First UK patients get Novartis' Luxturna gene therapy for blindness

[Bayard]

The first NHS patients have started treatment with Novartis’ Luxturna, a gene therapy for a sight-robbing inherited disease, after the drugmaker agreed a discount on its £613,000 list price last year.

Luxturna (voretigene neparvovec) was recommended for NHS funding in September for use in patients with vision loss caused by inherited retinal dystrophy from confirmed RPE65 gene mutations, including children with Leber’s congenital amaurosis (LCA).

At the time, cost-effectiveness watchdog NICE said around 86 people in England would be eligible for the treatment, which has been shown in trials to prevent progressive sight loss and even improve vision in some patients. NHS England now says that number could be as high as 100.

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The therapy is however only suitable for patients who retain some degree of vision, and there needs to be sufficient retinal cells remaining to take up and express the gene therapy.

https://pharmaphorum.com/news/first-uk-patients-get-novartis-luxturna-gene-therapy-for-blindness

[Duppers]

1. Wow, that's wonderful news.

Sadly, many RWers still confuse "messing-with-god's-work" gene therapy with genetic engineering. This is...

CRISPR technology to repair the DNA of defective nonreproductive cells — allowing them to treat cancer, genetic disorders and other diseases. In gene therapy, the genetic changes affect only the patient. In genetic engineering, scientists alter the entire genetic structure of the resulting human being — changes that are then passed on to future generations.


Long live good science!