AviadoBio’s ASPIRE-FTD AAV Gene Therapy Trial Opens in the UK for Frontotemporal Dementia

LONDON – May 15, 2025   AviadoBio, an AAV-focused gene therapy company dedicated to developing and delivering potentially transformative medicines for neurodegenerative disorders, has announced the opening of its Phase 1/2 ASPIRE-FTD clinical trial in the UK. This trial will evaluate AVB-101, an investigational AAV gene therapy, in individuals with frontotemporal dementia (FTD) who have GRN gene mutations (FTD-GRN). The AAV vector in AVB-101 is designed for targeted gene delivery to the brain.

Cambridge University Hospitals NHS Foundation Trust (CUH), recognized for its expertise in FTD care and support, is now actively recruiting patients for the ASPIRE-FTD trial utilizing this AAV-based therapeutic. University College London (UCL) is also expected to begin patient recruitment shortly for the study involving this specific AAV gene therapy. The Advanced Neurotherapies Centre at Cardiff and Vale University Health Board, a leading European center for MRI-guided AAV gene therapy infusions, will provide the capability to dose participants in the ASPIRE-FTD study with this AAV candidate from the UK and other regions.

FTD is a severe form of early-onset dementia with varied symptoms. In individuals with FTD-GRN, a mutation leads to reduced production of progranulin protein. AVB-101 is a single-administration AAV gene therapy designed to deliver a functional copy of the GRN gene directly to the brain. The goal of this AAV-mediated gene transfer is to restore progranulin levels and potentially halt the progression of the disease in patients with FTD-GRN.

Dr. David Cooper, Chief Medical Officer of AviadoBio, stated that launching ASPIRE-FTD and treating the first patients with AVB-101, their lead AAV product, are significant milestones in FTD-GRN research and AAV gene therapy development. He highlighted AviadoBio’s UK research heritage and expressed pride in bringing this clinical trial of their AAV therapeutic to the UK to improve accessibility for individuals with familial FTD in the region.

Professor William Gray, Local PI and Neurosurgeon at Cardiff University and Cardiff and Vale University Health Board, commented on the importance of being able to surgically deliver AVB-101, the AAV gene therapy, in Cardiff, offering hope to FTD patients in the UK and beyond. He noted that the Advanced Neurotherapies Centre is a leading center for first-in-human clinical trials involving direct brain delivery of advanced AAV gene and cell therapies for neurodegenerative diseases, making this trial of an AAV product a major step forward in the search for an FTD treatment.

Professor James Rowe, Consultant Neurologist at Addenbrooke’s Hospital and Professor of Cognitive Neurology at the Cambridge Centre for Frontotemporal Dementia, emphasized the transformative potential of halting FTD-GRN with a single procedure utilizing this AAV gene therapy for patients and their families. He stressed the importance of combining clinical excellence with research to drive progress in this challenging field, with the hope that studies like this AAV trial will lead to a potential cure for FTD.

Professor Jonathan Rohrer of the UCL Dementia Research Centre highlighted UCL’s leadership in the Genetic Frontotemporal dementia Initiative and expressed pride in participating in this trial of an AAV-delivered therapy. He noted the potential of targeted delivery of a single low dose of AAV gene therapy to the thalamus to be disease-modifying for FTD.

AVB-101, an AAV-based therapeutic, is administered as a one-time treatment via a minimally invasive stereotactic neurosurgical procedure directly into the thalamus, a key connectivity hub in the brain with widespread projections to the cortex, an area significantly affected in FTD-GRN. This direct delivery method of the AAV vector bypasses the blood-brain barrier, limiting the therapy’s exposure primarily to the brain, where it is most needed for effective gene transfer by the AAV particles. This approach also has the potential to reduce the required dose of the AAV vector and systemic exposure in other parts of the body.