ADA-SCID is a rare, life-threatening, inherited disease caused by mutations in the ADA gene. Patients with ADA-SCID are unable to fight off and frequently succumb to complications from bacterial, viral and fungal infections.
“With some patients in this trial approaching 5 years of follow-up, treatment with OTL-101 continues to be well-tolerated with higher rates of overall and event free survival for patients with ADA-SCID compared to a historical control group of patients receiving hematopoietic stem cell transplants,” said Donald B. Kohn, M.D., professor of Microbiology, Immunology & Molecular Genetics at the University of California, Los Angeles and the principal investigator of the study. “We are very encouraged by the results and look forward to advancing this potentially transformative treatment option for patients with this serious and life-threatening condition.”
Bobby Gaspar, M.D., Ph.D., chief scientific officer of Orchard commented, “These results demonstrate that by engrafting autologous, gene-modified, long-term repopulating hematopoietic stem cells, we are able to see durable recovery of the immune system. With 100% overall survival and 100% event free survival in this trial maintained at 24 months, we believe OTL-101 has the potential to enable patients with ADA-SCID to lead healthier lives with restored immunity to fight infections.”
Andrea Spezzi, M.D., chief medical officer of Orchard continued,“For the remainder of 2019, we are focused on completing the clinical and manufacturing activities to enable a BLA filing in 2020, bringing us closer to our goal of providing patients with a new treatment option.”
You can read the full press release on the Orchard Therapeutics website.