Libmeldy is an autologous haematopoietic stem cell (HSC) gene therapy product for early-onset MLD made specifically for each patient using the patient’s own blood stem cells.1See indication
Metachromatic leukodystrophy (MLD) is a rare, inherited, neurometabolic disease that causes rapid and progressive demyelination and neurodegeneration, leading to severe neurological disability and death.2 MLD is caused by mutations in the arylsulfatase A (ARSA) gene which lead to ARSA enzyme deficiency.1See disease info
Libmeldy is indicated for the treatment of children with early-onset MLD. CD34+ cells are transduced ex vivo with a lentiviral vector encoding ARSA cDNA. It is an autologous gene therapy, meaning a patient’s own stem cells are genetically corrected by inserting a functional copy of the ARSA gene and these are returned to their body.1See full indication
ARSA, arylsulfatase A; cDNA, complementary DNA; HSC, haematopoietic stem cell; MLD, metachromatic leukodystrophy
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