Dialog Box


Stem Cell Therapy

University of Manchester & Orchard Therapeutic's MPSIII Stem Cell Program (UK) / Sanfilippo Type A

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 biotechnolgy company Orchard Therapeutics to bring its stem cell gene therapy program to human clinical trial.  


The technique works by genetically  correcting the patients own stem cells and implanting them via a bone marrow transplant to release the missing enzyme which travels to the brain.  The new study will take place at CMFT (Central Manchester Foundation Trust), supported by the NIHR/Wellcome Trust Manchester Clinical Research Facility.


For more information, visit:

Orchard Therapeutics announces manufacturing alliance with PharmaCell B.V

Manchester Univesity Press Release  

United Press international: Stem cell gene therapy to be tested in humans 

Manchester University Sanfilippo Announcement