• Honorary Professor of Cell and Gene Therapy
• Professor of Advanced Therapeutics, University of Edinburgh
• Past Chairman (2015-2023), European Study Group on Lysosomal Diseases

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 four 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.

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 was 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.

In 2023 Brian moved to the University of Edinburgh to take up a Chair in Advanced Therapeutics at the Institute for Regeneration and Repair. The group continues to develop innovative gene and cell therapies, especially in neurological lysosomal diseases, and bring these treatments to patients. In particular, the focus is on making novel stem cell gene therapies available to a wider group of patients with neurological diseases and dementias. 

Brian retains an Honorary Professorship in Cell and Gene Therapy at the University of Manchester, where he continues as an academic principal investigator on two gene therapy trials for neurodegenerative metabolic diseases.

Neurodegenerative lysosomal storage diseases mainly affect children and often lack effective treatments. Over several years of preclinical work, we developed several different treatments and gene therapies including haematopoetic stem cell gene therapies (HSCGT) for devasting mucopolysaccharidoses (MPS) diseases which result in childhood dementia. In close collaboration with clinical colleagues at Manchester University NHS Foundation Trust (MFT), we are now running first-in-human phase I/II trials of these therapies in MPSIIIA (Sanfilippo disease) and MPSII (Hunter syndrome).

Please note that the research lab is now at the University of Edinburgh.

Bigger Lab, Institute for Regeneration and Repair, University of Edinburgh

Awards

News

New hope for children with devastating rare genetic disorder, thanks to world-first research in Manchester

Early results of gene therapy trial for ‘childhood dementia’ show promise

Groundbreaking gene therapy trial for Hunter syndrome opens

Media

• BBC News First in Human Trail of IDS-ApoEII – Ollie’s story
• Living with rare disease Hunter syndrome | Action Medical Research -Danny’s story
• Lysosomal Disease Group Patient Impact Study and video
• Documentary - La Caixa and Xavi Hernandez - Raising awareness on gene therapy for Sanfilippo Disease -Spanish

Group Members and Affiliated Staff

• Dr Rebecca Holley - Senior Trial Manager
• Rebecca Holley – Trial Project Manager / Support Officer
• Alexandra Haig – Trial Project Manager
• Dr Shaun Wood – Honorary Research Fellow

American Society of Gene & Cell Therapy

The European Society of Gene & Cell Therapy

The British Society for Gene & Cell Therapy

The European Study Group on Lysosomal Diseases

Funders

Ongoing clinical trials are funded by Orchard Therapeutics, LifeArc and the University of Manchester.

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