Medera, a clinical-stage biopharmaceutical company developing next-generation cardiovascular therapeutics, has announced that the final patient has been dosed in Cohort B of its ongoing MUSIC-HFpEF Phase 1/2a trial of SRD-002 gene therapy for heart failure with preserved ejection fraction (HFpEF).
The patient was treated successfully using Medera’s proprietary minimally invasive intracoronary infusion method and tolerated the procedure well. Completion of Cohort B enrollment underscores the clinical feasibility of targeted cardiac gene therapy for complex HFpEF, a condition with substantial unmet medical need.
Heart failure affects an estimated 64.3 million people worldwide, and HFpEF accounts for nearly half of all cases, yet therapeutic options remain limited. SRD-002 is a one-time gene therapy designed to directly address the molecular drivers of HFpEF—including impaired calcium handling, myocardial stiffness, and diastolic dysfunction—via an adeno-associated virus type 1 vector carrying the cardiac SERCA2a gene.
“The completion of enrollment in Cohort B represents an important milestone in our clinical development program,” said Ronald Li, CEO and Founder of Medera.
“With all five patients in Cohort B now dosed at the higher therapeutic dose, we continue to build a robust safety and efficacy dataset across both cohorts. The very encouraging results to date support our confidence in advancing this first-in-human gene therapy approach for patients with HFpEF, a condition affecting approximately half of all heart failure patients worldwide with limited disease-modifying therapeutic options.”
Cohort B patients received doses substantially higher than previous studies, while still far below levels typically required for systemic IV delivery, highlighting the efficiency of Medera’s cardiac-targeted approach.
The dosing strategy was optimized through Medera’s proprietary human-based mini-Heart platform, co-developed with AstraZeneca, which enabled rational dose selection and contributed to FDA Investigational New Drug (IND) clearance and Fast Track Designation.
The trial continues to show a favorable safety profile. No gene therapy-related serious adverse events have been reported across either cohort. Patients in Cohort A completed 12-month follow-up, demonstrating improvements in heart failure classification and quality-of-life measures, as well as stabilization of key hemodynamic parameters.
“Completing enrollment in Cohort B is a testament to the dedication of our clinical investigators and the commitment of patients participating in this groundbreaking trial,” said Marat Fudim, Advanced Heart Failure Specialist and Associate Professor at Duke University Medical Center.
“This milestone positions us well to gather the additional safety and efficacy data needed to support progression toward Phase 2b and potential disease-modification studies.”
