Lingyi Biotech’s LY-M001, China’s First Gene Therapy Drug for Type 1 Gaucher Disease, Receives NMPA Implied Approval for IND

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

LY-M001 Injection is China’s first independently developed AAV gene therapy drug targeting Type I or Type III Gaucher disease. 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 optimized 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 supporting the goal of long-term treatment for Gaucher disease.

An investigator-initiated clinical study (IIT) led by President Huang He of The First Affiliated Hospital, Zhejiang University School of Medicine has also officially commenced and completed dosing of the first patient. Preliminary IIT data showed that LY-M001 demonstrated favorable efficacy and safety, with no adverse events reported.

Dr. Lin Qing, Founder and Chief Executive Officer of Lingyi Biotech, said, “We are very pleased that the IND application for our first innovative drug, LY-M001 Injection, has received NMPA implied approval. 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 development of LY-M001 Injection. We look forward to providing patients with a safer, more effective and more accessible treatment option, and will continue to address major unmet clinical needs while making positive contributions to the advancement of medicine.”

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

About Lingyi Biotech

Founded in February 2021, Lingyi Biotech is a globally leading company in the development of drugs for monogenic genetic diseases. The company is committed to original innovation and focuses on areas of urgent clinical need, including metabolic diseases, central nervous system disorders 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 rare disease animal model generation and validation platform. By addressing the lack of effective animal models in rare disease R&D, Lingyi Biotech is dedicated to the development and industrialization of first-in-class gene therapy pipelines, creating value through genes and improving human life and health.

About PackGene Biotech

PackGene Biotech (PackGene) is a CRO & CTDMO technology company specializing in recombinant adeno-associated virus (rAAV) vector packaging. It provides fast, cost-effective, high-quality and scalable one-stop CMC solutions for cell and gene therapy (CGT) companies, covering early-stage R&D, preclinical development, clinical trials and drug approval for recombinant AAV vector therapeutics.

Since its founding in 2014, PackGene Biotech, as a leader in the AAV vector CRO service field, has provided tens of thousands of batches of customized AAV samples to clients in more than 20 countries, including leading global multinational pharmaceutical companies and various research institutions. In the CTDMO service field, PackGene Biotech leverages five major technology platforms, including the π-Alpha 293 high-yield AAV production platform and the π-Omega high-yield plasmid production platform, together with a complete quality and analytical system, to provide compliant services for pharmaceutical companies’ GMP-scale production needs for AAV and plasmids.

With the mission of “make gene therapy affordable” and the responsibility of accelerating the launch of innovative gene therapies, PackGene Biotech is committed to becoming a long-term global partner in gene therapy R&D and manufacturing. Driven by innovation, PackGene Biotech focuses on two key directions—advanced vector production technologies and breakthrough vector tools—to build an international brand in the CRO and CTDMO fields of gene therapy.