BOSTON, April 16, 2026 (GLOBE NEWSWIRE) -- Medera Inc. ("Medera"), a clinical-stage biopharmaceutical company developing next-generation cardiovascular therapeutics, and its clinical development division Sardocor, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to AAV-SERCA2a, an investigational gene therapy drug designed to treat cardiomyopathy associated with Duchenne muscular dystrophy (DMD-CM), building on prior human clinical experience with SERCA2a gene therapy and Medera’s ongoing clinical programs. AAV-SERCA2a is currently being evaluated in the first-in-human MUSIC-DMD clinical trial and aims to restore cardiac calcium handling by increasing expression of SERCA2a, a key regulator of heart muscle contraction and relaxation, using Medera's targeted intracoronary gene delivery approach designed to achieve therapeutic cardiac exposure at substantially lower vector doses than conventional systemic gene therapy approaches.

Fast Track Designation is an FDA program designed to facilitate the development and expedite the review of therapies for serious or life-threatening conditions with unmet medical need, and may allow eligibility for Priority Review and Accelerated Approval pathways, subject to FDA agreement. The designation enables more frequent FDA interactions and the ability to submit a Biologics License Application on a rolling basis, with the purpose of bringing important new therapies to patients earlier. The designation reflects FDA recognition of both the serious unmet need in DMD-CM and the potential of Medera’s targeted gene therapy approach.

"Cardiac failure has become the leading cause of death in patients with Duchenne muscular dystrophy, yet therapies targeting the underlying cardiac biology remain extremely limited," said Ronald Li, PhD, CEO and Founder of Medera. “Our therapeutic approach is designed to address the fundamental calcium-handling defect driving cardiac deterioration in these patients, and the FDA's Fast Track Designation reinforces both the urgency of this unmet need of DMD-CM and the potential of our targeted intracoronary delivery, which may enable therapeutic efficacy at substantially lower vector doses than conventional systemic methods."

AAV-SERCA2a aims to restore calcium handling in DMD-CM patients by increasing expression of SERCA2a. Its delivery via Medera's proprietary minimally invasive intracoronary infusion methodology is designed to achieve therapeutic efficacy with approximately 100-fold lower viral vector doses compared to conventional systemic intravenous gene therapy approaches, supporting a potentially improved safety and tolerability profile and building on extensive prior clinical experience targeting SERCA2a in heart failure.

"Cardiac complications have become the leading cause of mortality in DMD patients, yet options targeting the underlying disease mechanism remain critically lacking,” said Pat Furlong, Founder of Parent Project Muscular Dystrophy. “The Fast Track Designation reflects the importance of advancing novel approaches for this underserved population, and we are encouraged by the progress of this first-in-human trial."

DMD affects over 300,000 people worldwide, including 40,000 patients in the U.S. and EU. Current treatment options are limited, with no approved disease-modifying therapies targeting the underlying pathophysiology of DMD-CM.

For more information on the MUSIC-DMD trial, please visit ClinicalTrials.gov: NCT06224660. The Company continues to advance the MUSIC-DMD study and expects to generate initial clinical data to inform future development and regulatory discussions.

About Duchenne Muscular Dystrophy-Associated Cardiomyopathy

DMD is a rare, life-threatening genetic disorder affecting skeletal and cardiac muscle in boys and young men. With improved respiratory care extending survival, cardiac complications have emerged as the leading cause of death in DMD patients. Nearly all patients develop cardiomyopathy by age 18, characterized by widespread fibrosis of the heart muscle leading to heart failure and potentially fatal arrhythmias. Current treatment options are limited and largely based on standard heart failure therapies with limited proven benefit in DMD patients.

About Medera Inc.

Medera is a clinical-stage biopharmaceutical company focused on targeting difficult-to-treat and currently incurable diseases by developing a range of next-generation therapeutics. Medera operates via its two preclinical and clinical business units, Novoheart and Sardocor, respectively.

Novoheart capitalizes on the world's first and award-winning "mini-Heart" Technology for revolutionary disease modelling and drug discovery, uniquely enabling the modelling of human-specific diseases and discovery of therapeutic candidates free from species-specific differences in accordance to the FDA Modernization Act 2.0. Novoheart's versatile technology platform provides a range of state-of-the-art automation hardware and software as well as screening services, for human-specific disease modelling, therapeutic target discovery and validation, drug toxicity and efficacy screening, and dosage optimization carried out in the context of healthy and/or diseased human heart chambers and tissues. Global pharmaceutical and academic leaders are using Novoheart's technology platform for their drug discovery and development purposes. The Novoheart platform has facilitated and accelerated the development of Sardocor's lead therapeutic candidates that are currently in clinical trials.

Sardocor is dedicated to the clinical development of novel next-generation therapies for Medera. Leveraging Novoheart's human-based drug discovery and validation platforms, Sardocor aims to expedite drug development and regulatory timelines for its gene and cell therapy pipeline. Sardocor has received Investigational New Drug (IND) clearances from the FDA for three ongoing AAV-based cardiac gene therapy clinical trials targeting Heart Failure with Reduced Ejection Fraction (HFrEF), Heart Failure with Preserved Ejection Fraction (HFpEF) with the Fast Track Designation, and Duchenne Muscular Dystrophy-associated Cardiomyopathy (DMD-CM) with the Orphan Drug Designation and Fast Track Designation. This designation further strengthens Medera’s broader AAV-SERCA2a platform, which is being clinically evaluated across multiple cardiac indications including HFrEF and HFpEF. Medera's cardiac gene therapy pipeline is built on its investigational AAV-SERCA2a platform, which currently includes three clinical programs: SRD-001 for HFrEF, SRD-002 for HFpEF, and SRD-003 for DMD-CM. Additionally, Sardocor's pipeline includes four preclinical gene therapy and three preclinical small molecule candidates targeting various cardiac, pulmonary, and vascular diseases.

For more information, please visit www.medera.bio. 

Contacts

Ally Stubin

Public Relations

ICR Healthcare

Ally.stubin@icrhealthcare.com

646.667.1861

Stephanie Carrington

Investor Relations

ICR Healthcare

Stephanie.carrington@icrhealthcare.com

646.277.1282