The World's First Clinical Trial of Gene Therapy for Infant Pompeii Disease has Officially Started at Peking Union Medical College Hospital

Recently, as a clinical contract research organization, Beijing Leadingpharm-Deep Blue Ocean (Zhengzhou) and Beijing Jinlan Gene Technology Co., Ltd. jointly launched the phase I/Ⅱ clinical trial of the gene therapy drug GC301 adeno-associated virus injection for infantile Pompeii disease at Peking Union Medical College Hospital, Chinese Academy of Medical Sciences. GC301 injection, as the world's first long-acting therapeutic drug for Pompeii disease, is expected to bring better treatment methods and hope to the population of infantile Pompeii disease.

GC301 injection is an AAV gene therapy drug designed and developed by Jinlan Gene for the treatment of Pompeii disease. It adopts a strategy of one-time intravenous injection with widespread systemic expression, aiming to directly compensate for GAA enzyme gene defects in tissues such as liver, myocardium, skeletal muscle, and central nervous system. This drug has obtained two implied clinical trial licenses from CDE, and has been approved by the Drug Clinical Trial Ethics Committee of Peking Union Medical College Hospital of the Chinese Academy of Medical Sciences. It has been registered and published on the drug clinical trial registration and information disclosure platform, and is now recruiting participants from the society.

Deep Blue Ocean Biopharmaceutical is a sub brand of Leadingpharm in the field of clinical CRO, established in 2007. Currently, it has a highly professional clinical service team of over 600 people, providing diversified research and development support and clinical technical services to more than 300 well-known domestic and foreign enterprises. The project comprehensively covers drugs such as biopharmaceuticals, chemical drugs, traditional Chinese medicine, medical devices, cell and gene therapy, meeting the clinical research customization needs of different R&D enterprises, and opening up key bottlenecks for domestic and foreign customers before the launch of new drugs. Through 16 years of experience accumulation, Deep Blue Ocean Biomedical has completed more than 500 clinical research projects and conducts 10-20 Class I innovative drug research projects annually. It has taken the lead in conducting research in fields such as medical aesthetics, specialty food, cell and gene therapy drugs, filling multiple gaps at home and abroad.
 

Relying on the Leadingpharm CXO service system, Deep Blue Ocean has created an "interactive" clinical service model, fully involved in the project initiation stage, becoming a full partner in new drug creation, integrating medical, pharmaceutical, pharmaceutical research and development and other technical experts for top-level design, and minimizing project research and development risks. At the same time, establish a special government guidance fund to provide investment and financing at different stages for innovative projects, and accelerate the process of drug listing to the greatest extent possible. In addition, a strong team of quantitative pharmacology and in vitro and in vivo bridging teams conduct digital model research for specific diseases and populations, develop scientifically high-quality clinical research plans, and improve the efficiency of new drug clinical development.