Biochemical Engineering
Orchard Therapeutics to file for approval of MLD gene therapy
4th September 2019
Orchard Therapeutics has said it plans to file for approval for a gene therapy for the life-threatening rare disease metachromatic leukodystrophy (MLD) in Europe, after publishing new data. The biotech said it planned to file data from its OTL-200 gene therapy with regulators early next year in Europe after results from a late-stage trial showed clinical benefit on cognitive and motor function. Source: Pharmaphorum 4/9/2019
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