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.

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Biospace Inc.