China’s First Gene Therapy Drug for Gaucher Disease Type I Approved! Congratulations to Lingyi Biotech on the Implied IND Approval for LY-M001

On January 16, 2024, Lingyi Biotech (Hangzhou) Co., Ltd. (“Lingyi Biotech”) announced that the Investigational New Drug (IND) application for LY-M001 Injection, its independently developed Class I therapeutic biological product, successfully received implied approval from the National Medical Products Administration (NMPA) (acceptance number: CXSL2300730). PackGene warmly congratulates its strategic partner Lingyi Biotech on this important progress.

LY-M001 Injection is China’s first independently developed AAV gene therapy drug targeting Gaucher disease type I or type III. The product uses recombinant adeno-associated virus (rAAV) as the vector and enables expression of the glucocerebrosidase required by patients after a single intravenous infusion. Lingyi Biotech has developed an improved glucocerebrosidase gene therapy vector with fully independent intellectual property rights, which can achieve long-term stable expression in vivo and degrade harmful glycolipid metabolites, thereby aiming to provide long-term treatment for Gaucher disease.

An investigator-initiated trial (IIT) led by President Huang He of the First Affiliated Hospital, Zhejiang University School of Medicine has also been officially launched, with the first patient already dosed. Preliminary IIT data show that LY-M001 has demonstrated good efficacy and safety, with no adverse events observed.

Dr. Qing Lin, Founder and Chief Executive Officer of Lingyi Biotech, stated, “We are very pleased that the IND application for our first innovative drug, LY-M001 Injection, has received implied approval from the NMPA. This important milestone recognizes our team’s outstanding innovation capabilities from drug discovery to clinical development. The U.S. IND application for LY-M001 Injection was accepted by the U.S. Food and Drug Administration (FDA) in December 2023. During the registrational clinical stage, we will maintain close collaboration 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 contribute actively to the development of the pharmaceutical industry with the goal of addressing major unmet clinical needs.”

PackGene is honored to support the advancement of Lingyi Biotech’s AAV gene therapy drug by providing high-quality CMC services for this project. Looking ahead, PackGene will continue to deepen its commitment to the field of gene therapy, providing one-stop CMC solutions for drug innovation and helping advance more high-quality projects.

About Lingyi Biotech

Founded in February 2021, Lingyi Biotech is a globally leading company focused on the development of therapeutics for monogenic genetic diseases. Committed to original innovation, the company focuses on disease areas with urgent clinical needs, including metabolic disorders, central nervous system diseases, and ophthalmic diseases. Starting from key technologies such as animal models of genetic diseases, Lingyi Biotech aims to provide one-time solutions for patients.

In the field of rare diseases, the company has a world-class team for gene therapy discovery, research and development, and industrialization, as well as a globally leading platform for the generation and validation of rare disease animal models. By addressing the industry challenge of lacking effective animal models for rare disease R&D, Lingyi Biotech is committed to the development and industrialization of first-in-class gene therapy pipelines, creating value through genes and improving human health.