Spark Therapeutics, Inc.

Quick facts

Marketed products

• voretigene neparvovec-rzyl · 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

• AAV2-hRPE65v2,voretigene neparvovec-rzyl · 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

• AAV2-hRPE65v2 · 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.

Phase 1 pipeline

• AAV2-hCHM · Ophthalmology
  Gene therapy for Leber congenital amaurosis

Competitive intelligence

For the full portfolio CI brief — phase mix, recent regulatory actions, upcoming PDUFA dates, patent timeline, class-overlap with competitors:

• Spark Therapeutics, Inc. portfolio CI brief
• Spark Therapeutics, Inc. pipeline updates RSS