NEW YORK – Neurophth on Thursday said the first patient with ND1-mutated Leber hereditary optic neuropathy (LHON) has received its investigational gene therapy NFS-02 in a Phase I/II trial.
The Wuhan, China-based firm, which last week announced it had raised nearly $95 million in a financing round, is evaluating the safety, tolerability, and efficacy of NFS-02 in the Phase I/II trial. The single-arm dose-finding study is enrolling patients with LHON caused by ND1 mitochondrial mutations in the US and China.
LHON, a maternally inherited disease, leads to vision loss in young men and is caused largely by mutations in the mitochondrial genes ND1, ND4, or ND6. NFS-02 is designed to deliver normal ND1 genes via a recombinant adeno-associated virus to patients' damaged optic ganglion cells and aims to restore the cells' visual function by repairing the mitochondrial respiratory chain.
The first dose of the gene therapy in the study was administered to a patient at the Eye Hospital of Wenzhou Medical University in Wenzhou, China. "In the coming months, we will continue to accelerate the patient enrollment process of the China-US Phase I/II clinical trial," Neurophth Chief Medical Officer Xiaoning Guo said in a statement. "Furthermore, we are actively exploring other indications where gene therapy may offer significant therapeutic benefit in the hope of meeting more unmet clinical needs."
Neurophth's most advanced gene therapy in its pipeline is NR082 (rAAV2-ND4), which is undergoing a Phase III trial in patients with mtND4-mediated LHON. That trial is fully enrolled in China.