LV-GALNS + LV-GLB1 + LV-MAN2B1

LV-GALNS + LV-GLB1 + LV-MAN2B1 is a combination of three ex vivo lentiviral gene therapy vectors developed under the PLATFORM project by the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) and Fondazione Telethon. The platform approach utilizes a shared lentiviral vector backbone, promoter, regulatory elements, and manufacturing processes, differing only by the disease-specific transgene. LV-GALNS encodes N-acetylgalactosamine-6-sulfatase (GALNS) for the treatment of Mucopolysaccharidosis type IVA (MPS IVA); LV-GLB1 (including an optimized LV-enhGLB1) encodes beta-galactosidase (GLB1) for GLB1-related disorders such as Mucopolysaccharidosis type IVB (MPS IVB) and GM1 gangliosidosis; and LV-MAN2B1 encodes lysosomal alpha-mannosidase (MAN2B1) for Alpha-mannosidosis. The therapy involves transducing autologous hematopoietic stem and progenitor cells (HSPCs) ex vivo, which are then reinfused into the patient to serve as a circulating and tissue-resident source of the respective functional enzymes, enabling cross-correction of affected non-hematopoietic cells, particularly in skeletal and neurological tissues.

Other names

HSPC-GT platform for LSDsLV-GALNSLV-GLB1LV-GLB-1LV-GLB 1LV-MAN2B1LV-MAN-2B1LV-MAN 2B1LV-enhGLB1LV-enhGLB-1LV-enhGLB 1PLATFORM project