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Medera, University of Kansas report first patient treated in gene therapy trial
Medera and its clinical development division Sardocor, together with the University of Kansas Medical Center announced the treatment of the first patient in the MUSIC-DMD Phase 1b clinical trial. The trial is investigating AAV1.SERCA2a, a one-time gene therapy treatment for cardiomyopathy secondary to Duchenne muscular dystrophy. The patient was treated via left radial artery access using Medera's proprietary minimally invasive intracoronary infusion methodology. The patient tolerated the procedure well and was discharged following an overnight hospital stay for observation. The MUSIC-DMD Phase 1b trial is an open-label, controlled study evaluating the safety and efficacy of AAV1.SERCA2a, an adeno-associated virus serotype 1 gene therapy that delivers the SERCA2a gene directly to heart muscle cells, in adult males with DMD-associated cardiomyopathy. The study will enroll up to 12 participants across low-dose, high-dose, and control groups. DMD-associated cardiomyopathy is characterized by calcium overload in heart muscle cells, leading to progressive fibrosis and heart failure. AAV1.SERCA2a aims to restore calcium handling in DMD-CM patients by increasing SERCA2a protein production, potentially reversing the underlying disease process.
Medera, Novoheart announce collaboration with scientists at University of CA
Medera and Novoheart announced a groundbreaking collaboration with physician-scientists at the University of California San Diego School of Medicine and Mayo Clinic. The goal of the partnership is to develop the first human mini-heart models of hypoplastic left heart syndrome using stem cells from real patients and improve the understanding of this disease with the goal of developing more personalized treatment strategies for children born with this devastating condition. On September 5, 2024, Medera and Keen Vision Acquisition Corporation, announced they had entered into a definitive merger agreement
Medera, Novoheart present breakthroughs in human mini-heart platforms, therapy
Medera and Novoheart, its wholly owned pre-clinical subsidiary pioneering human-based cardiac tissue engineering for disease modelling and drug screening, announced the successful presentation of seven scientific abstracts at the 2025 International Society for Stem Cell Research, ISSCR, Annual Meeting, held June 11-14 in Hong Kong. The annual ISSCR meeting brought together nearly 4,000 of the world's leading stem cell researchers and regenerative medicine experts. Showcased 7 presentations at ISSCR 2025, including a featured oral presentation on first-in-human gene therapy for heart failure, powered by their proprietary human mini-Heart technology. FDA-backed human-based screening platform recognized as an animal-free alternative in drug development, supporting IND and Fast Track approvals under the FDA Modernization Act 2.0. Breakthroughs in high-throughput screening, AI-driven drug classification, and patient-specific disease modelling underscored the platform's potential to de-risk development and accelerate clinical translation. On September 5, 2024, Medera and Keen Vision Acquisition Corporation announced they had entered into a definitive merger agreement.
Medera showcases interim data from first-in-human gene therapy trial
Medera announced that interim data from its ongoing MUSIC-HFpEF Phase 1/2a clinical trial was presented at the Heart Failure 2025 Congress taking place in Belgrade, Serbia. The late-breaking presentation, titled "Gene therapy for heart failure with preserved ejection fraction," was delivered by Marat Fudim, MD, MHS, Advanced Heart Failure Specialist and Associate Professor at Duke University Medical Center, during the "Late-Breaking Clinical Trials in Chronic Heart Failure" session. The MUSIC-HFpEF trial is investigating SRD-002, a one-time gene therapy treatment for heart failure with preserved ejection fraction delivered through a proprietary minimally invasive intracoronary infusion methodology. SRD-002 utilizes an adeno-associated type 1 virus vector carrying the cardiac isoform of the sarcoplasmic reticulum calcium ATPase pump to directly target the molecular pathways underlying the core pathology of HFpEF by enhancing myocardial relaxation and reducing stiffness. The trial is evaluating the therapy in HFpEF patients with exercise-induced diastolic dysfunction, including those with LVEF greater than or equal to 50% and symptomatic HFpEF defined by prior hospitalization or New York Heart Association Class II or III symptoms. The presentation highlighted interim data from the ongoing trial, which, as of the data cutoff date, has treated five patients in Cohort A with a low dose of 3x1013 viral genomes per patient and one patient in Cohort B at a dose of 4.5x1013 vg per patient. With follow-up ranging from 4 to 16 months, no gene therapy-related serious adverse events have been reported. Four out of five patients in the low-dose group have shown improvements in NYHA heart failure classification at 6 months, with clinically meaningful improvements in 6-minute walk test, decreases/stabilization in NT-Pro-BNP, and high-sensitivity troponin observed in some patients. The enrollment of patients at the higher dose of 4.5x1013 vg per patient is ongoing.
Medera presents updated results from gene therapy trial for heart failure
Medera announced that updated results from its ongoing MUSIC-HFpEF Phase 1/2a clinical trial investigating SRD-002, a one-time gene therapy treatment delivered through a proprietary minimally invasive intracoronary infusion methodology, were presented at the 2025 HFpEF Summit held March 19-21, 2025, in Beverly Hills, CA. Heart failure is a global pandemic with an estimated 64.3 million cases worldwide, costing over $100B per year. Heart failure with preserved ejection fraction accounts for nearly half of all heart failure cases, but has limited disease-modifying therapeutics. SRD-002 is a gene therapy utilizing an adeno-associated type 1 virus vector carrying the cardiac isoform of the sarcoplasmic reticulum calcium ATPase pump that directly targets the molecular pathways underlying the core pathology of HFpEF by enhancing myocardial relaxation and reducing stiffness. The presentation titled, "Targeting Diastolic Dysfunction in HFpEF- SERCA2a Gene Therapy," was delivered by Marat Fudim, MD, MHS, Advanced Heart Failure Specialist and Associate Professor at Duke University Medical Center and site principal investigator, on Friday, March 21st. The presentation highlighted data from the trial, which, as of the data cutoff date of February 25, 2025, has treated five patients in Cohort A with a low dose of 310 viral genomes per patient and one patient has been dosed in Cohort B at a dose of 4.510 vg per patient. With follow-up ranging from 2 to 15 months, no gene therapy-related serious adverse events have been reported. Four out of the five patients in the low-dose group have shown improvements in New York Heart Association heart failure classification at 6 months, with clinically meaningful improvements in 6-minute walk test, decreases/stabilization in NT-Pro-BNP, and high-sensitivity troponin observed in some patients. The enrollment of patients at the higher dose of 4.5x1013 vg per patient is ongoing. The HFpEF Summit is a biannual, two-day meeting bringing together internationally recognized leaders in the clinical, biological, and translational study of HFpEF. This summit focused on the latest insights and innovations for scientists, clinicians, researchers, and other healthcare professionals interested in all aspects of HFpEF. On September 5, 2024, Medera and Keen Vision Acquisition Corporation announced they had entered into a definitive merger agreement.
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