Solid Biosciences and Mayo Clinic Partner to Advance Cardiac Gene Therapy Innovations

Solid Biosciences Inc., a life sciences company specializing in genetic medicines for neuromuscular and cardiac diseases, announced a collaboration with the Mayo Clinic. This partnership aims to advance a novel AAV gene therapy platform designed to address sudden cardiac death-predisposing genetic cardiomyopathies and channelopathies, which affect large patient populations and currently have high unmet medical needs.

According to the terms of the agreement, Solid Biosciences will receive an exclusive license from Mayo Clinic’s Windland Smith Rice Sudden Death Genomics Laboratory. This license will cover the Suppression and Replacement (Sup-Rep) gene therapy platform, which Solid will use to develop and commercialize six cardiac gene therapy programs. The therapies will incorporate Solid's next-generation AAV capsids, such as the AAV-SLB101, and their advanced manufacturing capabilities to target and replace defective genes responsible for serious genetic heart diseases. Mayo Clinic will handle the research and development up to the IND-enabling study phase. At this point, Solid will decide whether to continue with the development and commercialization of each therapy program.

Dr. Michael J. Ackerman, a Genetic Cardiologist and Director of Mayo Clinic’s Windland Smith Rice Sudden Death Genomics Lab, expressed optimism about the collaboration. He emphasized the potential of the Sup-Rep gene therapy platform to offer innovative treatments for patients at risk of sudden cardiac death due to genetic heart conditions. Dr. Ackerman is eager to develop new solutions that could significantly improve patients' lives despite their diagnoses.

Bo Cumbo, President and CEO of Solid Biosciences, highlighted the strategic importance of this collaboration in positioning Solid as a leader in cardiac precision genetic medicine. Cumbo outlined the company's plans, starting with the CPVT IND submission expected in the first half of 2025. The subsequent development of the Sup-Rep AAV platform, alongside additional therapies targeting significant conditions, aims to cement Solid's leadership in this field. Cumbo looks forward to working closely with Mayo Clinic and Dr. Ackerman in transforming care for cardiac patients.

The Mayo Clinic has a financial interest in Solid Biosciences and the technology mentioned in the announcement. They plan to use any revenue generated to support their mission in patient care, education, and research.

Solid Biosciences focuses on innovative genetic medicine, working on a range of gene therapy candidates. These include treatments for Duchenne muscular dystrophy, catecholaminergic polymorphic ventricular tachycardia (CPVT), TNNT2-mediated dilated cardiomyopathy, and BAG3-mediated dilated cardiomyopathy. The company's diverse pipeline aims to address rare neuromuscular and cardiac diseases by leveraging expertise in science, technology, disease management, and patient care. Founded by individuals directly impacted by these conditions, Solid Biosciences is dedicated to enhancing the lives of those living with these severe diseases.