PackGene Supports Lingyi Biotech's Gaucher Disease Gene Therapy LY-M001 in Receiving FDA IND Approval
Lingyi Biotech
Gene Therapy Drug Developer
On January 24, 2024, Lingyi Biotech Co., Ltd. (hereinafter referred to as "Lingyi Biotech") independently developedLY-M001Injection solution receives approval from the U.S. Food and Drug Administration (FDA) New Drug Clinical Trial (IND) License (IND Number: 30210).
LY-M001 Injection is the first self-developed AAV gene therapy drug in China 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 a proprietary improved glucocerebrosidase gene therapy vector with complete 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 been initiated, and two subjects have already been dosed. Preliminary IIT data shows that LY-M001 demonstrates good performance in both efficacy and safety, with no adverse events reported.
About Lingyi Biotech
Lingyi Biotech, established in February 2021, is a globally leading company in the development of innovative drugs for monogenic hereditary diseases. The company boasts an internationally top-tier team in gene therapy discovery, research and development, and industrialization, focusing on the R&D and commercialization of FIC gene therapy pipelines. Committed to original innovation, the company concentrates on disease areas with urgent clinical needs such as metabolism, the central nervous system, and ophthalmology. Starting with key technologies like animal models of genetic diseases, it aims to provide patients with one-time solutions according to the highest quality standards.
PackGene Biotech is a CRO & CTDMO technology company specializing in the packaging of recombinant adeno-associated virus (rAAV) vectors. It provides rapid, cost-effective, high-quality, and scalable one-stop CMC solutions for the early research, preclinical development, clinical trials, and drug approval of recombinant AAV vector drugs for cell and gene therapy (CGT) companies. Since its establishment in 2014, as a leader in the AAV vector CRO service field, PackGene Biotech has delivered tens of thousands of customized AAV batches to customers across more than 20 countries, including top 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 Cell AAV High-Yield Platform and the π-Omega™ Plasmid High-Yield Platform, supported by comprehensive quality and analytical systems, to offer compliant services for the GMP-scale production needs of AAVs and plasmids for pharmaceutical companies.
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