This section highlights FDA-related milestones and regulatory updates for drugs developed by Tenaya Therapeutics (TNYA).
Over the past two years, Tenaya Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
TN-201 and TN-401. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
TN-201 - FDA Regulatory Timeline and Events
TN-201 is a drug developed by Tenaya Therapeutics for the following indication: Fully functional MYBPC3 gene.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- TN-201
- Announced Date:
- March 31, 2025
- Indication:
- Fully functional MYBPC3 gene
Announcement
Tenaya Therapeutics, Inc. announced that interim data from the first three patients enrolled in the company's MyPEAK-1 Phase 1b/2 clinical trial of TN-201 were highlighted in a Late-Breaker presentation at the 2025 American College of Cardiology Scientific Sessions (ACC.25).
AI Summary
In March 2025, Tenaya Therapeutics, Inc. announced encouraging interim data from the first three patients treated with TN-201 in its MyPEAK-1 Phase 1b/2 clinical trial. The data was featured in a Late-Breaker presentation at the 2025 American College of Cardiology Scientific Sessions (ACC.25). TN-201 is a gene therapy developed to treat MYBPC3-associated hypertrophic cardiomyopathy by delivering a functional MYBPC3 gene to heart cells. The initial results showed that the treatment was generally well tolerated at the 3E13 vg/kg dose, with most adverse events being mild, manageable, or reversible. Serial biopsy data demonstrated robust transduction and increasing RNA levels over time, indicating positive therapeutic effects. The early safety profile and encouraging biological responses provide optimism that TN-201 could impact the treatment landscape for patients with this aggressive form of heart disease.
Read Announcement- Drug:
- TN-201
- Announced Date:
- March 24, 2025
- Indication:
- Fully functional MYBPC3 gene
Announcement
Tenaya Therapeutics, Inc announced the publications of positive preclinical data for TN-201, the company's gene therapy candidate for Myosin-Binding Protein C3 (MYBPC3)-associated hypertrophic cardiomyopathy (HCM), in Nature Communications.
AI Summary
Tenaya Therapeutics recently published positive preclinical data for TN-201, its gene therapy candidate for treating MYBPC3-associated hypertrophic cardiomyopathy (HCM). The study, appearing in Nature Communications, shows that TN-201 delivers a working copy of the MYBPC3 gene to heart muscle cells using an AAV9 vector through a single intravenous infusion. This approach increases the levels of MyBP-C protein, which is often insufficient in patients with this genetic heart condition.
The data demonstrated that even at low dosages, TN-201 produces dose-dependent increases in MyBP-C protein and improves key measures of cardiac function, including the reversal of left ventricular hypertrophy. These promising results support further clinical development of TN-201 and offer hope for a new treatment that targets the underlying cause of this severe heart disorder.
Read Announcement- Drug:
- TN-201
- Announced Date:
- March 19, 2025
- Indication:
- Fully functional MYBPC3 gene
Announcement
Tenaya Therapeutics, Inc announced that new clinical and disease burden data pertaining to the company's MYBPC3-associated hypertrophic cardiomyopathy (HCM) program will be presented at the upcoming American College of Cardiology's Annual Scientific Session being held March 29-31, 2025 in Chicago, IL.
AI Summary
Tenaya Therapeutics, Inc. announced that it will present new clinical and disease burden data for its MYBPC3-associated hypertrophic cardiomyopathy (HCM) program at the American College of Cardiology’s Annual Scientific Session in Chicago, IL, from March 29 to 31, 2025. The presentation will focus on results from the MyPEAK-1 Phase 1b/2 clinical trial, which is studying TN-201—an AAV9-based gene therapy aimed at addressing HCM caused by MYBPC3 mutations.
During the late-breaking session on March 31, Dr. Milind Desai from the Cleveland Clinic will share early safety and efficacy findings from the study. Additionally, a poster presentation on March 30 will outline disease burden and patient characteristics using data from the SHaRe registry. These presentations are expected to provide valuable insights into the potential of TN-201 as a new treatment option for patients with MYBPC3-associated HCM.
Read Announcement- Drug:
- TN-201
- Announced Date:
- January 13, 2025
- Indication:
- Fully functional MYBPC3 gene
Announcement
Tenaya Therapeutics, Inc today provided an update on its clinical development programs and outlined its strategic priorities for 2025.
AI Summary
Tenaya Therapeutics, Inc. provided an update on its clinical development programs, outlining strategic priorities for 2025. The company is focused on advancing its gene therapies for heart disease, particularly TN-201 and TN-401. TN-201 targets MYBPC3-associated hypertrophic cardiomyopathy, and Tenaya anticipates reporting additional safety and biopsy data from its ongoing MyPEAK-1 Phase 1b/2 trial later in 2025. Meanwhile, TN-401 is being developed for PKP2-associated arrhythmogenic right ventricular cardiomyopathy, with early clinical data from the RIDGE-1 Phase 1b trial expected in the second half of the year. The update reflects Tenaya’s commitment to accelerating patient enrollment and providing clinical endpoint data, all while strengthening their development programs and furthering their mission to deliver potentially curative therapies for heart disease.
