Xingmou Biotech Files IPO Prospectus: Developing China's Own Gene Therapies for Millions of AMD Patients

With the deepening of population aging and the widespread use of electronic products, the incidence of eye diseases such as dry eye, glaucoma, cataracts, and age-related macular degeneration is also increasing.

Among ophthalmic diseases, age-related macular degeneration (AMD) is one of the retinal diseases with the highest incidence. AMD is closely related to age and is one of the leading causes of blindness in people aged 50 and above, making it one of the four major blinding eye diseases.

Data shows that the prevalence of AMD in people over 50 can reach up to 15.5% in some regions, and the prevalence rate is as high as 21% in those over 70, with the risk of disease significantly increasing with age. In 2020, there were nearly 200 million AMD patients globally, and about 37 million AMD patients in China.

According toStarryGene CEO Dr. Cai YuanIntroduction: AMD is divided into dry and wet types, both of which can cause blindness. Wet age-related macular degeneration (wAMD) is the most devastating type. The wAMD population accounts for 10% to 15% of the total macular degeneration population, and 80%-90% of dry AMD will also transform into the wet type. In addition to age, screen flicker and LED blue light are also high-risk inducements for AMD. As the proportion of people using electronic products continues to rise, the wAMD population is expected to expand further in the future.

StarryGene (hereinafter referred to as: StarryGene) was established in August 2019 and is located in Hefei City, Anhui Province, China.Focusing on the development of gene therapy drugs and gene editing technology research and services for ophthalmic diseases, committed to developing gene therapy drugs for ophthalmic use that are independently accessible by Chinese people., allowing the Chinese people to see longer and better.

StarryGeneThe first-mover product has deeply cultivated the wAMD track,It has currently achieved significantly superior in vivo and in vitro efficacy compared to RGX-314, a leading product from the United States.Combined with its own new capsid, StarryGene has achieved the vitreous cavity injection procedure in this field., which will provide convenience for clinical use in the future and benefit tens of millions of AMD patient population.

When asked why he focused on ophthalmic diseases, Dr. Cai Yuan said,First, gene therapy in the field of ophthalmology has been clinically validated.In 2017, Luxturna, the first gene therapy for the treatment of inherited retinal dystrophy, was approved by the FDA, targeting IRDs caused by RPE65 biallelic mutations. Subsequently, more gene therapies entered clinical trials, ushering in a new era for the treatment of genetic eye diseases, marking a very important启蒙-style milestone for China's gene therapy sector.

Secondly, gene therapy for ophthalmic diseases has its unique advantages., firstly, safety: the eyeball, as an independent and enclosed sphere, ensures safety through its local injectability and replaceability compared to systemic or central nervous system medications; secondly, ophthalmic drugs have small dosages and lower costs, which are beneficial for industrialization and further enhance safety.

More importantly, originating fromStarryGene's core team has continuous attention and profound accumulation in the field of regenerative vision repair for ophthalmology, including gene therapy, stem cell therapy, and other related areas.

According to Dr. Cai Yuan, the founding team of StarryGene consists of senior researchers, professors, and doctors in the field of life sciences, with many years of in-depth research and management experience in ophthalmology, gene editing, and gene therapy. Their related research work has been published in top academic journals such as Nature, Cell, Science Advances, and Nature Communications.

Dr. Zilong Qiu, Chairman of StarryGene, is the Deputy Director of the State Key Laboratory of Neuroscience at the Center for Excellence in Brain Science and Intelligence Technology of the Chinese Academy of Sciences, the Head of the Molecular Basis of Neural Plasticity Research Group, and a Senior Researcher.

Dr. Zilong Qiu has long been engaged in biological research on neurodevelopmental disorders such as autism and Rett syndrome. His research achievements have elucidated the genetic, molecular, and neural circuit mechanisms of neurodevelopmental disorders. He established a non-human primate model of autism, which was successfully selected as one of the "Top Ten Scientific Advances in China" by the Ministry of Science and Technology in 2016 and one of the "Top Ten Advances in Life Sciences in China" by the China Association for Science and Technology in 2016.

Dr. Cai Yuan, CEO of StarryGene, is a postdoctoral fellow at the First Affiliated Hospital of the University of Science and Technology of China (Anhui Provincial Hospital). His long-term research focuses on the development of gene therapy technologies for hereditary eye diseases. During his entrepreneurial journey, he has continuously paid attention to gene therapies for retinitis pigmentosa, age-related macular degeneration, glaucoma, and myopia.

