Company Introduction

Neurophth is China's leading in-vivo gene therapy company for ophthalmic diseases. As a clinical-stage company, Neurophth is dedicated to discovering and developing gene therapies for patients suffering from blindness and other eye diseases globally.

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.

Company History
Our Story Started from 2008
  • 2008
    Prof. Bin Li started the research on gene therapy of Leber’s Hereditary Optic Neuropathy(LHON)
  • 2011
    Prof. Li’s team finished the world’s first investigational gene therapy study for LHON (IIT1)
  • 2016.07
    Wuhan Neurophth Biological Technology Limited Company was founded
  • 2017
    Prof Li’s team finished the first-patient-enrollment for the world’s first international LHON gene therapy study (IIT2)
  • 2018
    Prof Li’s team finished the enrollment of 159 subjects (from China and Argentina) for IIT2, which makes it the world’s largest gene therapy study
  • 2018.07
    Raised $2.3 Million series Angel round, led by Huada Miracle Lights and followed by BOHE Angel Fund and Northern Light Venture Capital
  • 2019.12
    Raised $18.4 Million series A round, led by Sequoia Capital China and Fosun’s InnoStar Venture, and followed by Northern Light Venture Capital
  • 2020.01
    NEUROPHTH THERAPEUTICS INC was founded in the U.S.
  • 2020.04
    Neurophth (Shanghai) Biological Technology Limited Company was founded
  • 2020.05
    Neurophth (Suzhou) Biological Technology Limited Company was founded
  • 2020.08
    Prof. Li’s team published the IIT1 clinical data of nearly 8 years’ follow-up in the journal of Ophthmology
  • 2020.09
    NR082(NFS-01 project) was granted Orphan Drug Designation by US FDA
  • 2021.01
    NMPA Accepted NR082(NFS-01 project) 's IND submission
  • 2021.02
    Closed RMB 400 million ($61.9 million USD) Series-B financing with a premier syndicate of investors, co-led by Guofang Capital and InnoVision Capital, with participating from existing investors Sequoia Capital China and Northern Light Venture Capital.  
  • 2021.03
    NMPA approved the Company’s IND application of NR082 (recombinant adeno-associated virus serotype 2 carrying ND4 gene; rAAV2-ND4) for LHON
  • 2021.09

    Suzhou Manufacturing Facility for Gene Therapy Products was completed

  • 2021.11

    Closed over $60 million USD Series-C financing with a premier syndicate of investors, co-led by CMG-SDIC Capital and Sequoia Capital China, with participating from new investors Sunshine Insurance and China Merchant Bank International Capital

  • 2022.01

    FDA approved the Company’s IND application of NR082 ( rAAV2-ND4 ) for LHON

  • 2022.01
    NFS-02 rAAV2-ND1 ) was granted Orphan Drug Designation by US FDA
  • 2022.01
    NR082 rAAV2-ND4 ) was granted Orphan Drug Designation by EMA
Our story is continuing...
Management Team
  • Prof. Bin Li

    Founder, Chairman, Chief Executive Officer

  • Su Xiao Ph.D.

    Co-founder, Chief Technology Operating Officer

  • Prof. Qiutang Li

    Chief Scientific Officer

  • Bose Kalampanayil, Ph.D

    Chief Technology Officer

  • Su Zhang

    Chief Financial Officer

  • Xiaoning Guo Ph.D.

    Chief Medical Officer

  • Dabo Cai

    Co-founder, Vice-General Manager, Head of sale operation

  • Yiyuan Chen

    Senior Vice President, Regulatory Affairs

  • Eric Lin

    Secretary of the Board, Vice President

  • Yang Cao Ph.D.

    Vice-President, Quality

  • Luke Li Ph.D.

    Vice President, Commercial

  • Jane Yang

    Executive Director, Human Resources and Administration

  • 300+
  • 57%
    Ph.D. & M.S.
  • 34%
    Staff hold over 10 years of experience in bio-industry
  • 70%
    Directors & above have oversea experince