Solid Biosciences (SLDB) H.C. Wainwright 26th Annual Global Investment Conference 2024 summary

Company overview and strategy

• Focuses on AAV-based gene transfer therapies for rare neuromuscular and cardiac genetic diseases, expanding from a single indication to multiple areas in recent years.

• Invests heavily in delivery and manufacturing platforms, emphasizing high full-to-empty capsid ratios for improved safety, efficacy, and cost.

• Pipeline includes programs for Duchenne muscular dystrophy, Friedreich's ataxia, and several cardiac indications.

SGT-003 program and clinical progress

• SGT-003 is a next-generation micro-dystrophin gene therapy for Duchenne muscular dystrophy, featuring improvements in manufacturing, capsid, and transgene.

• Early safety data from the first two patients showed no significant adverse events, supporting a favorable safety profile.

• The INSPIRE Duchenne study is expanding in age range, number of patients, sites, and countries due to high demand and positive early results.

• 90-day biopsy and functional data from initial patients expected by end of 2024 or early 2025, depending on cohort size.

Manufacturing and platform advancements

• Achieves 77%-81% full-to-empty ratio for Duchenne batches and over 90% for CPVT, reducing required dosing and improving safety and cost.

• Manufacturing scale is at 1,000 liters with strong yields and minimal changes expected for expansion trials.