Join the club for FREE to access the whole archive and other member benefits.

Luxturna (voretigene neparvovec)

Prescription gene therapy product for treatment of inherited retinal disease

Voretigene neparvovec (Luxturna) is a novel gene therapy for the treatment of Leber's congenital amaurosis.  It was developed by Spark Therapeutics and Children's Hospital of Philadelphia. It is the first in vivo gene therapy approved by the FDA.


Leber's congenital amaurosis, or biallelic RPE65-mediated inherited retinal disease, is an inherited disorder causing progressive blindness. Voretigene is the first treatment available for this condition. The gene therapy is not a cure for the condition, but substantially improves vision in those treated. It is given as an subretinal injection.

Visit website: https://luxturna.com/about-luxturna/

See also: Company Novartis - Global medicines company for developing and producing drugs.

Details last updated 19-Feb-2020

Luxturna (voretigene neparvovec) News

First gene therapy for a rare eye disease, now NHS-approved

BBC - 17-Feb-2020

This life-changing new gene therapy will halt sight loss and improve vision

Read more...