Neurophth Secures RMB 130 Million Series A Financing Led by Sequoia Capital China and Fosun Future Healthcare to Advance Ophthalmic Gene Therapy

Recently, VCBeat learned that Wuhan Neurophth Biotechnology Limited Company (“Neurophth”), China’s first company dedicated to the research and development of gene therapy drugs for ophthalmic diseases, has completed a RMB 130 million Series A financing round. The round was led by Sequoia Capital China Fund and Fosun Star Future Capital, with Northern Light Venture Capital participating as a co-investor. The funds will be used to construct a GMP-compliant production facility for ophthalmic gene therapy drugs, advance the clinical development of its gene therapy drug for Leber’s Hereditary Optic Neuropathy (LHON), and support the R&D of gene therapies for other diseases. Previously, in August 2018, Neurophth had completed its angel financing round, led by Miracle Light (a subsidiary of BGI), with Mint Angel Fund and Northern Light Venture Capital as co-investors.

In the film *Adoring*, released at the end of 2019, Wu Lei portrays Chen Leyun, a teenage patient who lost his vision due to Leber’s Hereditary Optic Neuropathy (LHON). For many people, this disease may be entirely unfamiliar. LHON is an ophthalmic genetic disorder caused by mitochondrial gene mutations and follows a maternal inheritance pattern. It predominantly affects males aged 14–21 years. Patients typically experience bilateral onset, with vision deteriorating rapidly to blindness, making it one of the globally recognized causes of blindness among adolescents. Currently, there are no effective clinical treatments for this condition. The efficacy of therapeutic approaches such as pharmacological treatment, acupuncture, and stem cell therapy has been less than promising. Gene therapy stands as the only currently feasible and effective treatment option.

“The eye is a closed spherical structure, and many hereditary ophthalmic diseases are associated with genetic mutations. Therefore, gene therapy is widely recognized as an effective approach for treating inherited eye disorders,” Professor Li Bin, Founder and Chairman of Neurophth, told VCBeat. In the global field of ophthalmic gene therapy research, gene therapies for inherited eye diseases such as retinitis pigmentosa, choroideremia, Leber’s hereditary optic neuropathy (LHON), Leber’s congenital amaurosis (LCA), and Stargardt’s disease have garnered significant attention and have sequentially achieved phased breakthroughs. In December 2017, Luxturna, the first ophthalmic gene therapy drug developed by the U.S. biotechnology company Spark Therapeutics, received FDA approval for the treatment of Leber’s congenital amaurosis type 2 (LCA2). However, with a price tag of up to $850,000 per dose, this medication is virtually unaffordable for ordinary families.

Professor Li Bin, Founder of Neurophth

“We aim to develop a gene therapy drug that is affordable for Chinese patients,” stated Professor Li Bin. Prior to founding his company, Professor Li was an ophthalmologist who spent 25 years engaged in the diagnosis, treatment, and scientific research of ocular diseases at the Zhongshan Ophthalmic Center of Sun Yat-sen University and Tongji Hospital affiliated with Tongji Medical College of Huazhong University of Science and Technology. Since 2008, Professor Li has been conducting research on gene therapy for Leber’s Hereditary Optic Neuropathy (LHON).In 2011, Professor Li Bin’s team completed the world’s first clinical study on gene therapy for Leber’s hereditary optic neuropathy.Of the nine patients who participated in the clinical trial, eight years of follow-up data have been collected to date. Among them, six patients with Leber’s hereditary optic neuropathy (LHON) showed significant improvement in visual acuity, and three LHON patients demonstrated marked expansion of their visual fields. These results have been published in subspecialty journals of Nature and The Lancet, as well as in top-tier ophthalmology journals. In 2016, this study received"One of the Top 10 Global Advances in Gene Therapy," "Top 10 News in Ophthalmology in China," and "Top 10 Research Highlights of the Retina Disease Group" in 2017such honors. During the same period, Professor Li Bin founded Neurophth, focusing on the research and development of ophthalmic gene therapies, located in Wuhan Optics Valley Biological City, a national-level biotechnology industrial park.

