ORTX - Orchard's gene therapy shows encouraging action in a rare neurometabolic disorder study

Orchard Therapeutics (ORTX) has reported initial data from an ongoing proof-of-concept trial evaluating OTL-201, an autologous hematopoietic stem cell gene therapy candidate, for the treatment of mucopolysaccharidosis type IIIA (MPS-IIIA, or Sanfilippo syndrome type A), an inherited neurometabolic disorder caused by the buildup sugar molecules in the body leading to intellectual disabilities and loss of motor function. Data were presented at the American Society of Hematology Annual Meeting.According to the data, the therapy was generally well-tolerated with no treatment related adverse events or serious adverse events to date.Evidence of hematological engraftment as suggested by recovery of neutrophils and platelets post myeloablative conditioning was observed.N-sulphoglucosamine sulphohydrolase enzyme expression reached supra-physiological levels in plasma, total leukocytes and multiple cell subpopulations, including CD3+ and CD15+ cells, within 3 months of receiving OTL-201.Reduction of urinary heparan sulfate from 60.8 mg/mmol creatinine at baseline to the normal range by three months post-treatment with gene therapy.Three patients have been

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Orchard's gene therapy shows encouraging action in a rare neurometabolic disorder study