Dingxin Gene: Advancing Innovative Therapeutics through Precision Gene Delivery

Gene therapy is the next big trend.

RRGENER Founder Zhou Rui Graduated from Shenyang Pharmaceutical University. In His First Decade in the Industry, He Gained Extensive Experience in the Development and Management of Biopharmaceutical Enterprises. In the Following Years, He Reinvented Himself by Transitioning to Pharmaceutical Investment. As the Investment Director of Huitianfu, One of China’s Top Funds, He Established the First Secondary Market Pharmaceutical Industry Thematic Fund in China and Managed a Leading Public Pharmaceutical Investment Fund in China, Exerting Wide Market Influence. With a Multifaceted Background, Zhou Rui Pays More Attention to the Overall Development Trends of the Pharmaceutical Industry.He is optimistic about the development of the gene therapy field and industry-disruptive opportunities, and firmly believes that delivery technology is the cornerstone of the gene therapy industry's growth., while the nature of pharmaceutical professionals led him to ultimately seek his original aspiration, deciding to personally step into the industry and return to the real economy by establishing an innovative drug company based on precise gene delivery.

In the summer of 2020, Rui Zhou and Dr. Qingqiao Tan hit it off and founded RRGENER. As the CTO, Dr. Qingqiao Tan has nearly 20 years of experience in biopharmaceutical R&D and has participated in the development of 15 biologics, three of which have been launched. In an interview, Dr. Tan stated, "Currently, traditional chemical drugs and biologics are powerless against certain diseases, especially rare diseases. We want to try to address these clinical pain points through alternative approaches."Gene therapy offers new opportunities to cure diseases.”

Group photo of RRGENER founding team (From left to right: General Manager Liu Xinxing, Founder and Chairman Zhou Rui, CTO Tan Qingqiao)

At the same time, Dr. Xinxing Liu, who has rich operational management experience in the gene therapy field, decided to join the company as the general manager. Dr. Liu participated in the incubation of a gene therapy company led by top scientists in China and led the clinical transformation of a system-administered AAV gene therapy project. In an interview, Dr. Liu stated, "As a new industry,Whether in terms of the R&D system, regulatory science, or clinical commercialization, gene therapy companies in China are still in the enlightenment stage. To stand out, they need to excel in execution, industrial transformation, and industry resource integration—qualities that RRGENER possesses.。”

RRGENER insistsBegin with the end in mindThe innovative development philosophy and the good reputation of its founder, Rui Zhou, have attracted multiple partners with industry leadership experience and young talents to join. RRGENER has also received strong support from many frontline clinical experts, professors, friends, and investors. In less than a year and a half since its establishment, the company has completed two rounds of financing, raising nearly 200 million RMB, and gained recognition from well-known investment institutions and industry investors.Final confirmation of three preclinical candidate molecules is about to be completed, laying a solid foundation for the next stage of development.。

AAV virus is the most commonly used delivery tool in gene therapy. Due to its excellent safety, different tissue tropism, low immunogenicity risk, and the ability to stably express exogenous genes in the body over the long term, it is widely recognized as the most promising gene therapy vector. AAV virus is a non-enveloped virus about 26nm in size, with an icosahedral capsid structure, belonging to the Dependovirus genus of the Parvoviridae family.

RRGENER has made multi-dimensional upgrades and optimizations based on traditional AAV viral vectors, focusing on the ultimate treatment scenarios. Dr. Tan Qingqiao introduced that RRGENER has carried out the most refined modifications at the foundational level of AAV vector design, creating a high-throughput screening and iterative technology platform for rAAV libraries to enhance tissue tropism, transfection efficiency, expression durability, and safety. This AAV vector platform can continuously deliver engineered serotypes, meeting the precise delivery requirements of different candidate molecules, and can achieve immune escape to meet the need for repeated dosing.

"After our modification of the AAV viral vector, the virus transduction efficiency in certain specific target cells can be increased more than 10 times," said Dr. Tan Qingqiao. "Every step of our modification and optimization is aimed at better treating diseases, a technology upgrade guided by therapeutic scenarios to improve the accessibility of gene therapy drugs."

RRGENER has always emphasized the treatment scenarios, which the company selected at its inception — "clear hearing and sharp eyesight" — the fields of otology and ophthalmology. "In terms of indications, we have chosen commercially promising areas — the otology and ophthalmology tracks — but in terms of technology, we are not path-dependent," Dr. Tan Qingqiao told VCBeat. "Both ophthalmology and otology involve localized drug delivery with a natural immune barrier, smaller drug dosages, higher safety, and effects that can be 'immediately visible — clear hearing and sharp eyesight.'"

Specifically, RRGENER focuses on ophthalmic and otologic indications, targeting mature treatment options for chronic ophthalmic diseases and congenital deafness. The company's AAV gene therapy uses ophthalmology and otology as entry points, establishing a well-formed AAV gene therapy research and development and technical system. It has basically completed the preclinical candidate molecule confirmation for its first chronic eye disease and first congenital deafness R&D pipelines, entering the forefront of related product R&D in China. The company is expanding the clinical translation of novel AAVs and broadening the scope of therapeutic applications.

RRGENER's first business layout in the gene therapy track focuses on ophthalmology and otology. RRGENER's second major technology platform is the Non-virus RNA Gene Therapy Platform, developed based on a non-viral vector drug delivery system. Dr. Liu introduced that this technology platform primarily uses small interfering ribonucleic acid (siRNA) to regulate and suppress the expression of target gene proteins to achieve the purpose of treating specific diseases. The company has established a nucleic acid drug research and development and technology system mainly focused on siRNA drug development.

Based on this therapeutic mechanism, the company has leveraged the GalNAc liver-targeted delivery system as an entry point to develop GalNAc liver-targeted delivery treatments for chronic metabolic diseases. It has currently completed the preclinical candidate molecule confirmation for its first R&D pipeline and is expanding the clinical translation of extrahepatic delivery technologies while broadening the scope of therapeutic applications.

At the same time, based on the non-viral vector drug delivery system, RRGENER has also begun to seek cooperation opportunities for nanoparticle targeted delivery systems. The company is also actively selecting suitable nano-delivery systems according to pipeline characteristics and clinical needs.

"A worker must first sharpen his tools if he is to do his job well." RRGENER starts with the underlying delivery technology platform for gene therapy, using AAV viral vectors and non-viral vectors to precisely address clinical treatment pain points of rare diseases and chronic conditions across different fields such as ophthalmology, otology, cardiovascular, oncology, and the central nervous system (CNS), focusing on developing affordable and effective gene therapies and specialty drugs for patients.

Currently, RRGENER has built and launched a nearly 1,000-square-meter gene therapy R&D center in Shanghai, preliminarily establishing an early R&D team and platform. Within a year of its establishment, RRGENER has completed two rounds of strategic financing. The company will also initiate a new round of financing soon to advance and expand its clinical pipeline as well as strengthen talent team development.RRGENER always values its talent team, widely recruits aspiring individuals, and builds a gene therapy ecosystem to boost the development of the entire industry.。

As the founder of RRGENER, Zhou Rui has been leading the founding team to practice the values of "Innovation, Passion, Integrity, Collaboration, Joy, and Gratitude," hoping to attract more talented individuals to join and lead the company's growth together, allowing humanity to be free from illness and enabling life to shine brilliantly!