Stem Cell Therapy
University of Manchester/Orchard Therapeutic's Stem Cell Program (UK) / Sanfilippo Types 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 (SGSH for Type A or NAGLU for Type B). These cells are then transplanted back into the body.
Orchard Therapeutics is now completing the preclinical activities required to enable the start of clinical studies in Sanfilippo Type A towards the end of 2018.
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 B, but this is further from starting a clinical trial.
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