Recently, the Beijing Key Laboratory of Precision Diagnosis and Innovative Translational Research for Pediatric Rare Diseases (hereinafter referred to as "the Laboratory"), co-established with the participation of Genecradle Therapeutics, has been officially approved. Supported by Beijing Children's Hospital, Capital Medical University, the Laboratory is jointly built by Beijing Genecradle Therapeutics Co., Ltd., Peking University People's Hospital and the Institute of Biophysics, Chinese Academy of Sciences. Focusing on precision diagnosis and innovative translational research for pediatric rare diseases, the Laboratory aims to build a collaborative system of "clinical demand-guided basic research breakthroughs, technology and product R&D, and industrial translation of achievements", so as to bridge the critical path from bench to bedside.

Director of the Laboratory

Hao Chanjuan

Deputy Director of the Laboratory

Wu Xiaobing

Deputy Director of the Laboratory

Wang Shidong

Deputy Director of the Laboratory

Zhou Haining

Pediatric rare diseases feature a vast variety of subtypes, high diagnostic and therapeutic challenges, and pressing unmet clinical needs, making them a key research priority in the global medical community. As a leading enterprise in China's gene therapy sector, Genecradle Therapeutics has independently developed technical platforms and systems covering drug R&D, manufacturing and clinical development, and continuously broken through core technical barriers. Its self-developed pipeline covers multiple high-incidence pediatric rare diseases including spinal muscular atrophy (SMA), Pompe disease and Wilson's disease. Multiple product candidates have obtained China's Breakthrough Therapy Designation and FDA Orphan Drug Designation, with clinical progress ranking among the forefront in China.

As the representative industrialization partner among the co-founding entities, Genecradle Therapeutics will give full play to its systematic strengths in gene drug R&D, pharmaceutical research and industrial practice. It will deeply integrate its capabilities with the clinical resources and diagnostic expertise of Beijing Children's Hospital and Peking University People's Hospital, as well as the basic research strength of the Institute of Biophysics, CAS, to build an integrated collaborative innovation system spanning from basic research to technological innovation, and from clinical application to industrial translation.