(RTTNews) - Gene therapy leader Orchard Therapeutics plc (ORTX) announced Monday the Swiss Agency for Therapeutic Products (Swissmedic) has approved Libmeldy (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, for the treatment of early-onset metachromatic leukodystrophy (MLD).
MLD is a rare, fatal genetic disorder caused by a mutation in the gene responsible for encoding the enzyme arylsulfatase A (ARSA) leading to neurological damage and developmental regression.
Libmeldy aims to correct the underlying genetic cause of MLD by inserting a working copy of the ARSA gene into the genome of a patients' own HSCs. The genetically modified cells are infused back into the patient, where they can naturally migrate across the blood-brain barrier into the central nervous system, engraft, and express the functional enzyme.
In clinical trials, treatment with Libmeldy resulted in the preservation of cognitive development and maintenance of motor function past ages at which untreated patients showed severe cognitive and motor impairments.
