Solid Biosciences (SLDB) Goldman Sachs 47th Annual Global Healthcare Conference 2026 summary

Pipeline overview and clinical programs

• Robust pipeline includes advanced programs for Duchenne muscular dystrophy (DMD) and Friedreich's ataxia (FA), with a deep cardiac gene therapy portfolio.

• Two clinical-stage programs: Duchenne 003 in phase III and INSPIRE phase I/II with 51 patients dosed.

• First-in-class dual route administration for FA targets both cardiac and CNS manifestations.

• Over 50 academic and small company agreements for capsid platform development.

• Multiple new capsids expected for licensing by year-end, currently in non-human primate studies.

Duchenne program: scientific and clinical insights

• Next-generation microdystrophin transgene includes unique R16/R17 repeats for nNOS binding, enhancing blood flow and reducing inflammation.

• POLARIS-101 capsid shows superior muscle and cardiac tissue targeting, with up to 20x better heart expression than AAV9.

• Rapid capsid clearance from blood (90% by day 4) may improve safety profile.

• Consistent biomarker improvements across Western blot, mass spec, and positive fiber analyses.

• Significant reductions in ALT, AST, and embryonic myosin heavy chain, indicating improved muscle integrity and regeneration.

Cardiac and safety outcomes

• Cardiac function tracked via ejection fraction; subset of patients with low baseline values returned to normal or improved.

• No drug-induced liver injury, aHUS, TMA, or myocarditis observed; overall safety profile remains strong.

• Only one hospitalization for a lab abnormality, resolved with antibiotics.

• Troponin levels decrease and ejection fraction remains stable or increases over time.