Medera Inc., a clinical-stage biopharmaceutical company, and its wholly owned subsidiary Novoheart showcased significant advances in human cardiac tissue engineering at the 2025 International Society for Stem Cell Research (ISSCR) Annual Meeting held June 11-14 in Hong Kong. The companies presented seven scientific abstracts demonstrating how their proprietary human mini-Heart technology has accelerated clinical translation of novel gene therapies for heart failure.
The presentations highlighted the FDA's growing acceptance of human-based models as alternatives to animal testing, with Medera's platform receiving regulatory recognition under the FDA Modernization Act 2.0. "Regulatory momentum is clearly building toward human-specific models in place of traditional animal testing," said Ronald Li, PhD, CEO and co-founder of Medera and Novoheart. "At ISSCR 2025, we demonstrated that engineered human mini-Hearts not only predict human outcomes better, as well as directly support clinical and regulatory decisions—including trial design and therapeutic validation."
Clinical Translation Success
The featured oral presentation showcased how data from Novoheart's human mini-Heart platform informed both FDA Investigational New Drug (IND) approval and Fast Track Designation for the MUSIC-HFpEF gene therapy trial (NCT06061549). This trial investigates SRD-002, a one-time gene therapy delivered through proprietary minimally invasive intracoronary infusion methodology for patients with heart failure with preserved ejection fraction.
The platform has also supported the ongoing MUSIC-HFrEF trial (NCT04703842) evaluating SRD-001 gene therapy for patients with heart failure with reduced ejection fraction. Disease-specific mini-Heart models enabled rational dose selection and provided mechanistic insights into SERCA2a-targeted AAV gene therapy approaches.
Technological Breakthroughs
Several presentations highlighted significant technological advances in the platform's capabilities. Automated high-throughput screening technology demonstrated the ability to reduce cell use and screening time by over 90% through robotically controlled functional testing of stem cell-derived micro-tissues in 96-well plate formats.
Novel ensemble algorithms trained on contractility and electrophysiology data from human cardiac tissues were shown to outperform conventional approaches for drug classification. The researchers also demonstrated how matrix optimization using collagen versus fibrin yields more physiologically relevant tissue phenotypes for improved drug screening and disease modeling.
Addressing Unmet Medical Needs
The work addresses critical gaps in heart failure treatment, particularly for HFpEF, which accounts for 50% or more of the overall heart failure population. Heart failure affects an estimated 64.3 million cases worldwide with rising prevalence trends. Despite the growing epidemic, HFpEF-focused interventional trials have had limited success, with only sacubitril-valsartan (Entresto) and empagliflozin (Jardiance) showing benefits for reducing cardiovascular mortality and heart failure hospitalization. However, these agents are not disease-modifying, highlighting the critical need for therapeutic interventions targeting physiological mechanisms involved in HFpEF.
Personalized Medicine Applications
The platform's versatility extends to rare genetic cardiomyopathies, with presentations demonstrating patient-derived iPSC-cardiomyocyte models for understanding and treating conditions such as Duchenne Muscular Dystrophy-associated cardiomyopathy. This personalized medicine approach enables transcriptomic analysis and bioengineered tissue modeling specific to individual patient genetics.
Regulatory Recognition and Future Impact
Dr. Kevin Costa, co-founder of Novoheart, emphasized the platform's alignment with evolving regulatory guidance: "By creating more physiologically relevant human cardiac tissues, we significantly reduce the time and cost of bringing new therapies to patients while aligning with evolving FDA guidance on alternatives to animal testing."
The FDA's recognition of these in vitro human cardiac models reflects a broader regulatory embrace of non-animal technologies to advance safer and more targeted therapies. Sardocor, Medera's clinical development unit, has received IND clearances for three ongoing AAV-based cardiac gene therapy clinical trials, including Fast Track Designation for HFpEF and Orphan Drug Designation for DMD-associated cardiomyopathy.
The company's pipeline includes four preclinical gene therapy candidates and three preclinical small molecule candidates targeting various cardiac, pulmonary, and vascular diseases. Medera is currently pursuing a merger with Keen Vision Acquisition Corporation (NASDAQ: KVAC), announced in September 2024.
