Metachromatic Leukodystrophy (MLD)

Orchard Therapeutics’ Biologics License Application for OTL-200, a gene therapy under investigation for metachromatic leukodystrophy treatment, has been accepted by the FDA. This rare disease treatment received approval in Europe back in 2020. Notably, the FDA has granted Orchard’s application Priority Review statu...

Gene Therapy to “reverse” Metabolic disorders! Metabolic disorders are collectively common, frequently severe and in many instances difficult or impossible to treat. Accordingly, there is a compelling need to explore novel therapeutic modalities, including gene therapy, and examine multiple phenotypes where the ri...