Read Announcement- Drug:
- TN-201
- Announced Date:
- December 16, 2024
- Estimated Event Date Range:
- December 17, 2024 - December 17, 2024
- Target Action Date:
- December 17, 2024
- Indication:
- Fully functional MYBPC3 gene
Announcement
Tenaya Therapeutics , will announce initial Cohort 1 data from the MyPEAK-1 Phase 1b/2 clinical trial of TN-201 gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM) on Tuesday, December 17.
AI Summary
Tenaya Therapeutics, Inc. will reveal its initial Cohort 1 findings from the MyPEAK-1 Phase 1b/2 clinical trial. This study focuses on TN-201, a gene therapy that targets MYBPC3-associated hypertrophic cardiomyopathy (HCM), a form of heart disease. The company aims to develop treatments that work at the root cause of heart-related illnesses. The data release is scheduled for Tuesday, December 17, 2024, during a live webcast and conference call at 8:00 a.m. ET. Participants can view the webcast by registering on the company's Investor Relations page.
The announcement is an important step for Tenaya Therapeutics as they continue to advance their pipeline of innovative therapies to treat both rare and common cardiac conditions. The company’s approach includes cutting-edge genetic medicine strategies to help address underlying issues that lead to heart disease.
Read Announcement
TN-401 - FDA Regulatory Timeline and Events
TN-401 is a drug developed by Tenaya Therapeutics for the following indication: For the treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by Plakophilin-2 (PKP2) gene mutations.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- TN-401
- Announced Date:
- April 24, 2025
- Indication:
- For the treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by Plakophilin-2 (PKP2) gene mutations.
Announcement
Tenaya Therapeutics, Inc. announced interim data from its ongoing RIDGE (NCT06311708) natural history and seroprevalence study of adults with arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by mutations in the Plakophilin 2 (PKP2) gene will be presented at the Heart Rhythm Society's annual Heart Rhythm meeting taking place in San Diego, CA from April 24-27, 2025.
AI Summary
Tenaya Therapeutics, Inc. announced interim data from its ongoing RIDGE (NCT06311708) study, which focuses on adults with arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by PKP2 gene mutations. This largest non-interventional natural history and seroprevalence study has enrolled over 175 participants, offering important insights into the disease’s progression and current treatment challenges. Initial findings highlight a high burden of arrhythmias among patients, even with standard care approaches, and reveal that a large majority of patients may be eligible for the investigational TN-401 gene therapy due to low rates of preexisting AAV9 antibodies. The interim data, shedding light on potential gene therapy benefits for this genetic heart disorder, will be presented at the Heart Rhythm Society’s annual Heart Rhythm meeting in San Diego, CA, from April 24-27, 2025.
Read Announcement- Drug:
- TN-401
- Announced Date:
- January 13, 2025
- Indication:
- For the treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by Plakophilin-2 (PKP2) gene mutations.
Announcement
Tenaya Therapeutics, Inc today provided an update on its clinical development programs and outlined its strategic priorities for 2025.
AI Summary
Tenaya Therapeutics, Inc. provided an update on its clinical development programs and strategic priorities for 2025. The company is focused on accelerating its gene therapy programs for cardiomyopathies with TN-201 and TN-401. For TN-201, a gene therapy targeting MYBPC3-associated hypertrophic cardiomyopathy, the company has initiated dosing in Cohort 2 of the MyPEAK-1 Phase 1b/2 clinical trial and plans to report additional data from Cohort 1 in the first half of 2025. Patient enrollment in both cohorts is ongoing with detailed safety assessments, biopsy data, and clinical endpoints expected later in the year. Meanwhile, TN-401, aimed at treating PKP2-associated arrhythmogenic right ventricular cardiomyopathy, is in the RIDGE-1 Phase 1b trial with initial clinical data anticipated in the second half of 2025. Tenaya plans to build on its recent progress to further evaluate and advance these gene therapy candidates.
Read Announcement- Drug:
- TN-401
- Announced Date:
- November 25, 2024
- Indication:
- For the treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by Plakophilin-2 (PKP2) gene mutations.
Announcement
Tenaya Therapeutics, Inc. announced that the first patient has been dosed with TN-401 gene therapy in the RIDGE-1 Phase 1b clinical trial at the University of California, San Francisco.
AI Summary
Tenaya Therapeutics recently reached a major milestone by dosing the first patient in its RIDGE-1 Phase 1b clinical trial at the University of California, San Francisco. This early-stage study is testing TN-401, an AAV9-based gene therapy designed to treat arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by mutations in the PKP2 gene.
TN-401 works by delivering a fully functional copy of the PKP2 gene to heart muscle cells, aiming to restore protein levels that are deficient in patients with ARVC. The therapy is expected to repair cell-to-cell connections in the heart, potentially reducing abnormal heart rhythms and disease progression. The trial, which involves a one-time intravenous dose, will help determine the safety, tolerability, and initial clinical effectiveness of TN-401, with early data anticipated in 2025.
Read Announcement
