Orchard extends its collaboration with Manchester University to include MPS-IIIB

Orchard Therapeutics, a clinical-stage biotechnology company, has acquired an exclusive licence to develop lentivirus-based autologous ex-vivo gene therapy for Sanfilippo syndrome type B (or MPS-IIIB) from The University of Manchester.

This new licence represents an extension of the current collaboration between Orchard, The University of Manchester and Manchester University NHS Foundation Trust in MPS-IIIA. Autologous ex-vivo lentiviral haematopoietic stem cell gene therapy is anticipated to correct neurological manifestations through the engraftment of subpopulations of haematopoietic stem cells in the central nervous system, thereby providing supranormal and widespread enzyme expression throughout the brain.

“The incorporation of MPS-IIIB into our development pipeline is a significant milestone in the consolidation of our neurometabolic franchise, which is currently focused on the development of autologous ex-vivo haematopoietic stem-cell gene therapy for children suffering from MPS-IIIA,” stated Dr Jesus Garcia-Segovia, Orchard’s VP Clinical Development, CNS and Metabolic Disorders. “We are very excited at the possibility of bringing effective treatments capable of addressing the high unmet medical need in children suffering from these devastating conditions.”

Prof. Brian Bigger, professor of Cell and Gene Therapy in the Faculty of Biology, Medicine and Health, The University of Manchester commented: “It’s incredibly exciting for us to work with our trusted partner Orchard Therapeutics to translate another autologous ex-vivo gene therapy that has demonstrated efficacy in a preclinical mouse model of MPS-IIIB into clinical development and scale-up.”

“MPS-IIIA and MPS-IIIB are devastating diseases. Orchard and its collaborators are highly motivated to develop gene therapies to address the root cause of these disorders and will work tirelessly to make treatments available to patients as soon as possible,” added Dr Andrea Spezzi, Orchard’s chief medical officer. “We are now focussing all our efforts on completing the preclinical activities required to enable the start of clinical studies in MPS-IIIA towards the end of 2018 and thereafter in MPS-IIIB.”

The technology, developed in Professor Brian Bigger’s laboratory, and recently published in the journal Brain, involves the use of a high-titre lentiviral vector to drive the expression of a codon-optimized α-N-acetylglucosaminidase (NAGLU) gene under the control of the myeloid-specific CD11b promoter (LV.CD11b.NAGLU).