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Neurophth has raised RMB130 million ($18.4 million) in a series A round with plans to bring an ophthalmology gene therapy to market in three to five years.
Sequoia Capital China and Fosun’s InnoStar Venture led the round; Northern Light Venture Capital also participated.
Wuhan Neurophth Biological Technology Inc. is developing a pipeline of seven preclinical gene therapies for ocular diseases such as Leber hereditary optic neuropathy (LHON) and building a GMP gene therapy production platform.
The company was co-founded in 2016 by Bin Li and Dabo Cai and raised an RMB21 million angel round in 2018 backed by Northern Light, BGI’s Miracle Light and BOHE Angel Fund. Su Xiao joined as co-founder in 2019.
Xiao serves as CEO and sits on Neurophth’s board; she was previously an associate director of upstream product development and GMP manufacturing at Vigene Biosciences Inc. and a senior scientist at Pfizer Inc. (NYSE:PFE). Cai, who is general manager, is a 12-year veteran of Pfizer and Novartis AG (NYSE:NVS; SIX:NOVN).
Li, who serves as CSO, has engaged in research and treatment of ophthalmic diseases for 25 years and is a professor of ophthalmology at Puyang Medical College. Li initiated the first gene therapy clinical trial for LHON in 2011 while he was a professor and chief ophthalmologist at Huazhong University of Science and Technology's Tongji Hospital.
The trial evaluated an adeno-associated virus serotype 2 (AAV2) vector encoding ND4 in LHON patients harboring the G11778A mutation in ND4; the candidate is now in an investigator-initiated Phase II/III trial in 159 patients. In a February Ophthalmology article, Li reported seven-year follow-up data from the initial study showing the therapy improved best-corrected visual acuity in six of eight evaluable patients. The company plans to report data from the Phase II/III trial at the Association for Research in Vision and Ophthalmology and American Society of Gene and Cell Therapy conferences.
Xiao told BioCentury that Neurophth is developing a gene therapy with the same AAV2 construct, but with an improved manufacturing process and quality control systems.
Neurophth has completed pilot production of its LHON therapy and is preparing for IND submissions in China and US. The company, which plans to sell its products globally, has established Shanghai and U.S. subsidiaries and expects to enter U.S. and European markets in three to five years.
The company plans to raise a series B round by year-end to support U.S. and China clinical trials, said Xiao.
Santhera Pharmaceuticals Holding AG (SIX:SANN) markets Raxone idebenone in the EU for LHON; there are no FDA-approved therapies for the indication.
GenSight Biologics S.A. (Euronext:SIGHT) is also developing a gene therapy for LHON; its AAV2-based ND4 gene therapy Lumevoq (GS010) is in Phase III testing.
The company plans to expand its pipeline to include non-ophthalmology gene therapies for sensorineural hearing loss and other genetic diseases, said Xiao.
Neurophth, which is located in what was the epicenter of China’s COVID-19 outbreak, also hopes to use its platform to develop undisclosed gene therapies to achieve long-term protection against COVID-19 by blocking virus infection and amplification in host cells.
ND4 - NADH dehydrogenase subunit 4