In the 8th batch of approved administrative licenses for the collection of human genetic resources in 2022, a total of 88 collection approval licenses and 134 international cooperative scientific research approval licenses were disclosed. In addition to the evaluation study of the clinical safety, tolerability and efficacy of GC101 injection for the treatment of type 1 SMA, there are three other rare disease-related clinical research projects involving Gaucher disease, hypertriglyceridemia and Duchenne muscular dystrophy.
About GC101 Injection and GeneCradle
GC101 Injection is a gene replacement therapy drug based on AAV viral vectors. Its structure and administration method are optimized for disease characteristics and biodistribution characteristics to achieve a safer and more effective purpose.
Beijing GeneCradle Technology Co., Ltd. is a biotechnology platform company focusing on the innovative research and development of gene therapy drugs. It focuses on the research and development of gene therapy products in the fields of hereditary neuromuscular diseases, genetic metabolic diseases, lysosomal diseases and ophthalmic diseases mediated by AAV vector delivery technology. With the mission of promoting China's rare disease gene drugs from basic to clinical and market, it is committed to turning "incurable" into "once and for all" cures, benefiting patients and families. Through the research and development and clinical application of rare disease gene drugs, we can have a deeper understanding of life and health, and transition to the treatment and rehabilitation of chronic diseases and other major diseases.