On January 16, 2024, LingyiMed (Hangzhou) Co., Ltd, a biotechnology company, successfully obtained an implied permission from the National Medical Products Administration (NMPA) for the Investigational New Drug (IND) clinical trials of its new therapeutic biological product, LY-M001 Injection. This development marks a significant step in the company’s research efforts, with the acceptance number registered as CXSL2300730.

gaucher disease

LY-M001 Injection is notable for being the first AAV gene therapy drug developed in China aimed at treating Type I or Type III Gaucher Disease. Utilizing recombinant adeno-associated virus (rAAV) as a vector, the drug facilitates the expression of glucocerebrosidase, essential for patients, through a single intravenous administration. LingyiMed has innovated in this field by developing a modified glucocerebrosidase gene therapy vector, which they hold full intellectual property rights to. This vector is designed for long-term stable expression in the body, targeting the degradation of harmful glycolipid metabolites, thereby potentially offering a long-term treatment option for Gaucher Disease.

The initiation of an IIT clinical research project, spearheaded by Dr. Dean Huang He of the First Affiliated Hospital of Zhejiang University School of Medicine, aligns with these developments. The project has reportedly started with the administration to the first patient and preliminary data suggests positive results in terms of efficacy and safety, with no adverse events recorded.

Dr. Lin Qing, the founder and CEO of LingyiMed, expressed confidence in the progress, emphasizing the milestone as a testament to the team’s capability in transitioning from drug discovery to clinical development. Additionally, LingyiMed endeavors in international markets are evidenced by the acceptance of their IND application for LY-M001 Injection by the U.S. Food and Drug Administration (FDA) in December 2023.

LingyiMed has made strides in AAV gene therapy drugs, thanks in part to the comprehensive CMC services provided by a notable CRO & CTDMO, PackGene. This collaboration has been pivotal for the project’s advancement. The CRO & CTDMO involved is set to continue its substantial role in the gene therapy sector, offering integrated CMC solutions to foster drug innovation and the advancement of high-quality projects.

PackGene, established in 2014, is known for its commitment to making gene therapy more accessible and affordable. The company has positioned itself as a leader in AAV vector CRO services, serving clients from over 20 countries, including top multinational pharmaceutical companies and research institutions. With its focus on innovative technology and advanced vector production, PackGene seeks to establish a strong international presence in the gene therapy CRO and CTDMO sectors.

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About PackGene

PackGene is a CRO & CDMO technology company that specializes in packaging recombinant adeno-associated virus (rAAV) vectors. Since its establishment in 2014, PackGene has been a leader in the AAV vector CRO service field, providing tens of thousands of custom batches of AAV samples to customers in over 20 countries. PackGene offers a one-stop CMC solution for the early development, pre-clinical development, clinical trials, and drug approval of rAAV vector drugs for cell and gene therapy (CGT) companies that is fast, cost-effective, high-quality, and scalable. Additionally, the company provides compliant services for the GMP-scale production of AAVs and plasmids for pharmaceutical companies, utilizing five technology platforms, including the π-Alpha 293 cell AAV high-yield platform and the π-Omega plasmid high-yield platform. PackGene's mission is to make gene therapy affordable and accelerate the launch of innovative gene drugs. The company aims to simplify the challenging aspects of gene therapy development and industrialization processes and provide stable, efficient, and economical rAAV Fast Services to accelerate gene and cell therapy development efforts from discovery phase to commercialization.

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