LY-M003

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Recently, a prospective, single center, open label, single arm, single dose clinical study evaluating the safety, tolerability, and efficacy of LY-M003 injection in the treatment of adult patients with Wilson's disease was launched at the First Affiliated Hospital of Zhejiang University School of Medicine (hereinafter referred to as "Zhejiang First Hospital"). The project was led by Professor Yu Chaohui, the leader of the Gastroenterology Department, as the main researcher. LY-M003 injection is an innovative gene therapy drug developed by Lingyi (Hangzhou) Biotechnology Co., Ltd. (hereinafter referred to as "Lingyi Biotechnology").

The Department of Gastroenterology at Zhejiang First Hospital is not only the earliest and largest clinical diagnosis and treatment center for digestive system diseases in Zhejiang Province, but also a leading unit in the research field of digestive system diseases in Zhejiang Province, thanks to its excellent professional level and academic status. It has become a national key clinical specialty, a national training base for digestive endoscopy diagnosis and treatment technology, a key medical discipline in Zhejiang Province, and a leading unit in the research field of digestive system diseases in Zhejiang Province.

Professor Yu Chaohui is currently the Vice President of the First Hospital of Zhejiang Medical University and the leader of the Gastroenterology Department. He is a professor, chief physician, and doctoral supervisor of Internal Medicine at Zhejiang University. He is also a leading talent in scientific and technological innovation under the National Thousand Talents Program, a young and middle-aged leading talent in scientific and technological innovation under the Ministry of Science and Technology, and an outstanding talent in the new century under the Ministry of Education. He has been selected as a second level, first level, and key funded talent in Zhejiang Province's 151 Talent Program, as well as a high-level innovative talent in health in Zhejiang Province. Professor Yu Chaohui has long been engaged in the diagnosis and treatment, clinical teaching, and scientific research of digestive system diseases. He is dedicated to the mechanism research and new drug development of metabolic liver disease, and has led multiple national level scientific research projects such as the National Key R&D Program, National Natural Science Foundation of China, and 973 Program sub projects.

Regarding Wilson's disease

Wilson's disease (WD), also known as Wilson's disease, is a rare autosomal recessive metabolic disorder caused by a mutation in the copper transport ATPase β (ATP7B) gene located on the long arm of chromosome 13 (13q14.3), which impairs copper transport ability and leads to the accumulation of copper ions in multiple organs such as the liver, brain, and kidneys, resulting in organ involvement. The clinical manifestations of WD are complex, mainly involving lesions in the liver and nervous system, including abnormal liver function, enlargement of the liver or spleen, acute liver failure, neurodegenerative changes, neurological dysfunction, selective nerve vulnerability, and executive dysfunction. Other systemic involvement manifestations may also occur, and may worsen progressively during the course of the disease.

At present, the standard treatment for Wilson's disease is copper chelator penicillamine, which increases urinary copper excretion, and zinc, which reduces copper absorption. Although widely used, there are also many shortcomings, such as frequent daily medication, poor efficacy in treating neurological symptoms, and significant individual differences in drug response and tolerance among patients. If the patient enters the decompensated cirrhosis stage, liver transplantation treatment may also need to be considered. Gene therapy, as an emerging treatment method, has the potential to fundamentally and long-term improve the symptoms of Wilson's disease through a single administration.

About LY-M003

LY-M003 injection is a gene therapy drug independently developed by Lingyi Biotechnology for Wilson's disease. Using recombinant adenovirus rAAV as a vector, the normal functioning ATP7B gene is introduced into the patient's liver cells through a single intravenous infusion, and the ATP7B protein with normal copper excretion activity is stably expressed in the liver cells for a long time. This innovative therapy aims to restore the copper metabolism ability of liver cells, reduce the accumulation of copper ions in the body, restore the activity of ceruloplasmin, thereby systematically improving systemic symptoms and achieving the goal of treating Wilson's disease. At present, LY-M003 injection has obtained FDA Orphan Drug Design (ODD) and Rare Pediatric Disease Design (RPDD) accreditation.

About Lingyi Biotechnology

Lingyi Biotechnology was founded in February 2021 and is a globally leading innovative drug development company for single gene genetic diseases. The company has a world-class team for gene therapy discovery, research and development, and industrialization, dedicated to the research and industrialization of the First In Class gene therapy pipeline. The company is based on innovation at the source, focusing on clinically urgent disease fields such as metabolism, central nervous system, and ophthalmology. Starting from key technologies such as animal models of genetic diseases, we provide patients with one-time solutions with the highest quality standards.

Lingyi Biotechnology has obtained IND clinical trial approval from NMPA and FDA for LY-M001 injection, an AAV gene therapy drug for Gaucher's disease, in January 2024, and has officially started registered phase I clinical trials. It has also obtained FDA orphan drug ODD qualification recognition. At present, LY-M001 injection has been administered to several adult and pediatric patients with Gaucher's disease, and preliminary data shows that LY-M001 injection has good safety and efficacy.

Article source: Lingyi Biology