Clinical Trial SuccessWeek 8 biopsies from the low-dose cohort demonstrated robust cardiac transduction and modest PKP2 protein gains in 2 out of 3 patients, which are encouraging given their correlation with clinical benefit.
FDA Lifting Of HoldEarly data provide positive validation of TN-401 MoA, coupled with FDA lift of TN-201 logistical clinical hold provides positive readthrough to TN-401, providing important de-risking event.
Safety ProfileTN-401 was well tolerated, with only mild, transient laboratory abnormalities and no dose-limiting toxicities, no thrombotic microangiopathy or cardiotoxicity, and no TN-401–related ICD shocks or arrhythmias.