[Njtechdiv] History Is Made: FDA Approves Spark’s Vision-Restoring Gene Therapy

[Mario Brusco]

if interested, I encourage anyone to read the full blog post at the 
address below. also, consider to have yourself tested for the gene 
mutation if you're interested in this matter with the advisement of your 
ophthalmologist or vision specialist.

History Is Made: FDA Approves Spark’s Vision-Restoring Gene Therapy
http://www.blindness.org/blog/index.php/history-is-made-fda-approves-sparks-vision-restoring-gene-therapy/

By Ben Shaberman
  on December 20, 2017

Spark Therapeutics’ vision-restoring RPE65 gene therapy has received 
marketing approval from the U.S. Food and Drug Administration, becoming 
the first gene therapy to gain regulatory approval in the U.S. for the 
eye or any inherited condition.

Known as LUXTURNA™ (voretigene neparvovec), the gene therapy restored 
vision in a clinical trial for people between the ages of 4 and 44 with 
Leber congenital amaurosis (LCA) caused by mutations in the gene RPE65. 
Study participants with severe vision loss reported putting away their 
navigational canes, seeing stars, being able to read, and recognizing 
faces of loved ones. Vision restoration has persisted for at least three 
years. The treatment is also designed to work for people with retinitis 
pigmentosa (RP) caused by RPE65 mutations.

...

“This is truly a historical moment in the fight against blindness. We 
are delighted by the FDA’s approval of LUXTURNA for people with RPE65 
mutations,” says Stephen Rose, PhD, chief research officer at FFB. 
“LUXTURNA’s approval also provides affirmation that gene therapies can 
be a safe, effective, and commercially viable approach to treating many 
forms of blindness, providing a big boost to an already burgeoning 
field. The approval is great news for a broad spectrum of people with 
inherited retinal diseases.”

Retinal gene therapy clinical trials are underway for people with a wide 
range of retinal diseases including: X-linked RP, choroideremia, Usher 
syndrome type 1B, Stargardt disease, X-linked retinoschisis, and 
achromatopsia.