AviadoBio Launches Groundbreaking UK Trial
AviadoBio has officially launched its Phase 1/2 ASPIRE-FTD clinical trial in the United Kingdom, marking a significant step forward in the search for a treatment for frontotemporal dementia (FTD) caused by GRN mutations (FTD-GRN).
The investigational gene therapy, AVB-101, is designed to address a devastating, early-onset form of dementia that affects individuals typically under the age of 65.
A New Frontier in Dementia Treatment
The trial, now actively recruiting patients at Cambridge University Hospitals NHS Foundation Trust (CUH), will soon be joined by University College London (UCL).
The Advanced Neurotherapies Centre at Cardiff and Vale University Health Board, one of the first facilities in Europe equipped for MRI-guided infusions, will support both UK and international participants with the delivery of AVB-101.
Frontotemporal dementia is a progressive and life-altering disease, often characterised by personality changes, executive dysfunction, apathy, mobility issues, and loss of language.
Patients with the GRN mutation produce lower levels of progranulin, a protein essential for brain cell health. AVB-101 seeks to restore these levels by delivering a functional GRN gene directly to the brain.
A Precision Approach to Gene Therapy
Unlike traditional therapies, AVB-101 is administered using a minimally invasive neurosurgical technique that bypasses the blood-brain barrier and targets the thalamus – a critical hub for brain connectivity.
This approach not only ensures precise delivery but also allows for lower dosing while limiting systemic exposure.
A neurosurgeon involved in the Cardiff delivery team highlighted the innovation behind the procedure, calling it a major step forward in the search for a treatment in FTD, and expressing hope that this trial could be the beginning of a real-world solution for patients long without effective options.
Meanwhile, the chief medical officer at AviadoBio expressed pride in reaching two major milestones: launching the ASPIRE-FTD trial and treating the first patients with AVB-101, calling it a significant moment in the development of gene therapy for FTD-GRN.
A Global Collaborative Effort
The ASPIRE-FTD trial is part of a wider collaborative landscape. UCL is a participant in the Genetic Frontotemporal Dementia Initiative, which unites clinical and research institutions across Europe and Canada in pursuit of breakthroughs for this challenging condition.
A UCL professor involved in the trial remarked that they are proud to be part of this groundbreaking trial, where the targeted delivery of AAV gene therapy to the thalamus could offer a disease-modifying solution for FTD. It’s a powerful example of science delivering hope.
A Promising Outlook
As the ASPIRE-FTD trial gains momentum across the UK, it represents more than just clinical progress – it offers a new direction for dementia care. With AVB-101, AviadoBio aims not only to slow the progression of FTD-GRN but to transform the standard of care through innovative, brain-targeted gene therapy.
In summary, the launch of the ASPIRE-FTD trial places the UK at the forefront of neurological research, ushering in a potentially transformative era for individuals living with frontotemporal dementia.
Through strategic partnerships, precise technology, and a commitment to restoring hope, AVB-101 could redefine the future of FTD treatment.
News Credits: AviadoBio opens UK trial for FTD gene therapy
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