Solid Biosciences Awarded Innovation Passport Designation Under the New UK Innovative Licensing and Access Pathway for SGT-003, an Investigational Gene Therapy for Duchenne Muscular Dystrophy

Core Insights - Solid Biosciences has received an Innovation Passport for its investigational gene therapy SGT-003, aimed at treating Duchenne muscular dystrophy, under the new Innovative Licensing and Access Pathway (ILAP) in the UK [1][2][3] - The ILAP is designed to accelerate the development and access of transformative medicines, facilitating early engagement with regulatory bodies [4] - SGT-003 is currently undergoing clinical trials, including the Phase 1/2 INSPIRE DUCHENNE trial and the Phase 3 IMPACT DUCHENNE trial, with the aim of becoming the first gene therapy for Duchenne in the UK [3][8][9] Company Overview - Solid Biosciences is focused on developing precision genetic medicines for neuromuscular and cardiac diseases, with a portfolio that includes SGT-003 for Duchenne muscular dystrophy [10] - The company aims to improve the lives of patients with rare diseases through innovative gene therapy solutions [10] Clinical Development - SGT-003 is designed with a differentiated microdystrophin construct and a proprietary capsid, AAV-SLB101, which targets integrin receptors and enhances muscle transduction [7] - The ongoing INSPIRE DUCHENNE trial is evaluating the safety and efficacy of SGT-003 in pediatric participants with Duchenne, while the IMPACT DUCHENNE trial is focused on supporting regulatory authorizations outside the US [8][9] Regulatory Framework - The ILAP, launched in 2021, provides a unique end-to-end regulatory access pathway, allowing for early multi-stakeholder engagement and support for clinical development and market access [4] - The Innovation Passport designation enables Solid Biosciences to work closely with the UK's MHRA and other regulatory bodies to expedite the development of SGT-003 [1][3]