Gene Therapy, Devices, Biologics, Small Molecules all for DMD

Solid Biosciences is dedicated to advancing genetic medicines specifically targeting neuromuscular and cardiac diseases. The company has developed a diverse pipeline of gene therapy candidates aimed at addressing high unmet medical needs in these areas. Notable programs include SGT-003 for Duchenne muscular dystrophy, SGT-501 for catecholaminergic polymorphic ventricular tachycardia (CPVT), and AVB-401 for BAG3-mediated dilated cardiomyopathy. These therapies leverage innovative approaches, such as the use of microdystrophin—a synthetic form of the dystrophin gene designed to fit into adeno-associated virus (AAV) vectors—allowing for effective delivery and expression in muscle tissues. 

In addition to these specific therapies, Solid Biosciences is exploring various gene transfer products, such as AVB-202-TT for Friedreich’s ataxia, which aims to replace the frataxin gene to mitigate the disease's progression. The company employs a dual route of administration for AVB-202-TT, enhancing its ability to target affected tissues. Solid's research emphasizes a strong mechanistic rationale and aims to bring groundbreaking genetic therapies to patients suffering from rare diseases, thereby improving their quality of life.

United States

Solid Biosciences (Solid) is a life science company singularly focused on solving Duchenne muscular dystrophy (Duchenne). We aim to be the center of excellence across the entire disease spectrum, serving as a catalyst to bring together those with the expertise in science, technology, disease management and care. Disease-focused and founded by a family directly impacted, our mandate is simple yet comprehensive - attack the roots of the disease and improve daily life for patients.

Cardiovascular Diseases

Central Nervous System (CNS)

Genetic Diseases

Musculoskeletal Diseases

Solid Biosciences

Duchenne muscular dystrophy

Genetic disorder

SGT-003

Friedreich’s Ataxia

SGT-212 (dual route)

Catecholaminergic Polymorphic Ventricular Tachycardia

Gene & Cell therapies

Heart disease

Cardiac gene therapy candidate for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT)

SGT-501 (Cardiac Gene Therapy)

Muscle degeneration

Muscle weakness

Solid’s SGT-001 is a novel adeno-associated viral (AAV) vector-mediated gene transfer therapy designed to address the underlying genetic cause of Duchenne muscular dystrophy (Duchenne). Duchenne is caused by mutations in the dystrophin gene that result in the absence or near absence of dystrophin protein. SGT-001 is a systemically administered candidate that delivers a synthetic dystrophin gene, called microdystrophin, to the body. This microdystrophin encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins, including neuronal nitric oxide synthase (nNOS). Data from Solid’s preclinical program suggests that SGT-001 has the potential to slow or stop the progression of Duchenne, regardless of genetic mutation or disease stage.
 
SGT-001 is based on pioneering research in dystrophin biology by Dr. Jeffrey Chamberlain of the University of Washington and Dr. Dongsheng Duan of the University of Missouri. SGT-001 has been granted Rare Pediatric Disease Designation, or RPDD, in the United States and Orphan Drug Designations in both the United States and European Union.

Solid Biosciences

Company Details