Tenaya Therapeutics (TNYA) said Tuesday that preliminary data from the first cohort of three patients 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."
Tenaya Therapeutics shares were down 12% in recent premarket trading.
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