Neurophth

Neurophth Biotechnology Co., Ltd. ("Neurophth") is the first company in China to start gene therapy for ophthalmic diseases. As a clinical-stage R&D enterprise, the company is dedicated to exploring and developing new therapies for patients with ophthalmic diseases worldwide. Founded in 2016, Neurophth is the culmination of Professor Li Bin's team's over a decade of exploration and accumulation in ophthalmic gene therapy technology since 2008 at Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology. As an ophthalmologist and retinal disease expert, Professor Li led the team to successfully complete translational research from laboratory to clinical application, and through a series of domestic and international multicenter clinical trials, confirmed the application value of adeno-associated virus (AAV) vector technology in ophthalmic gene therapy. The company's drug candidate NFS-01 is intended to treat Leber's hereditary optic neuropathy (LHON). This disease is a degenerative disorder that can cause severe vision impairment, and to date there is no effective treatment. In 2011, the team began the world's first exploratory clinical study of LHON gene therapy using this drug candidate. The 9 patients participating in the clinical trial have been followed up for 8 consecutive years, with no serious adverse reactions to date, and 5 of them showed significant improvement in vision. These results represent the longest follow-up record for gene therapy worldwide, and were published in Nature sub-journals, The Lancet sub-journals, and the top-tier ophthalmology journal Ophthalmology, fully demonstrating the long-term safety and efficacy of clinical application of gene therapy technology. After the encouraging results of the first study, Professor Li's team conducted a more comprehensive clinical study between 2017 and 2018, enrolling 159 subjects (including 10 from Argentina), which is currently the clinical trial with the largest sample size in the entire global gene therapy field. Similarly, the twelve-month follow-up results showed that NFS-01 had significant efficacy, and no serious adverse reactions were found. Leveraging its mature AAV gene therapy technology platform, Neurophth has established a rich product pipeline, including more than 10 ongoing projects targeting various ophthalmic diseases such as optic nerve damage diseases and vascular retinopathies. Our company is located in Wuhan, Suzhou, Shanghai, and the United States. With the same mission and vision, the company has brought together a team of experts with extensive industry and international experience, committed to serving patients worldwide and making Neurophth a leader in global ophthalmic gene therapy and an internationally competitive innovative technology company. Website/