 1 indication

Phase 1 1
NCT06228924Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVCArrhythmogenic Right Ventricular Cardiomyopathy
RECRUITING15 Analytics
PHASE1RECRUITING
Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC
Arrhythmogenic Right Ventricular CardiomyopathyUnlock trial analytics

Study Endpoints

Primary Endpoints

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

Secondary Endpoints

To assess changes in daily PVC and NSVT counts
Week 52
To assess frequency of ICD therapy administration
Week 52
To assess frequency of sustained VT
Week 52
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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-401GENETICTN-401 is a recombinant adeno-associated virus serotype 9 (AAV9) gene therapy containing Plakophilin-2 (PKP2) transgene. It is a single (one-time) intravenous dose.
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Eligibility Criteria

Age Range18 Years to 65 Years
SexALL
Healthy VolunteersNo
Study Sites7

Inclusion Criteria: * PKP2 mutation (pathogenic or likely pathogenic) * Arrhythmogenic Right Ventricular Cardiomyopathy as defined by the 2010 revised Task Force Criteria * Left Ventricular Ejection Fraction ≥50% * Functioning Implantable Cardiac Defibrillator with remote integration capabilities a...

Countries:United States
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Competitive Landscape -Arrhythmogenic Right Ventricular Cardiomyopathy 3 trials

CompanyTickerTrialsLead PhaseDrugs
Lexeo Therapeutics, Inc.LXEO3PHASE1LX2020
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Recent Changes (Last 90 Days)

LOWMay 26, 2026NCT06228924primaryCompletionDate: changed
LOWMay 24, 2026NCT06228924studyFirstPostDate: changed

Frequently asked questions about TN-401

What is TN-401 used for?

TN-401 is an investigational gene therapy being developed for the treatment of Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC), a cardiovascular condition. It is currently in Phase 1 clinical development and is not yet approved by regulatory authorities.

What does TN-401 target?

TN-401 is a gene therapy designed to address ARVC associated with PKP2 gene mutations. It is being studied in adults with PKP2 mutation-associated ARVC, with the goal of providing a therapeutic benefit by targeting the underlying genetic cause of the condition.

Who is developing TN-401?

TN-401 is being developed by Tenaya Therapeutics, Inc., a biopharmaceutical company. The company is conducting clinical trials to evaluate the safety and preliminary efficacy of TN-401 in patients with ARVC.

What phase is TN-401 in?

TN-401 is currently in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by the FDA or any other regulatory agency and is still undergoing clinical trials to assess its safety and efficacy.

What clinical trials is TN-401 in?

TN-401 is being studied in a Phase 1 open-label, dose escalation trial (NCT06228924) in the United States. The trial is recruiting 15 adults aged 18 years and older with PKP2 mutation-associated ARVC to evaluate the safety and preliminary efficacy of the treatment.