Professor Brian Bigger

Professor Brian Bigger is the Chair in Advanced Therapeutics at the Institute for Regeneration and Repair at the University of Edinburgh. He retains an Honorary Chair in Cell and Gene Therapy at the University of Manchester. The Bigger lab continues to develop innovative gene and cell therapies for neurological diseases, including lysosomal diseases and bring these treatments to patients.

Brian graduated from Bath University in the UK with a degree in Applied Biology and later a PhD in gene therapy from Imperial College London, with Professor Charles Coutelle. After a further 4 years in London developing a stem cell gene therapy approach for haemophilia B in collaboration with Professor Dominique Bonnet at Cancer Research UK, he subsequently worked on haematopoietic stem cell migration at the National Blood Service and Oxford University also in the UK.
In 2006 Brian set up the Stem Cell & Neurotherapies group at the University of Manchester, collaborating closely with clinical colleagues at the Manchester University NHS Foundation Trust. Brian's group works on pathology, diagnosis and clinical development of treatments for neurological diseases - with a particular focus on lysosomal storage diseases and other neurological disorders.

His group is the first to separate lysosomal storage from neuro-inflammation and show the role of both processes in the pathology of neurological lysosomal disease.

The first therapy developed in the lab (substrate reduction therapy for MPSIII) entered phase III clinical trial in mid 2014.

In 2025, his work has contributed to the first gene therapy treatment for Hunter Syndrome

Brian’s lab has developed three haematopoietic stem cell gene therapies for MPSIIIA, MPSIIIB and a blood brain barrier targeted approach for MPSII to date. The MPSIIIA treatment and later also MPSIIIB was one of the founding programmes of Orchard Therapeutics Plc in 2016 and Brian is also the academic PI on the subsequent phase I/II clinical trial in Manchester, now fully recruited. The MPSII HSC GT treatment and a GMP manufacturing protocol for transduction of hCD34+ cells in collaboration with NHS BT has been completed through funding from the Advanced Therapy Treatment Centre (iMATCH) and AVROBIO and Brian is also academic PI on the clinical trial, that opened in Manchester in early 2023. The team has also developed an AAV gene therapy for MPSIIIC including large animal studies to improve convection enhanced delivery of AAV into the sheep brain.