Medera presents updated results from gene therapy trial for heart failure

Medera announced that updated results from its ongoing MUSIC-HFpEF Phase 1/2a clinical trial investigating SRD-002, a one-time gene therapy treatment delivered through a proprietary minimally invasive intracoronary infusion methodology, were presented at the 2025 HFpEF Summit held March 19-21, 2025, in Beverly Hills, CA. Heart failure is a global pandemic with an estimated 64.3 million cases worldwide, costing over $100B per year. Heart failure with preserved ejection fraction accounts for nearly half of all heart failure cases, but has limited disease-modifying therapeutics. SRD-002 is a gene therapy utilizing an adeno-associated type 1 virus vector carrying the cardiac isoform of the sarcoplasmic reticulum calcium ATPase pump that directly targets the molecular pathways underlying the core pathology of HFpEF by enhancing myocardial relaxation and reducing stiffness. The presentation titled, “Targeting Diastolic Dysfunction in HFpEF- SERCA2a Gene Therapy,” was delivered by Marat Fudim, MD, MHS, Advanced Heart Failure Specialist and Associate Professor at Duke University Medical Center and site principal investigator, on Friday, March 21st. The presentation highlighted data from the trial, which, as of the data cutoff date of February 25, 2025, has treated five patients in Cohort A with a low dose of 310 viral genomes per patient and one patient has been dosed in Cohort B at a dose of 4.510 vg per patient. With follow-up ranging from 2 to 15 months, no gene therapy-related serious adverse events have been reported. Four out of the five patients in the low-dose group have shown improvements in New York Heart Association heart failure classification at 6 months, with clinically meaningful improvements in 6-minute walk test, decreases/stabilization in NT-Pro-BNP, and high-sensitivity troponin observed in some patients. The enrollment of patients at the higher dose of 4.5×1013 vg per patient is ongoing. The HFpEF Summit is a biannual, two-day meeting bringing together internationally recognized leaders in the clinical, biological, and translational study of HFpEF. This summit focused on the latest insights and innovations for scientists, clinicians, researchers, and other healthcare professionals interested in all aspects of HFpEF. On September 5, 2024, Medera and Keen Vision Acquisition (KVAC) Corporation announced they had entered into a definitive merger agreement.