Medera a clinical-stage biopharmaceutical company focused on targeting cardiovascular diseases by developing next-generation therapeutics, and its clinical development division Sardocor, together with the University of Kansas Medical Center, today announced the successful 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 (DMD-CM).
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First Patient Treated with SRD-001
The patient was successfully 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.
Gene Therapy for DMD Cardiomyopathy: SRD-001
“This milestone represents a pivotal advancement in the treatment of DMD-associated cardiomyopathy,” said Ronald Li, PhD, CEO and co-founder of Medera. “As survival rates improve due to respiratory interventions, cardiac complications have emerged as the leading cause of mortality in DMD patients. Our Sardocor division’s innovative, first-in-human gene therapy offers a promising solution to address this critical unmet medical need through our proprietary intracoronary infusion methodology that has been shown in other indications to safely and reliably deliver the therapeutic gene directly to affected heart tissue.”
MUSIC-DMD Phase 1b Trial
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 (AAV1) 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.
SRD-001 Clinical Trial – NCT06224660
Study Start (Actual): 2024-10-02
Primary Completion (Estimated): 2027-10
Study Completion (Estimated): 2030-10
Number of Participants (Estimated): 12 (Male)
Locations: USA
Ages Eligible for Study: 18 Years and older (Adult, Older Adult )
SRD-001 Phase 1b Participation Criteria
People must fulfill the inclusion requirements in order to be eligible. If a person has another ailment or is undergoing treatment that could prevent them from receiving gene therapy, they might not be able to participate. The complete set of inclusion and exclusion criteria can be found by clicking on this study link – NCT06224660 –.
How Can I Contact with Sardocor?
Name: Sardocor Corp.
Phone Number: +1-617-880-7616
Email: [email protected]
