Tenaya Therapeutics Overview

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Tenaya Therapeutics

Tenaya Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to discovering and developing potentially curative therapies for heart disease. By integrating three distinct product platforms—gene therapy, cellular regeneration, and precision medicine—the company targets both rare genetic cardiomyopathies and more prevalent forms of heart failure. Tenaya leverages an adeno-associated virus (AAV) engineering engine to optimize delivery to the heart, aiming to address the underlying drivers of cardiac disease rather than just managing symptoms.

• Company Snapshot: Founded 2016; HQ South San Francisco, California; Public (NASDAQ: TNYA); https://www.tenayatherapeutics.com
• Scale & Stage: ~97 employees; Market Cap ~$124.25M; Clinical stage
• Research Focus: Genetic cardiomyopathies (HCM and ARVC) and Heart Failure with preserved Ejection Fraction (HFpEF)
• Therapeutic Approaches: AAV9 gene therapy, small molecule HDAC6 inhibitors, and cellular reprogramming
• Clinical Footprint: 4 active trials as lead sponsor, targeting 445 total participants across 67 sites globally

Therapeutic Focus

Tenaya Therapeutics focuses on three primary scientific pillars to combat heart disease: Gene Therapy, Cellular Regeneration, and Precision Medicine. The company's strategy involves delivering functional genes to replace defective ones, regenerating heart muscle cells (cardiomyocytes) lost to injury, and using small molecules to correct specific signaling pathways. This multi-modality approach allows Tenaya to address a broad spectrum of cardiovascular conditions, from rare pediatric genetic disorders to heart failure resulting from myocardial infarction.

• Primary Focus Areas:
  • Genetic Cardiomyopathies: Targeting MYBPC3-associated hypertrophic cardiomyopathy (HCM) and PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC).
  • Heart Failure: Addressing HFpEF through precision medicine and exploring cardiac regeneration for post-infarction recovery.
• Strategy & Capabilities: Tenaya utilizes a proprietary AAV9 capsid and promoter library designed for high cardiac tropism and low liver uptake. Its internal manufacturing capabilities support the rapid transition from discovery to clinical application. The company also employs a "human-first" approach, using patient-derived induced pluripotent stem cells (iPSCs) to model diseases and screen for therapeutic candidates.

Tenaya Therapeutics Pipeline and Products

Late-Stage Pipeline (Phase 1b/2+)

Tenaya's most advanced candidates are currently in Phase 1b/2 dose-escalation and expansion studies. These programs represent the leading edge of cardiac gene therapy, holding multiple regulatory designations to expedite development.

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Compound	Phase	MOA	Indications	Next Milestone	Notable Features	Competitive Edge
TN-201	Phase 1b/2	AAV9 delivering functional MYBPC3 gene	MYBPC3-associated HCM	H1 2026 Data Update	Fast Track, Orphan Drug, & Rare Pediatric Disease designations	First-in-class gene therapy for the most common cause of genetic HCM
TN-401	Phase 1b/2	AAV9 delivering functional PKP2 gene	PKP2-associated ARVC	H1 2026 Data Update	Orphan Drug and Fast Track designations	Targets the root genetic cause of high-risk arrhythmias

References

Tenaya Therapeutics +2

TN-201 and TN-401 utilize the AAV9 vector to deliver functional copies of genes that, when mutated, lead to structural heart defects and life-threatening arrhythmias. TN-201 specifically targets Hypertrophic Cardiomyopathy (HCM) caused by mutations in the myosin-binding protein C3 gene, while TN-401 addresses Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC) caused by plakophilin-2 mutations.

Early-Stage Pipeline Highlights

• TN-301: A highly selective small molecule HDAC6 inhibitor in Phase 1 development for Heart Failure with preserved Ejection Fraction (HFpEF). Preclinical data presented in March 2026 also highlighted its potential to correct muscle decline in Duchenne Muscular Dystrophy (DMD), demonstrating superiority over certain existing compounds in mouse models Taiwannews +2. Tenaya plans to initiate patient-specific proof-of-activity studies in 2026 Tenaya Therapeutics.
• DWORF Program: An AAV-based gene therapy designed to deliver the DWORF (Dwarf Open Reading Frame) gene to increase calcium uptake in the heart, currently in preclinical development for dilated cardiomyopathy (DCM).
• Cellular Reprogramming: A discovery-stage program focused on transforming resident cardiac fibroblasts into functional cardiomyocytes to repair heart tissue damaged by previous myocardial infarctions.

Tenaya Therapeutics Clinical Trials Landscape

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Tenaya Therapeutics serves as the lead sponsor for 4 active clinical trials, including both interventional drug studies and observational natural history studies. These programs aim to enroll approximately 445 participants across 67 clinical sites, with a primary geographic focus on the United States, United Kingdom, and Europe. The clinical portfolio spans a diverse age range, including pediatric populations for its gene therapy candidates and adults for its heart failure programs.

Key Clinical Trial Updates

• TN-201 (MyPEAK-1™): In January 2026, Tenaya announced the rapid resolution and lifting of a clinical hold, allowing enrollment to resume for the Phase 1b/2 study BioSpace. An interim data update for Cohort 2 and updated results for Cohort 1 are expected in the first half of 2026 Firstwordpharma +2.
• TN-401 (RIDGE-1™): The Data and Safety Monitoring Board (DSMB) endorsed the transition to expansion cohorts in January 2026 Quartr. The company expects to present one-year follow-up data for the low-dose cohort and initial high-dose safety data by mid-2026 Taiwannews +2.
• Regulatory Alignment: Tenaya anticipates reaching alignment with regulatory authorities in the second half of 2026 regarding pivotal trial designs for both TN-201 and TN-401, which will define the path toward potential Biologic License Application (BLA) filings Tenaya Therapeutics +2.

Business Insights and Analysis

Business Development

Tenaya strategically utilizes collaborations to expand its research reach and capitalize on its cardiac delivery expertise.

• Alnylam Pharmaceuticals: In March 2026, Tenaya entered a multi-target research collaboration and licensing agreement with Alnylam to identify and validate up to 15 novel genetic targets for cardiovascular disease. Under the terms, Tenaya received an upfront payment of $10 million and is eligible for over $1.1 billion in milestone payments Pharmacally +2.
• Manufacturing Infrastructure: The company maintains an internal modular manufacturing facility that allows for process development and clinical-scale production of AAV-based therapies, reducing reliance on third-party providers.

Financial Overview

Tenaya Therapeutics is listed on the NASDAQ under the ticker TNYA. As of March 2026, the company reported that its current cash position is expected to fund operations into the second half of 2027, providing sufficient runway to reach critical clinical milestones for its lead gene therapy assets Tenaya Therapeutics +2.

Clinical Trial Participation Guide

For individuals interested in participating in Tenaya Therapeutics' research, Trial Radar offers integrated tools to streamline the process. You can utilize the Participation Assistant to "Simplify" complex study details, "Check Eligibility" for specific trials like MyPEAK-1 or RIDGE-1, and view the "What to Expect" section for a preview of the clinical journey. To find a participating center nearby or to reach out to a study coordinator, refer to the Contacts & Locations tab within each trial listing on the platform.

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