Solid Biosciences (SLDB) Chardan's 8th Annual Genetic Medicines Conference summary

Company overview and recent milestones

• Focused on precision genetic medicine for neuromuscular and cardiac diseases, with lead programs in Duchenne muscular dystrophy and CPVT.

• Dosed first two patients in Duchenne trial in Q2 and Q3, with third patient dosing targeted by end of month.

• Expanded clinical trial sites in the US, Canada, UK, and Italy, with broad physician engagement.

• Manufacturing scaled to support expanded trials, with high full-to-empty capsid ratios for gene therapy quality.

Clinical trial design and strategy

• Initial trial focused on safety in boys aged 4–7, showing a benign safety profile and no serious adverse events.

• Study design expanded to include older boys in the declining phase, aiming for more meaningful clinical outcomes.

• Aggressive pivot to larger, longer trials (18 months) with endpoints like rise time and stride velocity, designed with input from key opinion leaders.

• Parallel dosing now possible, increasing enrollment speed and meeting high patient demand.

Data readout and endpoints

• Initial data on microdystrophin expression and biochemical markers expected end of year or early Q1, depending on patient count.

• Functional measures in early patients may be limited due to age, with efficacy focus shifting to older, plateauing/declining populations.

• Endpoints include safety, expression, rise time, and stride velocity, moving away from subjective measures.