LV-GALNS

LV-GALNS is a third-generation lentiviral vector encoding the human N-acetylgalactosamine-6-sulfatase (GALNS) gene, developed for the treatment of Mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. Developed by the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), this gene therapy approach involves the ex vivo transduction of hematopoietic stem and progenitor cells (HSPCs). Once re-infused, these modified HSPCs engraft and differentiate into various hematopoietic lineages, including osteoclasts, which act as a resident source of the functional GALNS enzyme. This strategy aims to achieve supraphysiological enzyme levels to facilitate metabolic cross-correction in poorly vascularized and barrier-protected tissues like bone and cartilage, addressing the severe skeletal dysplasia characteristic of MPS IVA that is often refractory to standard enzyme replacement therapies.