On June 2, 2023, the GC301 adeno-associated virus injection, the world's first gene therapy drug independently developed by Beijing GeneCradle Technology Co., Ltd. (hereinafter referred to as "GeneCradle"), which has obtained clinical trial approval for the treatment of infantile Pompe disease, successfully held a Phase I/II clinical trial launch meeting at the Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, marking the official launch of the clinical trial of GC301 injection for the treatment of infantile Pompe disease in the main research center.

Group photo of the Phase I/II clinical study launch meeting of GC301 adeno-associated virus injectionGeneCradleGeneCradleGeneCradle Professor Qiu Zhengqing, chief physician of pediatrics at the Chinese Academy of Medical Sciences and Peking Union Medical College Hospital, Professor Han Xiaohong, principal investigator of the Clinical Pharmacology Center of the Chinese Academy of Medical Sciences and Peking Union Medical College Hospital, Dr. Dong Xiaoyan, co-founder of Beijing GeneCradle Technology Co., Ltd., Professor Zhu Zhiming, vice president of clinical operations, Dr. Wang Xiaodong, vice president of the Medical Affairs Department, and heads of clinical trial collaborators and other experts were invited to attend the project launch meeting.

Professor Qiu Zhengqing, Chief Physician and Principal Investigator of Pediatrics Department of Peking Union Medical College Hospital, Chinese Academy of Medical Sciences

Professor Qiu Zhengqing, chief physician of the Department of Pediatrics at the Peking Union Medical College Hospital, Chinese Academy of Medical Sciences and the main researcher of this clinical trial project, expressed at the meeting that he would like to thank the sponsor and all the collaborating units for their work in the early preparation of this project, which promoted the smooth launch of the clinical trial of GC301 injection for the treatment of infantile Pompe disease in this hospital. He also wished that this clinical project would be successfully completed with the cooperation and efforts of the hospital and all the collaborating parties.

Professor Han Xiaohong, Clinical Pharmacology Center, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Principal Investigator

Professor Han Xiaohong, head of the Clinical Pharmacology Center of Peking Union Medical College Hospital and the main researcher of clinical testing of this clinical trial, said that he was very happy to see the successful launch of the project in the hospital. This is not only a double improvement of the research team and clinical team's capabilities, but also a promotion of the exploration and practice of gene therapy in China. GeneCradle GeneCradle At the meeting, representatives of all parties and relevant clinical research leaders jointly gave a detailed explanation of the project plan. The participants had a full and heated discussion on the details of the clinical trial project execution, and expressed that they would fully cooperate with each other during the execution process to accelerate the clinical evaluation process of GC301 injection, a gene therapy drug for infantile Pompe disease. Dr. Dong Xiaoyan, co-founder of GeneCradle, said that the conduct of this clinical trial carries the trust and expectations of many parties. I believe that through our joint efforts, GC301 injection will definitely benefit more patients. GeneCradleGeneCradleGeneCradleGeneCradleThis clinical launch meeting marks the official launch of the world's first clinical trial of gene therapy for infantile Pompe disease in China. As the world's first long-acting treatment for Pompe disease, GC301 injection is expected to bring better treatment methods and hope to the infantile Pompe disease population. GeneCradle Subject RecruitmentGeneCradle

The single-arm, multi-center, open, intravenous Phase I/II clinical trial evaluating the safety and efficacy of GC301 adeno-associated virus injection in the treatment of infantile Pompe disease patients has officially started recruiting subjects. Please scan the QR code below to obtain more information about the enrollment.

About GC301 Adeno-associated Virus Injection

GeneCradle GeneCradle GeneCradleGC301 injection is an AAV gene therapy drug designed and developed by GeneCradle for the treatment of Pompe disease. It adopts a one-time intravenous injection strategy for widespread systemic expression in order to directly compensate for the GAA enzyme gene defects in tissues such as the liver, myocardium, skeletal muscle, and central nervous system. This drug has obtained two implicit clinical trial licenses from the Center for Drug Evaluation of the State Drug Administration, and has been approved by the Drug Clinical Trial Ethics Committee of the Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, and has been registered and publicized on the Drug Clinical Trial Registration and Information Disclosure Platform.

About GeneCradle

GeneCradle GeneCradle GeneCradle Beijing GeneCradle Technology Co., Ltd. is a national high-tech enterprise with the development of gene therapy drugs mediated by AAV vector delivery technology as its core business. Its mission is to promote China's rare disease gene drugs from basic to clinical and market, benefiting patients and families. The company focuses on the development of gene therapy drugs in the fields of hereditary neuromuscular diseases, genetic metabolic diseases, lysosomal diseases and ophthalmic diseases. By promoting the research and development and clinical application of gene drugs for rare diseases, it has a deeper understanding of life and health, and transitioned gene therapy technology and products from rare diseases to the treatment and rehabilitation of chronic diseases and other major diseases.