Solid Biosciences (SLDB) 12th Annual Cantor Fitzgerald Global Healthcare Conference summary

Strategic focus and pipeline overview

• Emphasis placed on gene therapies for Duchenne muscular dystrophy (DMD) and Friedreich's ataxia (FA), with other cardiac programs progressing more slowly due to resource prioritization.

• Proprietary platform develops novel delivery tools, including capsids and dual plasmids, shared with academic labs to advance next-generation gene therapies.

DMD program insights and trial progress

• INSPIRE trial for DMD dosed 53 patients, with high demand and rapid enrollment; only a few more patients will be dosed using commercial material for bridging data.

• Anticipates strong demand for phase III trials in both the US and Europe, expecting rapid commercial uptake if approved.

• Safety profile remains favorable, with no drug-induced liver injury, myocarditis, or TMAs, and minimal steroid use.

• Approximately 50% of ambulatory DMD patients remain eligible for therapy, estimated at 2,000–3,000 children.

Biomarker and clinical outcome strategy

• Utilizes a comprehensive set of 10–12 biomarkers to assess efficacy, including VGs per nucleus, Western blot, mass spec, muscle integrity markers, and chronic phase indicators.

• Declines in embryonic myosin heavy chain and improvements across multiple biomarkers suggest strengthening muscles and anticipated clinical benefit.