Neurophth Announces IND Approval by the NMPA for Second Gene Therapy NFS-02 (rAAV2-ND1)
Source:   Release Date: 2023-04-15

WUHAN and SAN DIEGO, April 17, 2023 – Neurophth Therapeutics, Inc. (“Neurophth”) announced today that the China National Medical Products Administration (NMPA) has approved the Company's Investigational New Drug (IND) application of NFS-02 (rAAV2-ND1).


Investigational NFS-02 is a novel gene therapy product that is being developed for the treatment of Leber hereditary optic neuropathy (LHON) associated with mtND1 mutations. Neurophth is conducting a Phase I/II, international multi-center study aimed at evaluating the safety, tolerability, and efficacy of NFS-02 in LHON patients with ND1 mutations. Previously in 2022, NFS-02 was granted orphan drug designation and IND clearance by the U.S. FDA.


“We are delighted that NFS-02 has received IND approval in China. It marks the second gene therapy to enter clinical trials after NR082 and demonstrates our strong R&D capabilities and operations.” said Professor Bin Li, Founder, Chairman and CEO of Neurophth, “Neurophth has accumulated a wealth of experience through long-term research. In March, a Maltese patient came to China to find NFS-02 and participated in the investigator-initiated trial, as it’s currently the only gene therapy for ND1-LHON. We will continue to execute our patient-first evaluation in the future.”


Dr. Xiaoning Guo, Chief Medical Officer of Neurophth, said, “NFS-02 is currently the only gene therapy for ND1-LHON under development worldwide, and there is still a significant unmet clinical need. In previous investigator-initiated trials (IITs), NFS-02 demonstrated safety and efficacy. We look forward to obtaining positive and reliable data from the clinical trials to pave the way for early drug approval and commercialization, making our vision of 'building a brighter future for patients' a reality.”


Ms. Yiyuan Chen, Senior Vice President and Head of Global Regulatory Affairs of Neurophth, commented, “NFS-02 received the IND clearance from the U.S. FDA in December 2022, and we are thrilled to receive another IND approval from the NMPA. We will continue to implement our global regulatory strategy to support the new drug development, making gene therapy accessible to more patients.”


About Neurophth

Neurophth is China’s leading gene therapy company for ophthalmic diseases. With subsidiaries in China (Wuhan, Shanghai, and Suzhou) and US (San Diego, California), Neurophth, a fully integrated company, is striving to discover and develop genomic medicines for patients suffering from genetic diseases globally. Our validated AAV platform, which has been published in Nature - Scientific Reports, Ophthalmology, and EBioMedicine, has successfully delivered proof-of-concept investigator-initiated trials data of 186 subjects with investigational gene therapies in the retina. Our most advanced investigational gene therapy drug candidate, NR082 (rAAV2-ND4), in development for the treatment of mtND4-mediated LHON, has been granted orphan drug designation (ODD) by the U.S. FDA and EMA. After the IND clearance by the China NMPA in March 2021 and the U.S. IND by FDA in January 2022, Neurophth has completed patient enrollment for Phase III clinical trial in China and initiated multiple clinical research centers in the U.S. Recently, our second gene therapy drug candidate NFS-02, has been granted IND clearance from U.S. FDA and NMPA. The pipeline also includes autosomal dominant optic atrophy, optic neuroprotection, vascular retinopathy, and five other preclinical candidates. To learn more about us and our growing pipeline, visit