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Gene therapy TN-201 shows promising results in treating MYBPC3-associated hypertrophic cardiomyopathy.

Company Fundamentals
03 Jun 2026
GlobeNewsWire
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Bullish
pluang ai news

Tenaya Therapeutics announced interim data from its MyPEAK-1 Phase 1b/2 trial showing that TN-201 gene therapy led to consistent cardiac remodeling and symptom improvements in patients with MYBPC3-associated hypertrophic cardiomyopathy (HCM). Patients in both low and high dose cohorts showed reductions in heart muscle thickening, improved heart function, and better exercise capacity, with benefits sustained up to two years in some cases. TN-201 has received PRIME designation from the EMA and is part of the FDA's Rare Disease Evidence Principles process, supporting its potential as a treatment for this severe genetic heart condition. Tenaya plans to share more long-term data later in 2026 and is working with regulators on pivotal study designs.

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