(Updates with stock price movement in the headline and first paragraph.)
Tenaya Therapeutics (TNYA) shares plunged nearly 51% in recent trading on Tuesday after initial data for this gene therapy study indicated a manageable safety profile with isolated elevations in liver enzymes of the first three dosed patients.
The company said Tuesday that preliminary data from the first cohort in a phase 1b/2 trial showed that its TN-201 gene therapy was well tolerated, with no cardiac toxicities or severe adverse effects.
TN-201 is being developed for the potential treatment of MYBPC3-associated hypertrophic cardiomyopathy, a condition caused by insufficient levels of myosin-binding protein C, the company said.
Tenaya said the initial results demonstrated "readily detectable vector DNA in the heart, evidence of transgene RNA expression, and increasing TN-201 mRNA and MyBP-C protein levels over time."
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