Lingyi Biotechnology Co., Ltd, a China-based gene therapy developer, has secured clinical trial approval from the US Food and Drug Administration (FDA) for its proprietary AAV gene therapy candidate, LY-M001, which is being developed to treat type I or III Gaucher’s disease.
LY-M001 utilizes a recombinant adeno-associated virus (rAAV) as a vector, and following a single intravenous infusion, it enables the expression of the patient’s required glucose brain glucoside lipase. Lingyi Biotechnology boasts a modified gene therapy vector platform with fully independent intellectual property rights. This platform is capable of stably expressing and degrading harmful glucose and lipid metabolites in the body over an extended period, thereby aiming to achieve long-term treatment outcomes for Gaucher’s disease.- Flcube.com
