Solid Bio teams with Mayo Clinic to advance cardiac gene therapies

Solid Biosciences signed a deal with Mayo Clinic for worldwide licenses to an AAV-based suppression-replacement gene therapy platform and multiple next-generation cardiac gene therapy programs developed by Mayo’s Windland Smith Rice Sudden Death Genomics Lab.

Per the deal, Solid will gain an exclusive license to Mayo’s ‘Sup-Rep’ gene therapy platform and use it to develop and commercialize six cardiac gene therapy programs developed using the platform. The platform uses a short hairpin RNA (shRNA) to knockdown both wild-type and mutant endogenous alleles and then generates normal protein via an shRNA-immune (shiMM) cDNA. According to Mayo Clinic, it has the potential to offer novel way of treating patients at risk for sudden cardiac death from their genetic heart disease.

The six programs will use Solid’s next-generation AAV capsids, including AAV-SLB101, and advanced manufacturing capabilities to suppress and replace genes implicated in potentially life-threatening genetic heart diseases. Mayo Clinic will be responsible for R&D efforts for each cardiac gene therapy candidate up to IND-enabling studies, at which point, Solid will have the option to pursue continued development and commercialization of each licensed program.

Massachusetts-based Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates including SGT-003 for the treatment of Duchenne muscular dystrophy, SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia, SGT-601 for the treatment of TNNT2-mediated dilated cardiomyopathy, SGT-401 for the treatment of BAG3-mediated dilated cardiomyopathy, and additional assets for the treatment of fatal cardiac diseases.