Orchard Therapeutics (NASDAQ:ORTX) has dosed the first patient in a study evaluating a cryopreserved formulation of OTL-103, an ex vivo autologous hematopoietic stem cell gene therapy for the treatment of Wiskott-Aldrich Syndrome (WAS).
WAS is an inherited immune disorder where autoimmunity and abnormal platelet function results in severe infections and bleeding episodes that can be life-threatening.
Orchard is conducting the trial so it may commercialize OTL-103 and other gene therapies using cryopreserved cells. “We believe transitioning this program to a cryopreserved formulation is an essential step for the eventual commercialization of OTL-103, enabling patients to access treatment, if approved, on a global scale,” Mark Rothera, president and CEO of Orchard, said in a statement.
Positive interim results from the registrational trial for OTL-13 for WAS, which used a fresh cell formulation, were recently published in the peer-reviewed journal Lancet Hematology, which can be accessed here.
