Lingyi Biotech's Investigational Gene Therapy LY-M001 Receives FDA Rare Pediatric Disease Designation for Gaucher Disease
Lingyi Biotech
Gene Therapy Drug Developer
Recently,LY-M001 Injection, a Gene Therapy Drug Independently Developed by Lingyi Biotech of Xingbei·FTO No.1 Park, Receives FDA Pediatric Rare Disease Designation(Rare Pediatric Disease Designation, RPDD), this is another significant milestone after LY-M001 received Orphan Drug Designation (ODD). This designation not only recognizes the potential of LY-M001 injection in treating rare diseases but also marks the acknowledgment of Lingyi Biotech's R&D capabilities in the field of pediatric rare diseases.
Drugs granted RPDD designation, in addition to potentially receiving the FDA's Priority Review Voucher (PRV), are also expected to gain more support in drug development and market access. This designation will also bring more international cooperation opportunities for Lingyi Biotech, accelerating its global market layout.
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, enabling the expression of the required glucocerebrosidase in patients after a single intravenous infusion. Lingyi Biotech has developed a proprietary improved glucocerebrosidase gene therapy vector with complete independent intellectual property rights, which can achieve long-term stable expression in vivo and degrade harmful glycolipid metabolites, thereby achieving the goal of long-term treatment for Gaucher disease.
Lingyi Biotech's Gaucher disease AAV gene therapy drug LY-M001 injection was granted IND approval for clinical trials by the NMPA and FDA in January 2024, and Phase I registrational clinical trials have officially commenced. To date, the LY-M001 injection has been administered to several adult and pediatric patients with Gaucher disease, with preliminary data indicating favorable performance in both safety and efficacy.
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, development, and industrialization, focusing on the R&D and commercialization of First-In-Class gene therapy pipelines. With a commitment to original innovation, the company 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, Lingyi Biotech aims to provide patients with one-time solutions under the highest quality standards.
About SimbayPark
Shanghai FT1 Life Science Park (Abbreviation"Simbay Park, Star North · Free Trade Zone No.1") is located in the Waigaoqiao Bonded Area, the core of Shanghai's Pudong New Area Free Trade Zone. It is an innovative service-oriented characteristic industrial park under the Star North Group, focusing on the incubation, cultivation, and transformation of medical enterprises.The park relies on the unique "Free Trade + Bonded" outward-oriented economic regional advantages, and by gathering the entire industry chain service resources of CRO+CDMO+CSO+EHS in the biopharmaceutical field, government innovation and entrepreneurship support policies, and numerous well-known investment institutions, it provides enterprises with truly in-depth technical incubation in the medical industry, acting as a steward for the transformation of corporate technological achievements, and is committed to creating an international biopharmaceutical industry innovation and entrepreneurship base.
