Leber’s hereditary optic neuropathy (LHON), a maternally inherited disease resulting in simultaneous or sequential bilateral visual loss, currently has no standard treatment.
Gene therapy, meanwhile, is seen as the first prospective treatment for this genetic disease, as an article published by Nature revealed in 2016. Since then, a couple of innovative biotechs flushed with funds and genetic data have initiated the first drugs for the market. Yet hitherto there has been no company from China.
Neurophth (纽福斯), the first-ever Chinese biotech trying to develop gene therapeutics for ophthalmological diseases, has announced that it carried out its Series A funding round on April 8, worth CNY 130 million and led by Fosun and Sequoia Capital China.
The funds raised will be invested in the translational research center for inherited retinal diseases and GMP manufacturing facility for AAV-based gene therapy products, clarified the company to EqualOcean. The young biotech firm was first financed by Miracle Light, BOHE Angel Fund and Northern Light Venture Capital in 2018.
Professor Bin Li, the founder of the firm, conducted the second international clinical trial in 159 patients with LHON. Preliminary safety and efficacy data from the 12th month of the trials were similar to the first clinical trial, with no safety concerns, thus "demonstrating that AAV2-ND4 gene therapy is a promising treatment for LHON," said the gene therapy company's global drug development advisor Alvin Luk to EqualOcean.
Elsewhere the first clinical trials were led by institutions in the EU region and the US. GenSight Biologics (SIGHT: EPA), a French biotech in gene therapy, has its candidate in the Phase III trial for ND4-mediated LHON.
Chinese gene therapies and biotechnologies have lately been mentioned with the spotlight on one Shenzhen-based firm, BGI Genomics (300676:SH), whose subsidiary venture capital Miracle Light invested in the previous round of Neurophth. Gene therapies are among the newest innovations offered by global biotechnological companies, with prices reaching USD 850,000 to USD 2.1 million despite little being known about their full potential or aftereffects. The theoretical risks and insurance-related problems shall be watched carefully by investors in the coming period.
Neurophth was founded by Prof Bin Li's team at Wuhan Tongji Hospital in 2016, the hospital that has recently drawn attention for its fight against the pervasive coronavirus.