pp-165 Suspension for injection
Dossier type
CTD
Dossier status
Under development
Country of origin
United States
GMP approvals
FDA
Comments
Indication: Geographic atrophy secondary to AMD (OCU410); Stargardt disease (OCU410ST)
Clinical stage: Phase 2/3 ongoing
Modality: Gene therapy (AAV5 vector encoding RORA nuclear hormone receptor)
Mechanism / Target: Delivers RORA transcription factor — reduces complement activation and oxidative stress in RPE cells; anti-inflammatory, anti-oxidant and neuroprotective effects in atrophic retinal conditions
Route / form: Subretinal injection (surgical)
Differentiation: First gene therapy approach to GA (complements anti-complement drugs pegcetacoplan/avacincaptad pegol); modifier approach for Stargardt independent of ABCA4 mutation type; single administration
Manufacturer #39190
Clinical-stage gene therapy company focused on retinal degenerative diseases. Develops modifier gene therapy enabling mutation-agnostic treatment across multiple RP genotypes. Wholly owned platform with Phase 3 assets in retinitis pigmentosa and geographic atrophy.
None