Lingyi Biotech's AAV Gene Therapy LY-M001 for Gaucher Disease Receives IND Approval in China and Advances Toward Global Clinical Development

January 16, 2024Lingyi Biotech Co., Ltd.Lingyi Biotech's self-developed Class I therapeutic biological product, LY-M001 Injection, has successfully obtained the tacit approval from the National Medical Products Administration (NMPA) for its Investigational New Drug (IND) application (Acceptance No.: CXSL2300730).

LY-M001 Injection is China's first independently developed AAV gene therapy drug targeting Type I or Type III Gaucher 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. Lingyi Biotech has developed an improved glucocerebrosidase gene therapy vector with fully independent intellectual property rights, which enables long-term stable expression in vivo and degrades harmful glycolipid metabolites, thereby achieving the goal of long-term treatment for Gaucher disease. The IIT clinical research project led by Dean Huang He of the First Affiliated Hospital of Zhejiang University School of Medicine has officially started and completed the first patient dosing. Preliminary IIT data shows that LY-M001 demonstrates good performance in both efficacy and safety, with no adverse events reported.

Dr. Lin Qing, founder and CEO of Lingyi Biotech, stated: "We are very pleased that the IND application for our first innovative drug, LY-M001 Injection, has received tacit approval from the NMPA. This significant milestone affirms the outstanding innovation capabilities of our team from drug discovery to clinical development. The U.S. IND application for LY-M001 Injection was accepted by the FDA in December 2023. During the registration clinical stage, we will maintain close cooperation and communication with clinical trial institutions and regulatory authorities to accelerate the clinical research of LY-M001 Injection. We look forward to providing patients with a safer, more effective, and more accessible treatment option, and will continue to aim at addressing significant unmet clinical needs, making a positive contribution to the advancement of the pharmaceutical industry."

About Lingyi Biotech

In the field of rare diseases, the company boasts a world-class team in gene therapy discovery, research, development, and industrialization. It also possesses a globally leading platform for the creation and validation of rare disease animal models, addressing the challenge of the lack of effective animal models in rare disease research. The company is committed to the R&D and industrialization of FIC (First-in-Class) gene therapy pipelines, leveraging genes to create value and improve human health.

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