LY-M001: China's First AAV Gene Therapy for Gaucher Disease Type I Achieves Successful First-in-Human Dosing, Paving the Way for Potential One-Time, Lifelong Treatment

Jan 06, 2024 09:02 CST Updated 09:02

Lingyi Biotech

Gene Therapy Drug Developer

Recently, the first gene therapy clinical research project in China for the treatment of Gaucher disease type I — "A Prospective, Single-Center, Open-Label, Single-Arm Study to Evaluate the Safety and Preliminary Efficacy of a Single Intravenous Infusion of LY-M001 Injection in Adult Patients with Gaucher Disease Type I" — has been successfully initiated at the First Affiliated Hospital of Zhejiang University School of Medicine (hereinafter referred to as "FAHZU"). The project is led by Dr. Huang He, the president of FAHZU, as the principal investigator, with the first patient dosed by Dr. Weiyan Zheng’s team from the Bone Marrow Transplantation Center of the Hematology Department. Lingyi Biotech Co., Ltd. (hereinafter referred to as "Lingyi Biotech") provided support.

President Huang He stated: "Gene therapy is a very cutting-edge and entirely new treatment technology. The gene therapy product LY-M001 injection developed by Lingyi Biotech has solid preclinical research data. Zhejiang Hospital will work hand in hand with Lingyi Biotech to actively promote this clinical study. The dosing process for the first patient went very smoothly, and the patient's condition was also very stable after dosing. Our research team will continue to closely monitor the patient’s physical condition and follow up on various indicators. We believe that the launch of this project and the successful dosing of the first patient are landmark events in the history of gene therapy research for Gaucher disease type I."

Dr. Zheng Weiyan, Chief Physician, stated: "It has been over a month since the administration of the drug to the subject. Regular follow-ups have shown that the overall condition is good, with no significant adverse reactions observed. In terms of preliminary efficacy indicators, the expected results have been achieved. The activity of glucocerebrosidase in the subject's body recovered to above normal levels within a few weeks and has continued to rise. We look forward to observing more safety and efficacy data for LY-M001 injection."”

Huang He, Dean of the First Affiliated Hospital of Zhejiang University School of Medicine

Dr. Lin Qing, founder of Lingyi Biotech, stated that he is very pleased to have the opportunity to collaborate with Director Huang's team on the clinical research of LY-M001 injection for Gaucher's disease. The initiation of this clinical study and the successful enrollment and dosing of the first patient also mark a significant milestone for Lingyi Biotech. The data from the first patient so far has been very encouraging, demonstrating good efficacy while ensuring safety. Zhejiang Hospital has deep expertise and resources in the diagnosis and treatment of rare diseases. Director Huang, the leader of the Hematology Department and Bone Marrow Transplantation Center at Zhejiang Hospital, has extensive experience in clinical research. The medical team assembled by Director Huang for this project also has rich experience in the screening and treatment of Gaucher's disease. We are highly anticipating the groundbreaking research of this project.

About LY-M001 Injection

LY-M001 Injection is the first self-developed AAV gene therapy drug in China targeting Type I and Type III Gaucher disease, classified as a Class 1 innovative biologic therapeutic product. The product uses recombinant adeno-associated virus (rAAV) as a vector, which expresses the required glucocerebrosidase in patients after a single intravenous infusion. Lingyi Biotech has developed a proprietary improved glucocerebrosidase gene therapy vector with full independent intellectual property rights, enabling long-term stable expression in vivo and degradation of harmful glycolipid metabolites, thereby achieving the goal of long-term treatment for Gaucher disease.

About Gaucher's Disease

Gaucher's disease is a rare autosomal recessive genetic disorder, and its pathogenesis is due toGBA1Gene mutations lead to a lack or reduction in the activity of glucocerebrosidase in the body, causing its substrate, glucocerebroside, to accumulate in the lysosomes of macrophages in the liver, spleen, bones, lungs, and even the brain, forming typical storage cells known as "Gaucher cells." This accumulation subsequently causes pathological changes in body tissues and organs, progressively worsening over time. Clinical manifestations often include hepatosplenomegaly, anemia, thrombocytopenia, bone pain, and neurological symptoms.

About the Bone Marrow Transplant Center of the Hematology Department at Zhejiang Medical University First Affiliated Hospital

The Bone Marrow Transplant Center of Zhejiang First Hospital was established in the early 1950s, developing a comprehensive technical system for the complete and curative treatment of various malignant hematological diseases. Its clinical efficacy has reached a leading level in China and an advanced international standard, earning multiple national and provincial awards.

Patient Recruitment

Dear Participant:

The First Affiliated Hospital of Zhejiang University is conducting a "Prospective, Single-center, Open-label, Single-arm Study to Evaluate the Safety and Preliminary Efficacy of a Single Intravenous Infusion of LY-M001 Injection in Adult Patients with Type I Gaucher Disease" (Protocol Number: LY-M001-GD-001), hereinafter referred to as "this study." This study has been approved by the hospital’s Ethics Committee and plans to recruit adult patients with Type I Gaucher Disease in China to participate in this study.

If you or someone around you is a patient who meets the following criteria, please contact us:

1. Aged ≥18 years and ≤60 years, gender不限;

2. The subject should be confirmed by laboratory testing.GBA1Double mutation of alleles and diagnostic conclusion of Type I Gaucher's disease (i.e., glucocerebrosidase activity reduced to less than 30% of normal values);

3. Subjects are patients with Type I Gaucher disease; those who have previously received specific treatment for Type I Gaucher disease must undergo a washout period of 5 half-lives.

4. Subjects and their partners have no fertility plans from the screening period to 6 months after the study ends, subjects have no plans to donate sperm or eggs, and they voluntarily adopt effective contraceptive measures (such as abstinence, condoms, etc.);

Whether to participate is entirely up to your discretion. If you are interested in participating, please contact the study doctor, who will provide you with a detailed introduction of the study and the matters you need to cooperate with, and make an initial assessment of whether you meet the eligibility criteria. Even if you choose not to participate, there will be no adverse consequences for you, and your relevant information will be kept confidential. The final decision on whether you can successfully participate in this study rests with the researchers. Your participation will contribute to the advancement of medical science, and we look forward to your involvement.

Contact: Clinical Coordinator

Contact Number: 19121572057

About Lingyi Biotech

Lingyi Biotech, established in February 2021, is a globally leading company at the forefront of developing drugs for monogenic hereditary diseases. The company focuses on original innovation and targets 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 one-time solutions for patients.

In the field of rare diseases, the company boasts a world-class team in gene therapy discovery, research, development, and industrialization. It also possesses a globally leading platform for the creation and validation of rare disease animal models, addressing the critical challenge of the lack of effective animal models in rare disease research and development.

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