ORTX - Orchard Therapeutics reports HSC gene therapy data in rare genetic disorder

Orchard Therapeutics (ORTX) announces data published in the New England Journal of Medicine evaluating the safety and efficacy of investigational gene therapy products, including OTL-101, for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID).Fifty ADA-SCID patients were treated with investigational gene therapy composed of autologous CD34+ hematopoietic stem cells (HSCs) transduced ex vivo with a self-inactivating lentiviral vector ((LVV)) encoding the human ADA gene.Results showed 100% overall survival and ?95% event-free survival at two and three years. The data were taken from three Phase 1/2 clinical studies (n=40). Results also showed sustained ADA gene expression, metabolic correction, and functional immune reconstitution in 48 out of the 50 patients.Discontinuation of immunoglobulin replacement therapy (IgRT) was seen in 26 out of 29 U.S. study patients (90%) who demonstrated sustained engraftment by two years and 19 of 19 U.K. study patients (100%) who had sustained engraftment by three years.Additionally, no deaths, monoclonal expansion

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Orchard Therapeutics reports HSC gene therapy data in rare genetic disorder