Founded in 2016 as a result of the technology and over 20 years of clinical practice and academic experience by Prof. Li Bin's team at Tongji Hospital of Tongji Medical College, Prof. Li conducted bench-to-bedside gene therapy studies, led to a series of China and international multi-center clinical trials that characterized the adeno-associated viral (AAV) vectors in inherited blindness.

The company's lead project, NR082( NFS-01,rAAV2-ND4), is designed to treat Leber's hereditary optic neuropathy (LHON), a maternally inherited degenerative disease leads to serious visual loss which there is currently no effective treatment. NR-082has been studied in 168 patients through two investigator-initiated trials (IITs) initiated in 2011 and 2017. In 2011, the team initiated the world’s first investigational AAV-mediated gene therapy study for ND4-associated LHON. After nearly 8 years’ long-term follow-up, the treatment displayed good safety profiles and have been maintained significant visual improvement as of today. These results demonstrated long-term safety and durability of clinical application of gene therapy technology were published in Ophthalmology 2020. Encouraged by the initial success, Prof. Li's team has completed an international gene therapy clinical study during 2017-2018 in 159 LHON patients in China and Argentina, which is the world's largest clinical trial for gene therapy. Similarly, the 12-month follow-up results showed that NFS-01 has remarkable curative effect and no obvious serious adverse events have been found. The result of these two studies were presented at ARVO and ASGCT 2020. NR-082 was also granted an orphan drug designation (ODD) by the U.S. Food and Drug Administration for the treatment of LHON associated with ND4 mutation in September 2020.

Leveraging the validated AAV platform, Neurophth has developed a rich pipeline of 10 products, aiming to serve patients with various retinal diseases including optic nerve damage-related diseases and vascular retinopathy. Headquartered in Wuhan with subsidiaries in Shanghai, Suzhou and US, we are a global, experienced team united by the same passionate commitment - to improving the lives of patients worldwide, and to making Neurophth a world leading company in ocular gene therapy and a globally competitive innovative biotechnology company.