Medera Advances Cardiac AAV Gene Therapy to Phase 2 for Heart Failure

BOSTON, MA — October 27, 2025 — Medera Inc., a clinical-stage biopharmaceutical company, today announced a significant clinical milestone, confirming that the first patient has been dosed in the Phase 2 portion of the MUSIC-HFrEF trial evaluating its gene therapy candidate, SRD-001, for patients with heart failure with reduced ejection fraction (HFrEF).

The initial patient dosing in the Phase 2 segment occurred on September 5, 2025, following a positive recommendation from the independent Data and Safety Monitoring Board (DSMB) in March 2025 to advance the trial based on a review of all nine patients in the preceding Phase 1b portion.

Targeting a Mass Market Incurable Disease

HFrEF is a highly prevalent form of heart disease, accounting for half of the estimated 64.3 million heart failure cases worldwide, and remains a chronic, progressive condition with high mortality rates. SRD-001 is being developed to address the urgent need for disease-modifying therapies that can reverse HFrEF pathophysiology.

• Trial Design: The Phase 2 portion is a randomized, double-blind, placebo-controlled study with a 1:1 randomization ratio. It aims to enroll a total of 50 patients to further evaluate the efficacy and safety of SRD-001.
• Administration: The therapy is administered using Medera’s proprietary minimally invasive intracoronary infusion methodology, a procedure well-tolerated by the first patient.

Brian Jaski, M.D., Principal Investigator of MUSIC-HFrEF, noted that the initiation of the placebo-controlled Phase 2 “marks an important step forward in evaluating the efficacy and safety of SRD-001 in a larger patient population with advanced heart failure.”

About the SRD-001 Gene Therapy

SRD-001 is an investigational gene therapy that utilizes an adeno-associated virus serotype 1 (AAV1) vector.

The vector delivers the transgene for sarco(endo)plasmic reticulum Ca2+ ATPase 2a isoform (SERCA2a), a protein essential for regulating calcium cycling and contractility in heart muscle cells. The therapy is being studied in subjects with ischemic or non-ischemic cardiomyopathy who have advanced heart failure symptoms (NYHA class III/IV).

Ronald Li, Ph.D., CEO and co-founder of Medera, stated, “The Phase 2 portion will further evaluate SRD-001 and advance our clinical program as we work toward developing next-generation therapeutics for HFrEF patients.”