Preliminary signs of the efficacy of gene therapy for MLD

https://www.biospace.com/article/releases/passage-bio-announces-pre-clinical-data-presentation-at-annual-asgct-meeting/

Passage Bio have been evaluating the pre-clinical efficacy of the adeno-associated virus (AAV)-mediated gene therapy for MLD. A mouse model was developed and utilised in reproducing key aspects of human metachromatic leukodystrophy (MLD) neuropathology to enable evaluation of efficacy of AAV gene therapy. This model reproduces important aspects of human leukodystrophy neuropathology, including key biomarkers. Preliminary findings showed the administration of Passage Bio’s gene therapy significantly reduced the neurological deficits of MLD in this model.