Jan 19, 2022LRATEluminex Biosciences Acquires Zuretinol Acetate from Retinagenix HoldingsEluminex announced today it acquired rights for a novel oral therapy to treat LCA or RP caused mutations of the RPE65 or LRAT gene.
Aug 16, 2021RPE65New Treatments for Retinitis PigmentosaExisting treatments only help a fraction of the estimated 100,000 Americans with this condition. But advances in gene therapy may soon help.
Apr 26, 2021RPE65An Update on Gene Therapy for Inherited Retinal Dystrophy: Experience in Leber Congenital Amaurosis This review provides an overview of retinal gene therapy development by summarizing significant contributions and important clinical trials.
Mar 12, 2021LCALCA, early-onset severe retinal dystrophy: current management and clinical trialsRPE65-related LCA pivotal trials, . . . have paved the way for a new era of genetic treatments in ophthalmology.
Feb 16, 2020RPE65Gene therapy to halt rare form of sight lossThe gene therapy is for patients who have retinal dystrophy as a result of inheriting a faulty copy of the RPE65 gene from both parents.
Apr 1, 2018Potential TreatmentsFirst Gene Therapy FDA-Approved for an Inherited Retinal DiseaseIn 1972, Friedman and Roblin proposed that it was theoretically possible to introduce “good” DNA to replace defective DNA.
Dec 19, 2017RPE65FDA Approves Gene Therapy for Inherited Blindness Developed by the UPenn and CHOPIn 2017, the FDA approved a gene therapy for RP caused by RPE65. The decision marked the first approved treatment for any form RP.