Eluminex Biosciences Acquires Zuretinol Acetate from Retinagenix Holdings
Eluminex announced today it acquired rights for a novel oral therapy to treat LCA or RP caused mutations of the RPE65 or LRAT gene.
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Aug 15, 2021
New Treatments for Retinitis Pigmentosa
Existing treatments only help a fraction of the estimated 100,000 Americans with this condition. But advances in gene therapy may soon help.
May 16, 2021
Dissecting the role of EYS in retinal degeneration: clinical and molecular aspects and its implications for future therapy
Ana B. Garcia-Delgado , Lourdes Valdes-Sanchez , Maria Jose Morillo-Sanchez , Beatriz Ponte-Zuñiga , Francisco J. Diaz-Corrales , Berta...
Apr 25, 2021
An 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, 2021
LCA, early-onset severe retinal dystrophy: current management and clinical trials
RPE65-related LCA pivotal trials, . . . have paved the way for a new era of genetic treatments in ophthalmology.
Jul 5, 2020
Biogen boosts gene therapy strategy with Harvard pact focused on inherited eye disease
Mutations in PRPF31 cause many more cases of RP than do mutations in RPE65, the gene targeted by Spark Tx Luxturna, Harvard researchers say.
Feb 16, 2020
Gene therapy to halt rare form of sight loss
The gene therapy is for patients who have retinal dystrophy as a result of inheriting a faulty copy of the RPE65 gene from both parents.
Mar 31, 2018
First Gene Therapy FDA-Approved for an Inherited Retinal Disease
In 1972, Friedman and Roblin proposed that it was theoretically possible to introduce “good” DNA to replace defective DNA.
Dec 19, 2017
FDA Approves Gene Therapy for Inherited Blindness Developed by the UPenn and CHOP
In 2017, the FDA approved a gene therapy for RP caused by RPE65. The decision marked the first approved treatment for any form RP.
May 15, 2017
Mutation screening in genes known to be responsible for Retinitis Pigmentosa in 98 Small Han Chinese Families
Results extended the mutation spectrum of known RP genes in Han Chinese, making a contribution to RP gene diagnosis and the pathogenetic . .
Oct 29, 1999
Leber Congenital Amaurosis
Leber's congenital amaurosis (LCA) is the earliest, most severe form of inherited retinal dystrophies responsible for congenital blindness.
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