Dr. Su Xiao, CEO and Co-founder of Neurophth, holds a Bachelor’s degree in Chemical Engineering and Industrial Biological Engineering from Tsinghua University and a Ph.D. in Chemical and Biomolecular Engineering from Johns Hopkins University in the United States. Dr. Xiao has nearly a decade of experience in translational science, gene therapy product development, and biopharmaceutical manufacturing. She is dedicated to advancing the accessibility and applicability of gene therapies, with a focus on breakthroughs in gene therapy process technologies and technical operations management. Prior to joining Neurophth, she served as Deputy Director of GMP Manufacturing at Vigene Biosciences in the U.S., Senior Scientist for Gene Therapy Process Development and Scale-up at Pfizer, and Researcher at the Biotechnology Pilot Plant Platform of the U.S. National Institutes of Health (NIH). In these roles, she led or participated in process development, clinical drug manufacturing, and regulatory submissions for multiple rare disease gene therapy projects.

Mr. Cai Dabo, General Manager and Co-Founder, possesses extensive experience in China’s business landscape and the ophthalmology sector. He has substantial expertise and a proven track record in market promotion and marketing, key account management, government relations, data analytics, and commercial operations. During his more than 12 years at multinational corporations such as Pfizer and Novartis, he received the “President’s Award” three times. Mr. Cai played a pivotal role in establishing Solu-Medrol (methylprednisolone sodium succinate) as the top-selling product within Pfizer’s glucocorticoid portfolio, and contributed to making Lucentis the leading revenue-generating product in Novartis’ ophthalmology division for the treatment of wet (neovascular) age-related macular degeneration (AMD).

As LHON is a hereditary disease caused by mitochondrial gene mutations, gene therapy for LHON faces two major challenges: first, exogenous genes are delivered into target cells via recombinant AAV viral vectors, transcribed and translated into proteins, which are then guided into mitochondria through specific signal peptides; second, the eye is the most delicate part of the human body with intricate structures, making it critical to select an appropriate administration site to enhance therapeutic efficacy while minimizing treatment risks.

Neurophth utilizes adeno-associated virus (AAV) vectors and ectopic expression technology to deliver the correct ND4 protein into mitochondria, thereby restoring mitochondrial respiratory chain function and effectively treating Leber Hereditary Optic Neuropathy (LHON). Neurophth has also achieved technological innovation in the selection of administration routes. The global first marketed ophthalmic gene therapy drug, Luxturna, is administered via subretinal injection, targeting the retina at the innermost layer of the eyeball wall. In contrast, the gene therapy drug developed by Neurophth is administered via intravitreal injection. "Our drug for LHON is the world's first gene therapy administered via the intravitreal route," explained Professor Li Bin. "The intravitreal injection procedure takes only a few seconds, which significantly shortens the duration of ophthalmic surgery and enhances medication safety compared to subretinal administration." Building on these achievements, Neurophth has filed more than 20 domestic and international PCT invention patents.

There are more than ten companies worldwide engaged in the research and development of gene therapy drugs. Among them, three major entities are conducting research on gene therapies for Leber’s Hereditary Optic Neuropathy (LHON): GenSight, the Bascom Palmer Eye Institute at the University of Miami (BPEI), and Neurophth. It has been disclosed that Neurophth’s product demonstrates significantly higher efficacy than its international counterparts, with no restrictions related to specific stages of disease onset for administration.

Currently, Neurophth is building two R&D platforms: a gene therapy R&D platform for ophthalmic genetic diseases, and a GMP manufacturing platform for gene therapy drugs. Professor Li Bin revealed that the company plans to establish an Ophthalmic Gene Therapy Translational Center, which will serve as an R&D base for animal and cell experiments related to ophthalmic drug development; meanwhile, it will also develop other ophthalmic product lines. Neurophth’s core pipeline targeting LHON is currently in the preclinical research stage. With the successful completion of pilot-scale production, the company is preparing for regulatory filings for clinical trials both domestically and internationally. Meanwhile, Neurophth has established subsidiaries in the United States and Shanghai and is making preparations for the implementation of its core pipeline in the U.S.

In early 2020, the COVID-19 epidemic struck suddenly. As a Wuhan-based biotechnology enterprise, Neurophth promptly established an emergency response team. While ensuring the safety and protection of its employees, the company actively donated anti-epidemic supplies to support medical personnel on the front lines.

Currently, vaccines alone are insufficient to address the challenges faced by patients already infected. Moreover, studies indicate that SARS-CoV-2 is likely to persist long-term and evolve into a pattern of seasonal outbreaks. Leveraging its scientific research strengths, Neurophth has undertaken the development of gene therapy drugs for novel coronavirus infection and designed multiple therapeutic strategies. Building upon its fully established platforms for process development, pilot-scale production, quality assurance, and quality control, the company aims to contribute effectively to the fight against COVID-19, thereby demonstrating its corporate social responsibility and commitment.