Source: Press Release
January 28, 2022
1 min read

Gene therapy for Leber hereditary optic neuropathy advances in Europe, US

Source: Press Release
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NR082, a gene therapy drug candidate for the treatment of Leber hereditary optic neuropathy, was granted orphan drug designation by the European Medicines Agency, according to a press release from Neurophth Therapeutics.

“The positive opinion from the [Committee for Orphan Medicinal Products] acknowledges the compelling IND-enabling data and clinical data of 186 subjects from three investigator-initiated trials,” Bin Li, MD, PhD, founder of Neurophth, said in the release.

In addition, the FDA recently cleared an investigational new drug application for NR082 for the treatment of Leber hereditary optic neuropathy associated with ND4 mutation, according to a separate release. U.S. clinical trial enrollment is expected to begin this year.

Three investigator-initiated trials demonstrated safety and efficacy of the therapy, with one trial showing clinical durability up to 90 months.

In 2020, the FDA granted orphan drug designation to NR082.


  • Neurophth Therapeutics receives IND clearance to initiate clinical trial for China AAV-ND4 gene therapy NR082 in Leber hereditary optic neuropathy. Published Jan. 18, 2022. Accessed Jan. 27, 2022.