Jinlan Gene Initiates Phase I/II Clinical Trial Recruitment for GC301, a First-in-Class AAV Gene Therapy for Late-Onset Pompe Disease

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On the morning of June 17, the kick-off meeting for the "Single-Arm, Multi-Center, Open-Label, Intravenous Phase I/II Clinical Study on the Safety and Efficacy of GC301 Adeno-Associated Virus Injection for the Treatment of Late-Onset Pompe Disease" was successfully held at the First Medical Center of the Chinese People's Liberation Army General Hospital. This is the world’s first gene therapy drug for the treatment of late-onset Pompe disease to receive clinical trial approval, independently developed by Beijing Genecradle Therapeutics Co., Ltd. Director Yang Guang is leading the study as the principal investigator.

GC301 Injection is a gene therapy drug that uses recombinant AAV as a vector to carry the GAA gene expression sequence.Animal experimental results show that, through a single intravenous infusion, GAA can be widely distributed in the heart, skeletal muscles, liver, nervous system, and other areas, achieving the goal of long-term improvement in vitality levels, continuous clearance of accumulated glycogen, and restoration of muscle strength.

This drug has received the Clinical Trial Approval Notice from the Center for Drug Evaluation of the National Medical Products Administration.The project is recruiting subjects.。

About Genecradle

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. Its mission is 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 diseases, lysosomal diseases, and ophthalmic diseases. By promoting the research, development, and clinical application of rare disease gene drugs, the company aims 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.

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Source: Genecradle