AviadoBio opens UK trial for FTD gene therapy

Aspire-FTD study aims to stop disease progression with one-time treatment

AviadoBio has launched its phase 1/2 Aspire-FTD trial in the UK, assessing AVB-101, a gene therapy targeting frontotemporal dementia (FTD) with GRN mutations (FTD-GRN). Cambridge University Hospitals NHS Foundation Trust (CUH) has begun recruiting patients, with University College London (UCL) expected to follow.

The Advanced Neurotherapies Centre at Cardiff and Vale University Health Board will provide MRI-guided infusions for UK and international trial participants. The center is one of the first in Europe with this capability.

FTD is a severe early-onset dementia affecting individuals under 65. It can cause personality changes, loss of language, executive dysfunction, apathy, and mobility issues. People with FTD-GRN produce less progranulin protein, contributing to disease progression. AVB-101 aims to restore progranulin levels by delivering a functional copy of the GRN gene directly to the brain.

David Cooper, chief medical officer at AviadoBio, said: “Launching ASPIRE-FTD and treating our first patients with AVB-101 have been significant milestones in FTD-GRN research and gene therapy development.”

Professor William Gray, neurosurgeon at Cardiff University, commented: “This trial represents a major step forward in the search for a treatment in FTD, potentially bringing a new therapy to reality for patients.”

AVB-101 is administered via a minimally invasive neurosurgical procedure, bypassing the blood-brain barrier. Targeting the thalamus, a key connectivity hub, may reduce systemic exposure and allow a lower dose.

UCL is part of the Genetic Frontotemporal Dementia Initiative, a collaboration across Europe and Canada. Professor Jonathan Rohrer noted: “We are proud to be included in this groundbreaking trial where targeted delivery of AAV gene therapy to the thalamus could be potentially disease-modifying for FTD.”