Gene therapy scid7/1/2023 ![]() Thirty (30) subjects enrolled in the U.S. Results of Phase 1/2 Clinical Studies for ex vivo LVV HSC Gene Therapyįifty patients with ADA-SCID were treated with an investigational gene therapy composed of autologous CD34 + HSCs transduced ex vivo with a self-inactivating LVV encoding the human ADA gene. “We are encouraged by the results we’ve seen across this large dataset of 50 treated patients, and believe they reinforce the promise of the HSC gene therapy approach for treating and potentially curing certain life-threatening genetic diseases.” ![]() “With sustained engraftment of up to three years, these data show the potential of HSC gene therapy to correct the underlying genetic cause of ADA-SCID, delivering positive outcomes in a single treatment,” said Bobby Gaspar, M.D., Ph.D., chief executive officer of Orchard Therapeutics. Without treatment, children born with ADA-SCID typically pass away by 2 years of age. Patients with ADA-SCID suffer from frequent, severe infections as well as non-immune symptoms including those affecting the gastrointestinal, skeletal and nervous systems. “We saw no reports of graft versus host disease, and the ability to discontinue immunoglobulin replacement therapy over time in most patients is also notable for the gene therapy, contributing to its overall benefit-risk profile as a potential treatment for ADA-SCID.”ĪDA-SCID is a rare and life-threatening primary immunodeficiency caused by a genetic mutation that affects white blood cell production. ![]() ![]() “Results from a one-time treatment with experimental lentiviral HSC gene therapy for ADA-SCID are compelling, most notably the overall and event-free survival rates (100% and ≥95%, respectively) observed at two and three years post-treatment,” said Donald Kohn, M.D., distinguished professor of Microbiology, Immunology & Molecular Genetics and Pediatrics at the University of California, Los Angeles (UCLA), member of the UCLA Broad Stem Cell Research Center, director of the UCLA Human Gene and Cell Therapy Program, and co-lead author of the NEJM paper. Additionally, no deaths, monoclonal expansion events, leukoproliferative complications, or emergence of replication-competent lentivirus were observed. study patients (90%) who demonstrated sustained engraftment by two years and 19 out of 19 UK study patients (100%) who had sustained engraftment by three years. Discontinuation of immunoglobulin replacement therapy (IgRT) was seen in 26 out of 29 U.S. Results also showed sustained ADA gene expression, metabolic correction, and functional immune reconstitution in 48 out of the 50 patients. and one in the UK, as well as from a compassionate use program (n=10) in the UK. The data were taken from three Phase 1/2 clinical studies (n=40), two conducted in the U.S. Results showed 100% overall survival and ≥95% event-free survival (defined as survival in the absence of enzyme replacement therapy reinstitution or rescue allogeneic hematopoietic stem cell transplant (HSCT)) at two and three years. Fifty (50) 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. 100% overall survival and ≥ 95% event-free survival observed at two and three years following one-time treatment with lentiviral HSC gene therapyĥ0 total participants represent largest published dataset of gene therapy-treated patients with a monogenic condition to dateīOSTON and LONDON, (GLOBE NEWSWIRE) - Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced data published in the New England Journal of Medicine ( NEJM) evaluating the safety and efficacy of investigational gene therapy products, including OTL-101, for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID).
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