Is there any new research being done for Leber congenital amaurosis?
There is and it’s called Luxterna.
What is Luxterna?
It’s an investigational, potential one-time gene therapy, for the treatment of patients with vision loss due to biallelic RPE65-mediated inherited retinal disease.
Let me say that again in English.
RPE65-mediated inherited retinal disease relates to several inherited retinal diseases but in particular, leber’s congenital amaurosis (LCA) as well as autosomal recessive retinitis pigmentosa.
Mutations in the RPE65 gene result in reduced vision. By injecting a working copy of the RPE65 gene subretinally, patients were able to regain some lost vision. Results of the phase 3 trial showed Luxterna administration led to clinically meaningful and statistically significant improvements in ability to navigate independently in low-to-moderate light conditions.
If approved, Luxterna has the potential to be the first FDA approved gene therapy for the eye. This drug was given priority review through the FDA which means it can take only 6 months to approve as compared to 12 months. The FDA set a deadline for approval as January 2018.