Medera Completes High-Dose Cohort Enrollment in Groundbreaking Cardiac AAV Gene Therapy Trial for HFpEF

BOSTON, MA —January 5, 2026— Medera Inc. announced today the successful dosing of the final patient in Cohort B of its MUSIC-HFpEF Phase 1/2a clinical trial. This milestone marks the completion of enrollment for the high-dose group (4.50×10¹³ viral genomes) evaluating SRD-002, a first-in-human AAV gene therapy designed to treat heart failure with preserved ejection fraction (HFpEF), a condition that currently lacks disease-modifying treatment options.

Targeting the “Powerhouse” of Cardiac Relaxation

HFpEF affects nearly half of the 64 million heart failure patients worldwide. Unlike other forms of heart failure, HFpEF is characterized by the heart’s inability to relax and fill properly due to myocardial stiffness and diastolic dysfunction.

SRD-002 utilizes an adeno-associated virus type 1 (AAV1) vector to deliver the SERCA2a gene directly to the heart. SERCA2a is a critical calcium pump that:

• Restores Calcium Handling: Improves the transport of calcium within cardiac cells, which is essential for healthy relaxation.

• Reduces Myocardial Stiffness: Directly addresses the biological drivers of diastolic dysfunction.

• Minimizes Systemic Exposure: Uses a proprietary minimally invasive intracoronary infusion, allowing for therapeutic efficacy at doses significantly lower than traditional systemic AAV delivery.

Clinical Feasibility and Early Success

The trial’s dosing strategy was optimized using Medera’s mini-Heart™ technology, a human-based disease modeling platform co-developed with AstraZeneca. This “human-first” approach contributed to the therapy receiving FDA Fast Track Designation.

Key findings from the trial to date include:

• Favorable Safety Profile: No gene therapy-related serious adverse events reported across low-dose (Cohort A) or high-dose (Cohort B) groups.

• Functional Improvements: Patients in the low-dose cohort showed improved NYHA heart failure classification and quality-of-life scores at 12 months.

• Hemodynamic Stabilization: Invasive measurements showed clinically meaningful improvements in pulmonary capillary wedge pressure (PCWP) during both rest and exercise.

The Path Toward Phase 2b

With Cohort B enrollment complete, the focus shifts to long-term monitoring. Patients will be followed for 24 months to evaluate the durability of the SERCA2a expression.

“Completion of enrollment in Cohort B underscores the clinical feasibility of targeted intracoronary cardiac gene therapy,” said Dr. Ronald Li, CEO and Founder of Medera. “The very encouraging results support our confidence in advancing this first-in-human approach toward potential disease-modification studies.”