Warm congratulations to Lingyi Bio on the successful results of the clinical study of LY-M001 injection for adolescent patients with Type I Gaucher disease at the Shanghai Children’s Medical Center.

Recently, a prospective, single-center, open-label, single-arm clinical study titled “Assessment of the Safety and Efficacy of a Single Intravenous Infusion of LY-M001 Injection in Adolescent Patients with Type I Gaucher Disease” has been successfully initiated and has achieved efficacy outcomes at the National Center for Children’s Health—Shanghai Children’s Medical Center, School of Medicine, Shanghai Jiao Tong University.

This study was initiated by Professor Wang Xumin (Chief Investigator), Department of Endocrinology and Metabolic Diseases, and the Clinical Research Ward. It was jointly participated in by a multidisciplinary team including the Department of Endocrinology and Metabolic Diseases, the Clinical Research Ward, the Department of Hematology and Oncology, and the Department of Radiology. In August this year, the medical team successfully administered LY-M001 to a 15-year-old adolescent patient with Type I Gaucher disease. This is also the first globally reported case of an adolescent patient with Type I Gaucher disease receiving gene therapy.

Gaucher disease (GD) is an autosomal recessive lysosomal storage disorder caused by genetic variants in the glucocerebrosidase (GBA) gene. It is characterized by lipid accumulation affecting multiple systems, leading to impaired functions of the blood, liver and spleen, bones, and nervous system, which manifest as clinical symptoms including hepatosplenomegaly, anemia, thrombocytopenia, bone pain, and skeletal lesions, as well as developmental delay, potentially resulting in lifelong disability and even death. Current main treatment options for Gaucher disease include enzyme replacement therapy, hematopoietic stem cell transplantation, substrate reduction therapy, and gene therapy. Gene therapy involves infusing a virus-based gene therapy product; after administration, it can specifically transduce target organs and long-term express the GCase protein in the target organ. The harmful glycosphingolipid metabolites accumulated in patients’ organs are then degraded by the GCase protein, thereby effectively treating Gaucher disease.

At present, the subject has completed an intensive follow-up for eight weeks post-treatment. The child is doing well, with no obvious adverse reactions observed. Enzyme replacement therapy has been discontinued; enzyme activity has returned to normal, and the quality of life has improved significantly compared with before.

In February 2022, the Clinical Research Ward at Shanghai Children’s Medical Center was officially put into operation, with a patient-centered approach. The hospital has carried out cutting-edge clinical application exploration for children’s rare diseases, aiming to break through bottlenecks in rare-disease treatment and address the “neck-sticking” problem of insufficient availability of children’s medicines in China. As a National Center for Children’s Health and the Shanghai Clinical Medical Research Center for Gene Therapy, the hospital is actively committed to the development and application of domestically innovative drugs and devices, especially clinical research related to gene therapy for rare diseases. The world-first gene therapy project for aromatic L-amino acid decarboxylase deficiency has completed preliminary IIT research and has entered the phase of registration-directed clinical development. Currently, multiple gene therapy projects targeting diseases including Type I Gaucher disease, Duchenne muscular dystrophy, MECP2 duplication syndrome, and acute myeloid leukemia have also been successfully carried out.

The completion of the world’s first gene therapy for an adolescent patient with Type I Gaucher disease at Shanghai Children’s Medical Center not only marks a major breakthrough in gene therapy technology, but also brings new hope and a bright prospect for rare-disease treatment, while demonstrating China’s strength and potential in medical innovation and interdisciplinary collaboration. LY-M001 injection is an innovative gene therapy drug developed by Lingyi (Hangzhou) Biotechnology Co., Ltd. Piptech Biotech is honored to support the progress of Lingyi’s AAV gene therapy drugs and to provide the project with high-quality CMC services. In the future, Piptech Biotech will continue to focus on gene therapy, providing one-stop CMC solutions for drug innovation and helping more high-quality projects move forward.

About LY-M001 Injection

LY-M001 injection is the first self-developed AAV gene therapy drug in China for Type I and Type III Gaucher disease. It is classified as a Type 1 innovative biologic for therapeutic use. The product uses recombinant adeno-associated virus (rAAV) as the vector. After a single intravenous infusion, it enables expression of the glucocerebrosidase required by the patient. Lingyi Biotech has developed an engineered glucocerebrosidase gene therapy vector with fully independent intellectual property rights, which can achieve long-term stable expression in vivo and degrade harmful glycosphingolipid metabolites, thereby achieving the goal of long-term treatment of Gaucher disease.

About Lingyi Biotech

Lingyi Biotech was established in February 2021. It is a globally leading company focused on the development of innovative drugs for single-gene genetic diseases. The company has an internationally first-class team for gene therapy discovery, research and development, and industrialization, and is dedicated to the development and industrialization of First-In-Class gene therapy pipelines. Lingyi Biotech is rooted in source innovation and focuses on clinical areas with urgent unmet needs, including metabolism, the central nervous system, and ophthalmology. Starting from key technologies such as genetic disease animal models, the company provides patients with one-time solutions using the highest quality standards.

Lingyi Biotech’s in-development AAV gene therapy drug for Gaucher disease, LY-M001 injection, received IND approval for clinical trials from both the NMPA and the FDA in January 2024, and the registered Phase I/II clinical trials have been formally initiated. It has also obtained FDA orphan drug designation (ODD). At present, LY-M001 injection has been administered to several adult and pediatric patients with Gaucher disease, and preliminary data show that LY-M001 injection demonstrates good performance in both safety and efficacy.