Pipeline

Data from Solid’s IGNITE DMD Phase I/II clinical trial evaluating the first-generation candidate SGT-001, which used the AAV9 capsid, showed that expression of the nNOS microdystrophin transgene may have the potential to slow or stop the progression of Duchenne. In studies of SGT-003 performed in the mdx mouse model of Duchenne and in vitro in human Duchenne cell lines, multiple fold increases in microdystrophin expression have been observed in comparison to AAV9, suggesting that therapeutic dose levels using AAV-SLB101 can be lower than first generation candidates.

Microdystrophin is a synthetic form of the dystrophin gene that was designed because the natural, full-size dystrophin gene is too big to fit into an AAV vector. Microdystrophin retains the most critical components of full-size dystrophin yet fits into an AAV vector.

Researchers are currently studying several different microdystrophin genes for their potential to drive production of functional versions of dystrophin protein in muscles of the body.

SGT-212 is a recombinant AAV-based gene replacement therapy for Friedreich’s ataxia (FA) designed to deliver full-length human frataxin (Fxn) via a dual route of administration: intradentate nucleus (IDN) infusion, using an MRI-guided device, followed by an intravenous (IV) infusion to increase therapeutic Fxn levels in the cerebellar dentate nuclei and in the cardiomyocytes, respectively. Restoration of Fxn levels is expected to repair the underlying mitochondrial dysfunction in neurons and cardiomyocytes to address both neurologic and cardiac manifestations of the disease.

SGT-212 was developed by FA212 LLC, a company founded by parents of children living with FA, the University of Pennsylvania, and Solid Biosciences.

SGT-501 is in development for Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT). CASQ2 & RYR2 proteins regulate cardiac calcium (Ca2+), important for electrical conduction and cardiac contraction / relaxation.

SGT-501 is in development for Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT). CASQ2 & RYR2 proteins regulate cardiac calcium (Ca2+), important for electrical conduction and cardiac contraction / relaxation.

AVB-401 is a novel gene transfer product candidate being developed for the treatment of BAG3 mediated dilated cardiomyopathy (BAG3). BAG3 is a rare cardiac disease and is characterized by mutations in the BAG3 gene. Sufficient levels of functional BAG3 are required for healthy cardiac function.