China-based gene therapy specialist Neurophth Biotechnology Ltd has announced obtaining Investigational New Drug (IND) approval from the US FDA for its second ophthalmology gene therapy, NFS-02 (rAAV2-ND1). The intended indication is Leber’s hereditary optic neuropathy (LHON) caused by the ND1 mutation.
NFS-02: A Promising Gene Therapy for LHON
NFS-02, a recombinant adeno-associated virus serotype 2 vector (rAAV2) product, is being developed to treat LHON associated with the ND1 mutation. Utilizing the recombinant adeno-associated virus as a carrier, the gene therapy can deliver the correct gene to the injured optic ganglion cells of patients through a single intravitreal injection. This process repairs the mitochondrial biological respiratory chain and restores the vitality and visual function of optic ganglion cells.
Global Multi-Center Study and Orphan Drug Designation
The approved global multi-center, single-arm, open-label, dosage exploration Phase I/II study is designed to assess the safety, tolerability, and efficacy of NFS-02. The therapy has also obtained Orphan Drug Designation (ODD) status from the US FDA, highlighting its potential to address a significant unmet medical need in patients with LHON.-Fineline Info & Tech
