
Novel Gene Therapy Drug Developer
Recently, NEUREGEN announced the completion of its angel round of financing worth tens of millions of RMB. This round of financing was led by the JSG United Runpu Medical Fund and will be mainly used for team building, technology platform construction, and the early-stage development of novel gene therapy drugs.
NEUREGEN was founded in December 2021 as a biotechnology company aimed at developing novel gene therapy drugs by leveraging its proprietary cell transdifferentiation technology and genetic substitution technology. Established by a closely-knit team of neurobiologists, ophthalmology experts, gene therapy specialists, and veterans from the biopharmaceutical industry, the company is committed to transforming emerging cell transdifferentiation technologies into solutions addressing unmet clinical needs. Currently, NEUREGEN has built a novel gene therapy drug development platform centered on cell transdifferentiation technology, encompassing key technologies such as transdifferentiation factor screening, viral and non-viral vector design, and in vivo and in vitro pharmacodynamic method development, with several critical patents already filed. The company’s product pipeline focuses on neurological diseases, prioritizing the development of proprietary, first-in-class gene therapies for blinding eye conditions and difficult-to-treat nervous system tumors.
Diseases of the central nervous system are currently a leading cause of disability worldwide and the second leading cause of death globally. As China continues to move into an era of population aging, the number of patients with neurological diseases in China is also growing. Over the past 30 years, the number of patients who died from central nervous system diseases has increased by 39%. The social and economic losses caused by central nervous system diseases are enormous, often accounting for more than 50% of the total healthcare and nursing expenditures in society.
In the field of neural regeneration, current gene therapies are unable to effectively reverse the progression and deterioration of damage caused by the central nervous system. The key lies in solving the problem of neuronal regeneration. Over the past few decades, solutions to this issue have focused on embryonic stem cell transplantation, but this approach faces risks of potential immune rejection as well as high costs. Therefore, in recent years, scientists have shifted their focus to in-situ transdifferentiation technology, which not only opens up a new path in the field of neural regeneration but also has garnered continuous attention within the industry due to its many advantages, such as avoiding immune rejection, reducing ethical risks, and relatively low cost.
For example, the in-situ regeneration technology of the central nervous system utilizes the characteristic of endogenous glial cells to self-divide and regenerate. By expressing neural transcription factors or other methods, glial cells are converted in situ into functional neurons.

Figure: Neuronal In-situ Transdifferentiation
Currently, the in-situ transdifferentiation technology is still in the early stage of development. Companies focusing on gene therapy with in-situ transdifferentiation technology as the core are scarce. There is a huge unmet clinical need in the field of neurological diseases. The unique technology of NEUREGEN has the potential to become a platform technology for treating various neurological diseases, providing patients with breakthrough new therapies and bringing new hope for treatment.
Dr. Yueguang Liu, Co-founder and Chief Scientific Officer of the company, stated: "We are honored to gain recognition from outstanding investors. NEUREGEN's proprietary core technology of in-situ cell transdifferentiation can now achieve the delivery and expression of transdifferentiation factors through various vector technologies, converting different types of glial cells into functional neurons in situ, or transdifferentiating malignant glioma cells into non-dividing 'harmless' cells. This is a completely groundbreaking technology currently being used to develop multiple original gene therapy drugs for treating various blinding eye diseases, nervous system tumors, and neurodegenerative diseases, benefiting a wide range of patients."
Dr. Wang Xiaoyan, partner of Jinsha River United Runpu Medical Fund, stated: Traditional drug and surgical therapies have significant limitations for many diseases, especially neurological disorders. Novel gene therapies based on in-situ cellular transdifferentiation technology are a valuable complement to conventional treatments and bring new hope to this field. We are pleased to have the opportunity to collaborate with the NEUREGEN team, and we hope that the company’s platform technology will soon provide patients with safer and more effective treatment options.
Runpu Healthcare Fund of Jinsha River Venture Capital is the first healthcare-focused fund initiated by Jinsha River United Capital in 2021. The team has over 10 years of investment experience and has led investments in nearly 50 healthcare projects, possessing extensive industry resources. The fund primarily focuses on pharmaceuticals, IVD, and biotechnology, targeting both early-stage and growth-stage high-quality projects. Currently, Runpu Healthcare Fund has invested in outstanding companies such as Ruijian Pharmaceuticals, JOINN Biologics, Changyu Pharmaceuticals, and Bowang Pharmaceuticals.