Tenaya wins $8M grant to advance cardio gene therapy trials

Tenaya Therapeutics announced that the California Institute for Regenerative Medicine (CIRM), a state agency that funds regenerative medicine, stem cell and gene therapy research, has awarded the company an $8 million clinical grant.

Proceeds from the grant will help fund Tenaya’s ongoing phase 1b RIDGE-1 clinical trial of its AAV9-based gene therapy, TN-401, being developed for the treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC), also known as arrhythmogenic cardiomyopathy, caused by mutations in the plakophilin-2 (PKP2) gene.

PKP2 gene mutations result in insufficient levels of critical proteins needed to maintain the structural integrity and cell-to-cell signaling of heart muscle cells. The TN-401 gene replacement therapy is designed to address the underlying cause of disease by delivering a functional PKP2 gene into heart muscle cells using an adeno associated virus serotype 9 capsid.

The RIDGE-1 clinical trial will assess the safety, tolerability and preliminary clinical efficacy of a one-time intravenous infusion of TN-401. The open-label, dose-escalation study is currently enrolling symptomatic adults who have been diagnosed with PKP2-associated ARVC.

Tenaya stocks plummeted in December following the release of early trial data from another AAV9-based gene therapy, TN-201, being developed for the potential treatment of MYBPC3-associated hypertrophic cardiomyopathy. While preliminary data from three patients in the first dose cohort of 3E13 vg/kg showed that TN-201 was generally well tolerated, with readily detectable vector DNA in the heart, evidence of transgene RNA expression, and increasing TN-201 mRNA and MyBP-C protein levels over time, investors expressed concern over increased liver enzyme levels and a lower level of RNA expression than anticipated.