![[FROM LEFT] Claudia Popp, Olga Tkachenko and Marta Carnielli](https://ds982gbwnvyso.cloudfront.net/Pictures/274x183/4/4/1/441_5m4a4767_toprasymposium2022_day3_20221019_816643.jpg)
Case Study- Luxturna
The ATMP Luxturna (voretigene neparvovec) is a GTMP which utilises a recombinant adeno-associated viral vector serotype 2 (rAAV2) capsid as a delivery vehicle for the human retinal pigment epithelium 65 kDa protein (hRPE65) cDNA to the retina. Luxturna is approved for patients with retinal dystrophy associated with biallelic RPE65 mutations ...
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