Neurophth is an integrated genomics medicine company whose leading investors include CMG-SDIC Capital, Sequoia Capital China, Sunshine Insurance, and China Merchant Bank International Capital.

Neurophth's lead candidate, NR082 (rAAV-ND4, NFS-01), is the first adeno-associated virus 2-based gene therapy to receive investigational new drug application approval in China, with the first patient being dosed in June 2021. NR082, a gene therapy candidate for the treatment of ND4-mediated Leber hereditary optic neuropathy, has been also granted the orphan drug designation by the U.S. Food and Drug Administration.

Neurophth's gene therapy granted orphan drug designation
Neurophth Therapeutics' lead candidate, NR082, an adeno-associated virus-based gene therapy, has been granted orphan drug designation by the U.S. Food...