Lingyi Biotech Announces Successful Completion of First-in-World Gene Therapy in Adolescent with Type I Gaucher Disease Using LY-M001 at Shanghai Children's Medical Center

Recently, a"Evaluation of LY-M001 Injection Single Intravenous Infusion Type IGaucher's DiseaseA Prospective, Single-Center, Open-Label, Single-Arm Clinical Study on the Safety and Efficacy in Adolescent PatientsSuccessfully carried out and achieved efficacy at the National Children's Medical Center, Shanghai Children's Medical Center affiliated with Shanghai Jiao Tong University School of Medicine.

This project was initiated by Professor Wang Xiumin from the Department of Endocrinology, Genetics, and Metabolism and the Clinical Research Ward as the principal investigator, with participation from a multidisciplinary team including the Department of Endocrinology, Genetics, and Metabolism, Clinical Research Ward, Hematology-Oncology Department, and Radiology Department.In August this year, the medical team successfully completed the drug treatment for a 15-year-old adolescent patient with Type I Gaucher disease. This is also the world's first case of an adolescent patient with Type I Gaucher disease receiving gene therapy.

Gaucher Disease (Gaucher disease, GD) is a condition caused by glucocerebrosidase (GBA).Gene MutationAutosomal recessive lysosomal storage disease, characterized by lipid deposition in multiple systems. The affected blood, liver, spleen, bones, and nervous system exhibit impaired function, resulting in corresponding clinical manifestations such as hepatosplenomegaly, anemia, thrombocytopenia, bone pain, skeletal lesions, developmental delays, potential lifelong disability, or even death. Current primary treatments for Gaucher's disease include enzyme replacement therapy, hematopoietic stem cell transplantation, substrate inhibition therapy, and gene therapy. Gene therapy involves the infusion of a viral vector-based gene therapy product, which can specifically transduce target organs post-administration and enable long-term expression of the GCase protein within those organs. The harmful glycolipid metabolites accumulated in various organs of Gaucher's disease patients are degraded by the GCase protein, effectively treating the disease.

In February 2022, the clinical research ward of Shanghai Children's Medical Center was officially launched. Centered on patients, it focuses on cutting-edge clinical application exploration for rare pediatric diseases, breaking through the treatment bottlenecks of rare diseases and solving the "choke point" issue of drug shortages for children in China. As the National Children's Medical Center and the Shanghai Gene Therapy Clinical Medicine Research Center, the hospital is actively committed to the development and application of innovative drugs and devices produced in China, particularly clinical research related to gene therapy for rare diseases. The world's first gene therapy project for treating Aromatic L-Amino Acid Decarboxylase Deficiency has completed its initial phase.IITResearch and enter the registered clinical stage. Currently, including those targeting Type I Gaucher disease,Duchenne Muscular DystrophyGene therapy projects for multiple diseases, such as MECP2 duplication syndrome and acute myeloid leukemia, have all been proceeding smoothly.