Lingyi Bio Announces First-in-Human Dosing of LY-M003 Gene Therapy for Wilson’s Disease

Lingyi Biotech stated that it will commit to collaborating with medical professionals worldwide to accelerate the clinical research and product development of LY-M003 injection, hoping to bring this innovative drug to patients with Wilson’s disease globally as soon as possible, achieving the therapeutic vision of "one dose, lifelong benefit."

Hepatolenticular Degeneration (WD), also known as Wilson's Disease, is a rare autosomal recessive inherited metabolic disorder. The disease is caused by mutations in the copper-transporting ATPase β (ATP7B) gene on chromosome 13, leading to the accumulation of copper ions in organs such as the liver, brain, and kidneys, causing organ damage. The clinical manifestations of WD are diverse, mainly including lesions of the liver and nervous system, such as abnormal liver function, enlargement of the liver or spleen, acute liver failure, and neurodegeneration.

Current treatment options include copper chelators and zinc agents, but they have many shortcomings, such as frequent dosing, poor efficacy, and significant individual differences. Gene therapy, as an emerging treatment method, has the potential to fundamentally improve the symptoms of Wilson's disease with a single administration.

LY-M003 Injection is a gene therapy drug independently developed by Lingyi Biotech for Wilson’s disease. It uses recombinant adeno-associated virus (rAAV) as a vector to deliver the functionally normal ATP7B gene into patients' hepatocytes via a single intravenous infusion, enabling long-term stable expression of the ATP7B protein with normal copper transport activity in hepatocytes. This innovative therapy aims to restore copper metabolism in hepatocytes, reduce copper ion accumulation in the body, and recover ceruloplasmin activity, thereby systematically improving systemic symptoms to achieve the treatment goal for Wilson’s disease. Currently, LY-M003 Injection has received FDA Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD).

Lingyi Biotech was founded in February 2021 and is a global leader in the development of innovative drugs for monogenic inherited diseases. The company boasts a world-class team in gene therapy discovery, research and development, and industrialization, focusing on the R&D and commercialization of First-In-Class gene therapy pipelines. With a commitment to original innovation, Lingyi Biotech 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, the company aims to provide patients with one-time solutions according to the highest quality standards.

Lingyi Biotech's AAV gene therapy drug LY-M001 injection for Gaucher disease received IND approval from the NMPA and FDA in January 2024, and Phase I/II clinical trials have officially commenced. It has also been granted Orphan Drug Designation (ODD) by the FDA. Currently, LY-M001 injection has been administered to several adult and pediatric patients with Gaucher disease, and preliminary data indicate that LY-M001 injection demonstrates favorable safety and efficacy.