Gene Therapies for Inherited Retinal Diseases

Abstract

Gene therapy is an emerging therapeutic modality that has demonstrated early success in treating genetic ocular disorders. The approval of voretigene neparavovec-ryzl (tradename Luxturna®), the first single-dose in vivo gene therapy and only on-market treatment for Leber congenital amaurosis Type 2, marked a turning point for inherited retinal diseases. Since then, dozens of in vivo gene therapy products have reached clinical development for other IRDs, including retinitis pigmentosa, achromatopsia, Leber hereditary optical neuropathy , X-linked retinoschisis, and Usher syndrome type 2. This review highlights the clinical and genetic landscape of inherited retinal diseas es targeted by ongoing clinical-stage gene therapy development and outcomes to date for patients treated with voretigene neparvovec. Finally, we discuss trends in gene therapy pricing and approaches in determining the cost effectiveness of single-dose gene therapies for IRDs.