RPE65 — Drug Target

All drugs that target RPE65 — marketed and clinical-stage. Includes 1 drug classes acting on this target.

Drug classes

Gene therapy

Phase 2 pipeline (1)

• AAV OPTIRPE65 · MeiraGTx UK II Ltd · Gene therapy · Ophthalmology
  Gene therapy targeting RPE65

Phase 1 pipeline (2)

• AAV2-hCHM · Spark Therapeutics, Inc. · Gene therapy · Ophthalmology
  Gene therapy for Leber congenital amaurosis
• AAV RPE65 · MeiraGTx UK II Ltd · Gene therapy · Ophthalmology
  Gene therapy for RPE65-mediated inherited retinal dystrophy