FDA assigns orphan drug designation to gene therapy NR082 for treatment of Leber's Hereditary Optic Neuropathy associated with ND4 mutation
The treatment uses recombinant adeno-associated virus serotype 2 to deliver the genetically modified ND4 gene. After a single intravitreal injection, the gene is translated and expressed in cells, which supplements the function loss caused by endogenous mutation.
Source:
Biospace Inc.