Neurophth Therapeutics has received orphan drug designation from the FDA for NR082 for the treatment of Leber hereditary optic neuropathy associated with ND4 mutation, according to a press release.
Between 70% and 90% of LHON cases are caused by ND4 mutation, according to the release.
“[NR082] 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 effectively supplements the function loss caused by endogenous mutation,” Alvin Luk, PhD, MBA, CCRA, CEO
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