Lingyi Biotech Announces IND Application Acceptance for Its First Gene Therapy Drug LY-M001 Targeting Gaucher Disease

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LY-M001 Injection is the first self-developed AAV gene therapy drug in China targeting Type I and Type III Gaucher's disease.This product uses recombinant adeno-associated virus (rAAV) as a vector, and after a single intravenous infusion, it can express the glucocerebrosidase required by patients. The company has developed an improved gene therapy vector for glucocerebrosidase with independent intellectual property rights, which can achieve long-term stable expression in the body and degrade harmful glycolipid metabolites, thereby achieving the goal of long-term treatment of Gaucher's disease.

LY-M001 Injection Receives FDA Orphan Drug Designation in the U.S. The IIT Clinical Research Project Led by Dean Huang He of the First Affiliated Hospital of Zhejiang University School of Medicine Has Officially Launched.