Tenaya Therapeutics received an $8 million grant from the California Institute for Regenerative Medicine (CIRM) to fund its Phase 1b RIDGE-1 clinical trial of TN-401 gene therapy. TN-401 is designed to treat arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by mutations in the •PKP2• gene, a condition affecting an estimated 70,000 people in the U.S. The therapy delivers a functional •PKP2• gene into heart muscle cells using an adeno-associated virus serotype 9 (AAV9) capsid.
This funding is crucial because it supports the development of a potentially curative therapy for a serious, progressive heart disease that currently lacks effective treatment options. Existing treatments for •PKP2•-associated ARVC only manage symptoms, while TN-401 aims to address the underlying genetic cause. This offers a significant advancement in the field of cardiac gene therapy and provides hope for patients with this debilitating condition. The successful development of a gene therapy like TN-401 could dramatically improve long-term outcomes for ARVC patients, potentially preventing disease progression and reducing the risk of life-threatening complications.
The RIDGE-1 trial is an open-label, dose-escalation study assessing the safety, tolerability, and preliminary efficacy of a one-time intravenous infusion of TN-401 in symptomatic adults with •PKP2•-associated ARVC. Initial data from the low-dose cohort is anticipated in the second half of 2025. Preclinical studies have demonstrated TN-401’s ability to restore PKP2 protein levels, normalize heart rhythms, and improve cardiac function. The $8 million grant will cover a portion of the clinical trial costs, allowing Tenaya to continue advancing this promising therapeutic candidate.
The CIRM grant, along with the promising preclinical data, signifies a positive step forward in the fight against ARVC. The upcoming release of clinical trial data will be a critical inflection point, providing key insights into TN-401’s potential to transform the treatment landscape for this devastating disease. Positive results could lead to further investment, accelerated development, and ultimately, a much-needed therapeutic option for patients with •PKP2•-associated ARVC.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
