Stem Cell Therapy
University of Manchester/Orchard Therapeutic's MPSIII Stem Cell Program (UK) / Sanfilippo Type A and B
Dr Brian Bigger from The University of Manchester has used stem cell gene therapy to treat Sanfilippo in mice. The University of Manchester has now signed a licensing agreement with a UK-based biotechnology company Orchard Therapeutics to bring its stem cell gene therapy program to human clinical trial.
The technique is known as "autologous ex-vivo gene therapy". It works by taking the patient's own stem cells (from the blood or bone marrow) and treating them with a virus to deliver a healthy copy of the faulty gene (the SGSH for Type A or NAGLU for Type B). These cells are then transplanted back into the body.
The Type A Clinical trial will take place at CMFT (Central Manchester Foundation Trust), supported by the NIHR/Wellcome Trust Manchester Clinical Research Facility.
Orchard Therapeutics announced in November 2017 that it acquired an exclusive license from the University of Manchester to develop a similar therapy for Sanfilippo Syndrome Type, but this is further from starting a clinical trial.
For more information, visit: