China-based gene therapy specialist Neurophth Biotechnology Ltd has announced that the first patient has been dosed in a Phase III clinical study for its NR082 (rAAV2-ND4, NFS-01). The study is being conducted in China to evaluate the efficacy and safety of NR082, a recombinant adeno-associated virus serotype 2 (rAAV2), in treating ND4-mediated Leber’s hereditary optic neuropathy (ND4-LHON).
Understanding Leber’s Hereditary Optic Neuropathy (LHON)
Leber’s hereditary optic neuropathy (LHON) is a maternally inherited mitochondrial disease with an incidence rate of approximately 1 in 31,000 to 1 in 54,000. Up to 90% of LHON cases are caused by three mitochondrial DNA mutations, with the G11778A mutation in ND4 being the most common, accounting for about 50% to 80% of cases. Currently, there is no effective treatment available for LHON.
NR082: Mechanism and Development
NR082 is a novel intraocular gene therapy designed to treat LHON associated with the ND4 mutation. The therapy uses recombinant adeno-associated virus as a carrier to deliver the correct gene to the damaged optic ganglion cells of patients through a single intravitreal injection. This process repairs the mitochondrial respiratory chain, restores the vitality of optic ganglion cells, and improves visual function.
Regulatory Milestones and Pipeline
Neurophth’s NR082 (rAAV2-ND4, NFS-01) is the first in vivo ophthalmology gene therapy to receive clinical trial approval in China. The drug has also obtained Orphan Drug Designation (ODD) in the US for use in ND4-LHON. Neurophth is further developing therapies for other conditions, including ND1-mediated LHON, autosomal dominant optic atrophy, optic nerve protection, and vascular retinopathy.
Future Outlook
The initiation of the Phase III clinical study for NR082 marks a significant step forward in the development of gene therapies for LHON. With its innovative approach and regulatory recognitions, Neurophth is well-positioned to address the unmet needs of patients suffering from this debilitating condition.-Fineline Info & Tech
