Lingyi Bio's Self-Developed Gene Therapy Drug LY-M001 Injection Receives FDA Rare Pediatric Disease Designation

Recently, Lingyi (Hangzhou) Biotechnology Co., Ltd. (hereinafter referred to as "Lingyi Biotech") has achieved an important milestone with its self-developed gene therapy drug, LY-M001 injection, receiving Rare Pediatric Disease Designation (RPDD) from the FDA. This recognition follows the Orphan Drug Designation (ODD) awarded to LY-M001 and is a testament not only to the potential of LY-M001 injection in treating rare diseases but also an affirmation of Lingyi Biotech's research and development capabilities in the field of pediatric rare diseases.

Medicines that have been granted Rare Pediatric Disease Designation (RPDD) by the FDA may not only be eligible to receive a Priority Review Voucher (PRV) but are also likely to receive additional support in the realms of drug development and market entry. This designation will further enhance Lingyi Biotech's international collaboration prospects and expedite its strategic positioning in the global marketplace.

The LY-M001 injection, a pioneering AAV gene therapy developed by Lingyi Biotech in China, is specifically designed to address Types I and III Gaucher disease and is categorized as a Class 1 innovative biological therapeutic product. Utilizing recombinant adeno-associated virus (rAAV) as a vector, this treatment allows for the expression of glucocerebrosidase, a crucial enzyme for patients, following a single intravenous administration. Lingyi Biotech has engineered an improved glucocerebrosidase gene therapy vector with full proprietary intellectual property rights, capable of long-term stable expression and degradation of harmful glycolipid metabolites within the body, with the aim of providing a sustained treatment solution for Gaucher disease.

Lingyi Biotech's AAV gene therapy for Gaucher disease, the LY-M001 injection, has received Investigational New Drug (IND) clinical trial approvals from both the National Medical Products Administration (NMPA) and the FDA in January 2024, and has since commenced registered clinical Phase I trials. To date, the LY-M001 injection has been administered to several Gaucher disease patients, both adults and children, with initial results indicating favorable outcomes in both safety and efficacy.