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On July 25, 2025, the School of Pharmaceutical Sciences at Peking University and Beijing Genecradle Therapeutics Co., Ltd. signed a collaborative research agreement at the National Key Laboratory of Natural and Biomimetic Drugs in China.Focusing on tRNA Gene Therapy Drug Development, Paving New Avenues for Rare Disease Treatment.This collaboration was witnessed by Professor De-Min Zhou, Director of the National Key Laboratory of Natural and Biomimetic Drugs and Dean of the Ningbo Institute of Oceanic Drug, Peking University, Professor Qing Xia from the School of Pharmaceutical Sciences, along with faculty and student representatives, Professor Xiao-Bing Wu, founder of Genecradle, and Wen-Hao Ma, head of the R&D Center at Genecradle. It marks the deep integration of Genecradle's gene therapy industrial technology capabilities in the industry with the research strengths of the School of Pharmaceutical Sciences at Peking University in the field of nucleic acid drugs.At the signing ceremony, Director Demin Zhou systematically reviewed the research accumulation of Peking University School of Pharmaceutical Sciences in the field of gene therapy and proposed the cooperation goal of developing a new generation of tRNA drugs through a joint research model. Professor Qing Xia's team presented their research achievements on tRNA drugs in the treatment of four rare diseases and conducted in-depth discussions on key issues of clinical transformation such as patient selection and production quality control. Professor Xiaobing Wu shared her experience in the development of gene therapy drugs and explained the core advantages of adeno-associated virus (AAV) vectors as an ideal delivery tool for gene therapy. She stated that Genecradle would leverage its AAV vector R&D strengths to help Peking University’s tRNA drugs make the leap from basic research to clinical application.This collaboration integrates Peking University's academic accumulation in tRNA drug development with Genecradle's technological achievements in the AAV vector field, focusing on innovative drug development for rare diseases. Currently, Genecradle has established several self-developed gene therapy drug pipelines and advanced them to Phase I/II/III clinical trials. Significant progress has been made in multiple projects targeting major rare diseases such as Spinal Muscular Atrophy (SMA) and Pompe disease, demonstrating strong translational potential. Through an industry-academia collaborative mechanism, both parties will accelerate the development of tRNA gene drugs, bringing new hope to more patients.
Beijing Genecradle Therapeutics Co., Ltd. is a national high-tech enterprise with core business in the development of gene therapy drugs mediated by AAV vector delivery technology. It aims to advance China's rare disease gene drugs from basic research to clinical and market applications, benefiting patients and their families. The company focuses on the development of gene therapy drugs for hereditary neuromuscular diseases, inherited metabolic disorders, lysosomal diseases, and ophthalmic diseases. By promoting the research, development, and clinical application of rare disease gene drugs, it seeks to deepen the understanding of life and health, transitioning gene therapy technologies and products from rare diseases to the treatment and rehabilitation of chronic and other major diseases.-END-
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