pp-191 Solution for injection

Indication: Non-syndromic retinitis pigmentosa (RP) — vision restoration in patients with advanced photoreceptor loss Clinical stage: BLA under rolling submission to FDA (pivotal Phase 2/3 data filed) Modality: Biological (AAV2-based optogenetic gene therapy) Mechanism / Target: AAV2 vector delivering multi-characteristic opsin (MCO) gene to degenerate retinal bipolar and ganglion cells; MCO protein restores light-responsiveness independent of remaining photoreceptors; mutation-agnostic mechanism Route / form: Single intravitreal injection Differentiation: First-in-class optogenetic gene therapy for RP; FDA RMAT Designation; mutation-agnostic (treats all genetic forms of RP unlike gene-specific therapies); BLA under rolling FDA submission 2025; single-injection treatment;