RPE65

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.

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.

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.

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.

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.

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.

WHAT WE HOPE TO UNDERSTAND