Orchard Therapeutics announced it will present two-year follow-up data in 20 patients from the registrational trial evaluating OTL-101, an autologous, ex vivo, hematopoietic stem cell gene therapy for the treatment of severe combined immune deficiency due to adenosine deaminase deficiency, or ADA-SCID, at the Transplantation and Cellular Therapy Meetings of ASBMT and CIBMTR. The reported data are the complete 24 month results from a trial evaluating the safety and efficacy of OTL-101. OTL-101 was administered post-busulfan conditioning in 20 pediatric patients, who lacked a medically eligible donor for bone marrow transplantation. Patients were followed for 24 months post treatment and compared with a historical control cohort of 26 patients with ADA-SCID who underwent hematopoietic stem cell transplant, or HSCT, 12 from matched related donors, or MRD, and 14 without a MRD. The median age at treatment for patients receiving OTL-101 was 9 months and 7.3 months for patients treated with HSCT. Treatment with OTL-101 resulted in 100% overall survival and 100% event free survival at 24 months. Genetically modified cells, as indicated by increasing and then sustained vector copy number in both peripheral blood mononuclear cells and granulocytes, were detectable in all patients treated with OTL-101 and were maintained through 24 months post-treatment. A similar pattern was observed in ADA enzyme activity. Evidence of immune reconstitution was observed in patients treated with OTL-101. Treatment with OTL-101 was well-tolerated and had a positive benefit-risk profile. There were no deaths or reports of graft-versus-host disease in the patients treated with OTL-101. Nine out of the 20 patients who received OTL-101 experienced a total of 27 serious adverse events.
https://thefly.com/landingPageNews.php?id=2868727
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