Tenaya Therapeutics

The FDA has granted RMAT designation to Tenaya Therapeutics' AAV9-based gene therapy TN-401 for PKP2-associated arrhythmogenic right ventricular cardiomyopathy. The decision is based on interim RIDGE-1 Phase 1b/2 data showing lower rates of premature ventricular contractions and non-sustained ventricular tachycardias. TN-401 already had Fast Track and orphan drug status; the RMAT designation enables expanded interactions with the FDA and may open accelerated approval pathways. Tenaya plans to present additional RIDGE-1 data and an update on regulatory plans for a pivotal trial in Q4 2026. » Más en de.investing.com

At an investor conference, Tenaya reported positive early efficacy and safety data for its three clinical programs: TN-201 showed sustained improvements in six evaluable patients up to two years after a single dose, TN-401 reduced premature ventricular contractions by a median of 64%, and TN-301 is being readied for Phase II. Management is working on dose selection, regulatory alignment and development plans and expects clarity by the end of 2024. Tenaya reports cash runway through the third quarter of 2027, stressing that this provides time for several clinical and regulatory milestones before additional financing would be needed. » Más en de.investing.com

At the Canaccord Growth Conference, Tenaya reported positive early safety and efficacy signals for gene therapies TN-201 and TN-401 and the reactivation of the small‑molecule program TN-301. Regulatory decisions on the design of approval‑relevant studies for TN-201 and TN-401, as well as details on TN-301, are expected in the fourth quarter and will determine further capital allocation. The company noted cash of roughly $78 million, expected to last into the third quarter of 2027, but stressed that additional financing will be required for the planned registration and Phase II studies. » Más en de.investing.com