February 25, 2019 : Orchard Therapeutics presents clinical proof-of-concept data for OTL-102, a gene therapy for the treatment of X-CGD. Patients with this rare immune inherited immunodeficiency disorder are prone to recurrent severe infections and complications, leading to frequent hospitalizations, significant morbidity and early mortality.
This ex vivo, autologous, hematopoietic stem cell based gene therapy, originally developed by Genethon (Evry, France), AFM-Téléthon’s laboratory, at a preclinical and clinical stage, utilizes a self-inactivating lentiviral vector (G1XCGD). The patients’ blood cells are collected, corrected ex vivo with the lentiviral vector before being given back to them.
The safety and efficacy of OTL-102, developped by Orchard Therapeutics, was assessed in seven evaluable patients (aged 2-27 years) with X-CGD.
“The proof-of-concept data set for OTL-102 demonstrates compelling efficacy in patients with X-CGD,” said Dr. Kohn, professor of Microbiology, Immunology & Molecular Genetics at the University of California, Los Angeles. “Of note, patients have shown sustained levels of functioning neutrophils associated with clinical benefit, freedom from infections and resolution of chronic inflammation. We look forward to continuing the development of this potentially transformative gene therapy for patients with X-CGD.”
Andrea Spezzi, M.D., chief medical officer of Orchard commented, “Today’s data, the first demonstration of hematopoietic stem cell gene therapy used to correct X-CGD, indicate OTL-102 has the potential to be a transformative new treatment option for these patients. In order to bring this new treatment to patients as rapidly as possible, we are in the process of designing a registrational trial and intend to seek regulatory input this year on the clinical development path forward.”