Solid Biosciences Inc.

Solid Biosciences' SGT-003 Gene Therapy Shows Promise in Duchenne Muscular Dystrophy Trial

4 months ago

• Solid Biosciences reported positive initial data from its Phase 1/2 INSPIRE DUCHENNE trial of SGT-003, a gene therapy candidate for Duchenne muscular dystrophy (DMD). • The trial showed an average microdystrophin expression of 110% in the first three patients, along with improvements in biomarkers indicating muscle health and resilience. • SGT-003 was well-tolerated in the first six participants, with adverse events typical of AAV gene therapy and no serious safety concerns reported. • Solid Biosciences plans to meet with the FDA to discuss a potential accelerated approval pathway for SGT-003 based on these encouraging early results.

Solid Biosciences' SGT-212 Receives FDA IND Clearance for Friedreich's Ataxia Gene Therapy Trial

4 months ago

• The FDA has cleared Solid Biosciences' IND for SGT-212, a gene therapy targeting both neurological and cardiac manifestations of Friedreich's ataxia. • SGT-212 utilizes a dual route of administration, delivering full-length frataxin to the cerebellum via IDN infusion and to the heart via IV infusion. • A Phase 1b trial is planned for the second half of 2025, assessing safety and tolerability in ambulatory and non-ambulatory adults with FA over five years. • SGT-212 aims to address the underlying mitochondrial dysfunction in neurons and cardiomyocytes by restoring frataxin levels.

Solid Biosciences' SGT-212 Receives FDA Fast Track Designation for Friedreich's Ataxia Treatment

5 months ago

• Solid Biosciences' SGT-212, a dual-route gene therapy for Friedreich's ataxia (FA), has received Fast Track designation from the FDA, expediting its development and review process. • SGT-212 delivers the frataxin gene via intravenous and direct intradentate nucleus infusions, targeting both neurological and cardiac manifestations of FA. • A Phase 1b clinical trial is planned for the second half of 2025 to assess the safety and tolerability of SGT-212 in adult FA patients. • The Fast Track designation will allow Solid Biosciences to have more frequent interactions with the FDA and the potential to be eligible for priority review.

Solid Biosciences' SGT-003 Receives FDA Fast Track Designation for Duchenne Muscular Dystrophy

a year ago

• Solid Biosciences' SGT-003, a next-generation gene therapy, has been granted Fast Track designation by the FDA for treating Duchenne muscular dystrophy (DMD). • The Fast Track designation aims to accelerate the development and review of SGT-003, potentially offering a substantial improvement over existing DMD therapies. • SGT-003 leverages Solid Biosciences' proprietary AAV-SLB101 capsid, showing increased biodistribution to cardiac and skeletal muscle in preclinical studies. • A Phase I/II clinical study is planned to assess the safety and tolerability of SGT-003 in pediatric DMD patients, with long-term efficacy evaluated over five years.