Professor Brian Bigger has been named in TIME’s 2026 Most Influential People in Health list for his work developing brain targeted stem cell gene therapies for childhood dementias. Professor Bigger’s lab, within the Centre for Regenerative Medicine, is developing stem cell gene therapies for childhood dementias and multisystem diseases. They use engineered viruses, or viral vectors*, to deliver stem cell and gene therapies in models of lysosomal storage diseases** through to clinical trials in patients. Professor Bigger’s research has recently been in the news as a clinical trial using brain targeted gene therapy with a special genetically engineered virus called a lentiviral vector, was successful in treating a young boy with Hunter Syndrome**. Following on from the success of this work while he was based at the University of Manchester, Professor Bigger’s work in Edinburgh now looks at potential regenerative therapies for older children, using iPSC, or Induced Pluripotent Stem Cell therapies. These are stem cells that have been created from other cells in the body. He has broadened the scope of conditions to look at other childhood dementias and more common multisystem diseases. It came as a tremendous surprise and honour to receive this recognition from TIME. I am humbled to be included in such an esteemed group of innovators and leaders, who are impacting and advancing the world of health. This recognition really belongs to the hard work of so many colleagues in Manchester and Edinburgh, as well as collaborators, patient organisations and families, scientists and clinicians, academia and industry, working together to make our stem cell gene therapies for childhood dementia a reality. I hope this recognition will mean that we can help more families with these devastating diseases through innovative stem cell and gene therapies for dementia and beyond. Professor Brian Bigger TIME100 Health TIME’s TIME100 Health represents 100 people who are most influential in the world of health right now. Based on months of research, the list comprises a “community of leaders—scientists, doctors, advocates, educators, and policy-makers, among others—who are changing the health of the world.” Brian Bigger Continuing his research career in gene and cell therapies, Brian Bigger set up the Stem Cell and Neuropathies lab at the University of Manchester in 2006. In 2023 he moved to the Institute for Regeneration and Repair at the University of Edinburgh as Professor of Advanced Therapeutics and currently leads a research group developing stem cell gene therapies for childhood dementias and multisystem diseases. The Science * A viral vector is a modified virus designed to deliver genetic material into cells. The types of these that Professor Bigger works with are called lentiviruses and adeno-associated viruses.Lentiviruses and adeno-associated viruses (AAV) use the host cell’s machinery to create copies of themselves. They do this in slightly different ways, but by changing what genes are in the modified virus to begin with, we can insert useful genes into host cells that could, for example, produce a protein that is missing in a certain disease or dampen inflammation in other cases.Professor Bigger’s lab is looking at how both lentiviruses and AAVs can be used as vectors to treat childhood dementias, where patients are missing a key enzyme needed to break down long chain sugars. They use a viral vector to add the missing gene to stem cells they have taken out of a patient. Using conditioning, the old stem cells that were missing the gene are removed and then replaced with the modified ones that now overexpress the necessary gene. Some of these cells are able to cross the blood brain barrier and grow in the brain thus delivering enzyme to the brain. In the case of Hunter Syndrome, the missing enzyme was tagged with a short protein called ApoEII to allow the whole enzyme to cross the blood brain barrier. This allowed for more of the enzyme in the brian where it is much needed to correct brain cells.The patient is then able to continually make the previously missing proteins within their own body, importantly including in the brain.**Hunter Syndrome is a lysosomal storage disorder that is caused by a genetic mutation inherited from parents. In lysosomal storage disorders, lysosomes, sacs of enzymes within the cell, don’t function properly because one of the enzymes doesn’t work. In Hunter Syndrome, the lack of a specific enzyme causes long chain sugars to build-up in body tissues. They can’t be broken down in any other way. This build-up of sugars causes abnormalities in many organs, including the heart, but also in the skeleton, respiratory system and the brain. Professor Bigger's work has led to a clinical trial at the University of Manchester, using a viral vector to replace the missing gene that codes for the specific enzyme, so the patient who received the therapy is now able to produce this enzyme and therefore avoid worsening symptoms. The Bigger lab group is also looking at gene therapy as a treatment for neurodegeneration in multiple diseases. TIME100 Health Bigger Research Group Publication date 11 Feb, 2026