In addition, Professor Xue Tian serves as the company's scientific advisor. Professor Xue Tian is the Executive Minister of the School of Life Sciences and Medicine at the University of Science and Technology of China, a professor and doctoral supervisor at the School of Life Sciences, and the Director of the "Neural Circuits and Brain Cognition Division" at the National Laboratory for Microscale Material Science.

Professor Tian Xue has long been engaged in research on phototransduction, neural circuits, and visual regeneration and repair in the field of photoreceptive neurobiology. He has published 44 SCI papers to date, with several corresponding author papers appearing in academic journals such as Nature and Cell. His work has been cited over 4,000 times, with an h-index of 27.

Currently, StarryGene has built a research and development team of 15 people. VCBeat serves as the financial advisor, helping to organize and optimize the company’s financial investments, industrial allocation, and development strategies, assisting the team in quickly finding suitable investors before changes occur in the capital market.

In February this year, StarryGene secured Pre-A round funding led by Guoke Ruihua, with co-investments from Beijing Phase II Zhongke Chuangxing Hard Technology Venture Capital Partnership (Limited Partnership) and Zhuhai Hengqin Yixierding Investment Partnership (Limited Partnership), to accelerate the advancement of its core wAMD pipeline product and the development of new pipelines.

Dr. Wang Yueyue, Managing Partner of Tianchao Capital, stated: "We are very optimistic about the application of gene therapy in fields such as ophthalmology and neurology. The founding team of StarryGene has deep expertise in visual science, neuroscience, as well as gene editing technology and platforms, possessing international-level and extensive research experience, theoretical foundations, and clinical resources. Their product pipeline is internationally competitive and surpasses overseas clinical benchmark products."

StarryGene is actively promoting gene therapy into major ophthalmic indications, currently possessing multiple products under research, among whichThe leading pipeline with the fastest progress focuses on wAMD, currently in the IIT clinical trial and IND application stage, and is expected to enter in-hospital clinical trials in May.

Through continuous research and development, StarryGene has achieved animal efficacy for multiple candidate AAV drugs in its wAMD pipeline that surpasses leading international Phase II clinical drugs.

In addition, StarryGene has also cooperated with Shanghai Weimei Gene to develop a new capsid,Cooperate with domestically produced devices for drug delivery, simplifying surgical procedures, avoiding the high risks and equipment shortages associated with subretinal injection delivery,Achieved the same intravitreal injection as traditional antibody drugs, which will effectively improve the compliance of both patients and doctors.

In addition to the fastest-progressing wAMD pipeline, StarryGene also has a presence in glaucoma, thyroid eye disease, non-glaucomatous blindness, and dry AMD.

Dr. Cai Yuan stated that gene editing can address many issues that existing drug systems cannot solve, such as the unmet clinical needs of a large number of patients with rare diseases. However, the development of gene editing also has its limitations. First, human understanding of gene editing is still very limited, and a series of potential genetic risks may affect the rapid progress and market entry of related products. Second, while gene editing can meet unmet clinical needs, the small market size for rare diseases can also influence corporate decision-making and strategic planning.

Based on this consideration, StarryGene, in addition to gene editing technology, alsoLayout of Major Ophthalmic Indications: Aiming to Meet the Clinical Needs of a Wider Population with the Product.

"At this stage of development, the company needs more talented people to join. 'We have done well in front-end R&D. However, in areas such as drug registration and clinical management, in addition to collaborating with high-quality teams, we want to build our own backend capabilities. We are also making efforts in this area, including attracting top talents in the field, partnering with experienced CDMOs, and continuously recruiting and nurturing talent.'"

"Since more biomedicine professionals gather in Suzhou and Shanghai, we have established a branch in Shanghai to promote clinical work within the hospital and attract high-level biomedicine professionals from Shanghai. We have also secured a leading project in Suzhou Industrial Park to attract top biomedicine professionals from Suzhou," said Dr. Cai Yuan.

Focusing on the present, StarryGene will further advance the clinical development and IND submission of its core products within hospitals, while accelerating the R&D of subsequent products to create a broader range of solutions. Looking to the future, StarryGene aims to launch 3 to 5 gene therapy drugs for major ophthalmic indications within 5 to 8 years, truly benefiting more patients with eye diseases.