RPE65 gene — Drug Target

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

Drug classes

Gene therapy

Marketed (1)

• voretigene neparvovec-rzyl · Spark Therapeutics, Inc. · Gene therapy · Ophthalmology
  Voretigene neparvovec-rzyl is a gene therapy that delivers a functional copy of the RPE65 gene into retinal cells to restore vision in patients with inherited retinal dystrophy caused by RPE65 mutations.

Phase 3 pipeline (1)

• AAV2-hRPE65v2,voretigene neparvovec-rzyl · Spark Therapeutics, Inc. · Gene therapy · Ophthalmology
  AAV2-hRPE65v2 is a gene therapy that delivers a functional copy of the human RPE65 gene into retinal pigment epithelium cells to restore vision in patients with inherited retinal dystrophy caused by RPE65 mutations.

Phase 2 pipeline (1)

• AAV2-hRPE65v2 · Spark Therapeutics, Inc. · Gene therapy · Ophthalmology
  AAV2-hRPE65v2 is a gene therapy that uses a viral vector to deliver a healthy copy of the RPE65 gene to the retina, treating inherited retinal dystrophy.