pp-164 Suspension for injection
Dossier type
CTD
Dossier status
Under development
Country of origin
United States
GMP approvals
FDA
Comments
Indication: Retinitis pigmentosa (RP) — multiple genetic subtypes (RHO, PDE6B, RPGR mutations and others)
Clinical stage: Phase 3 — liMeliGhT trial ongoing
Modality: Gene therapy (AAV5 vector encoding NR2E3 nuclear hormone receptor)
Mechanism / Target: Delivers NR2E3 transcription factor — regulates photoreceptor development, survival and gene expression networks; mutation-agnostic modifier mechanism supports photoreceptor survival independent of primary mutation
Route / form: Subretinal injection (surgical)
Differentiation: Mutation-agnostic approach covers >50% of RP patients across genetic subtypes with a single product; unlike voretigene neparvovec (only RPE65 mutation); potential for broad RP market
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.
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