TYF-ARSA

TYF-ARSA is an investigational gene therapy candidate designed to treat metachromatic leukodystrophy (MLD), a rare and fatal lysosomal storage disorder caused by mutations in the arylsulfatase A (ARSA) gene. The therapy utilizes an improved, self-inactivating lentiviral vector to deliver a functional human ARSA gene. It is being evaluated through two primary therapeutic strategies: an ex vivo hematopoietic stem cell gene therapy (HSC-GT), where a patient's own hematopoietic stem cells are genetically modified and transplanted back into the patient, and an in vivo direct injection approach via intrathecal and intravenous routes. By restoring ARSA enzyme activity, the treatment aims to facilitate the degradation of sulfatides, preventing their toxic accumulation in the central and peripheral nervous systems and halting progressive demyelination.