Clinical MilestonesAnalysts point to multiple Duchenne muscular dystrophy programs progressing toward initial in‑patient clinical readouts in 2026, and positive efficacy signals could materially lift the stock by demonstrating therapeutic benefit.
Pipeline Expansion Into OphthalmologyAdvancement of ocular programs toward a first clinical candidate expected in 2026 broadens the company's addressable markets beyond neuromuscular diseases and may attract new partnerships or investor interest.
Platform ValidationAnalysts expect that successful results from candidates using the company's endosomal escape vehicle delivery platform would validate its differentiated efficacy and safety profile, boosting confidence across the pipeline.