Solid Biosciences Granted Fast Track Designation for Duchenne Gene Therapy Candidate

Solid Biosciences (Nasdaq: SLDB) has received FDA Fast Track Designation for SGT-003, the company’s next-generation Duchenne muscular dystrophy (DMD) gene therapy candidate. The company will initiate a first-in-human Phase 1/2 clinical trial following FDA clearance of its Investigational New Drug (IND) application in November 2023. The company’s share price rose 50% in morning trading following the news.

Bo Cumbo, President and CEO at Solid Biosciences, said, “Receipt of FDA Fast Track Designation underscores the importance of rapidly developing SGT-003 to potentially aid the unmet needs of the Duchenne community. Having received IND clearance for SGT-003 last month, we are pleased to be expediting the development of a potentially life-changing therapy and look forward to continuing to work closely with the FDA.”

Highlights

The company will initiate an open-label, multicenter Phase 1/2 trial to determine the safety and tolerability of SGT-003 in pediatric patients with DMD. Long-term safety and efficacy will be evaluated for a total of 5 years following treatment.

DMD is a genetic muscle-wasting disease predominantly affecting boys, with symptoms usually appearing between three and five years of age.

The FDA’s Fast Track program facilitates the expedited development and review of new drugs that are intended to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs.

About Solid Biosciences 

Solid Biosciences is a life sciences company focused on advancing a portfolio of gene therapy candidates and neuromuscular and cardiac programs, including SGT-003, for the treatment of Duchenne muscular dystrophy (Duchenne), SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), AVB-401 for the treatment of BAG3-mediated dilated cardiomyopathy, AVB-202-TT for the treatment of Friedreich’s ataxia, and additional assets for the treatment of fatal cardiac diseases. Solid is advancing its diverse pipeline across rare neuromuscular and cardiac diseases, bringing together experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted, Solid’s mandate is to improve the daily lives of patients living with these devastating diseases. For more information, please visit www.solidbio.com.