– AVB-101 is an investigational one-time therapy designed to deliver a functional copy of the GRN gene directly to the brain, thereby potentially restoring progranulin levels and stopping disease progression in patients with frontotemporal dementia with GRN mutations (FTD-GRN)
– FTD is a leading cause of dementia in those <65 years old
AviadoBio, a pioneering gene therapy company dedicated to developing and delivering potentially transformative medicines for neurodegenerative disorders, today announced that its Phase 1/2 ASPIRE-FTD clinical trial is now open in the UK. The trial is evaluating AVB-101, an investigational gene therapy, in people with frontotemporal dementia (FTD) with GRN gene mutations (FTD-GRN). Cambridge University Hospitals NHS Foundation Trust (CUH), which hosts an internationally renowned center of excellence in providing support and care for families affected by FTD, is now recruiting patients. University College London (UCL) is also expected to open for patient recruitment shortly. The Advanced Neurotherapies Centre at Cardiff and Vale University Health Board, one of the first surgical centers in Europe currently able to perform MRI-guided infusions of gene therapies, will provide the capability to dose clinical trial participants from the UK and beyond in the ASPIRE-FTD study.
FTD is a devastating form of early-onset dementia that varies in each individual. Symptoms of FTD can include changes in personality or uncharacteristic behaviors, progressive loss of language, loss of executive function and cognitive abilities, apathy, and reduced mobility.1-3 People with FTD who have disease-causing GRN mutations produce a reduced amount of progranulin protein. AVB-101 is an investigational one-time therapy designed to deliver a functional copy of the GRN gene directly to the brain, thereby potentially restoring progranulin levels and stopping disease progression in patients with FTD-GRN.
“Launching ASPIRE-FTD and treating our first patients with AVB-101 have been significant milestones in FTD-GRN research and gene therapy development,” said David Cooper, M.D., Chief Medical Officer of AviadoBio. “AviadoBio was built on pioneering research from King’s College London and the UK Dementia Research Institute. The opening of our UK clinical trial sites reflects this strong research heritage, and we’re proud to bring this clinical trial to the UK to make it more accessible to people living with familial FTD in the region.”
“We are delighted to be able to surgically deliver AVB-101 in the ASPIRE-FTD trial in Cardiff, offering hope to patients living with FTD in the UK and beyond. The Advanced Neurotherapies Centre is funded by HealthCare and Research Wales and is currently the only center in the UK, and one of two European centers, conducting these groundbreaking first in human clinical trials, delivering advanced gene and cell therapies directly to the brain for neurodegenerative diseases. This trial represents a major step forward in the search for a treatment in FTD, potentially bringing a new therapy to reality for patients,” commented Professor William Gray, Local PI and Neurosurgeon at Cardiff University and Cardiff and Vale University Health Board.
“The potential to halt FTD-GRN with a single procedure could be truly transformative for patients and their families,” said Professor James Rowe, Consultant Neurologist at Addenbrooke’s Hospital and Professor of Cognitive Neurology at the Cambridge Centre for Frontotemporal Dementia.“Combining excellence in clinical care with cutting-edge research is vital to driving progress in this extremely challenging field. Through studies like this we hope to one day be able to provide a potential cure for FTD.”
“UCL is a leader in the Genetic Frontotemporal dementia Initiative, a group of research centers across Europe and Canada with expertise in familial frontotemporal dementia. We are proud to be included in this groundbreaking trial where targeted delivery of a single low dose of AAV gene therapy to the thalamus could be potentially disease-modifying for FTD,” said Jonathan Rohrer, Professor of Neurology at the UCL Dementia Research Centre.
AVB-101 is delivered as a one-time-only treatment using a minimally invasive stereotactic neurosurgical procedure directly to the part of the brain called the thalamus. The thalamus is a key hub for connectivity in the brain with widespread projections across the brain including the cortex – a key area affected in FTD-GRN. The neurosurgery procedure to deliver AVB-101 bypasses the blood-brain barrier, thereby limiting it to only the brain itself, where it is needed the most. At the same time, this potentially reduces the amount of dose required and systemic exposure in other parts of the body.
More information about the ASPIRE-FTD study can be found at https://clinicaltrials.gov/study/NCT06064890.
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