March 31, 2026-
AviadoBio has announced the initiation of patient enrollment for the fourth cohort in its Phase 1/2 ASPIRE-FTD clinical trial, evaluating AVB-101, an investigational AAV-based gene therapy for patients with frontotemporal dementia caused by progranulin (GRN) mutations (FTD-GRN).
The ASPIRE-FTD study is an open-label, multi-center trial designed to evaluate the safety, tolerability, and preliminary efficacy of a single administration of AVB-101. The therapy is delivered using a minimally invasive stereotactic neurosurgical procedure, in which MRI-guided infusions target the thalamus, a key brain region with extensive connections to the frontal and temporal lobes—areas heavily affected in FTD.
AVB-101 is designed to restore progranulin expression in the brain, addressing the underlying genetic cause of FTD-GRN. By delivering the therapeutic gene directly into the thalamus, the approach aims to efficiently distribute progranulin throughout critical neural networks, while minimizing systemic exposure and reducing the overall dose required.
To date, the ASPIRE-FTD trial has completed three dose-escalation cohorts, with 12 patients treated across five countries on three continents. The study now includes 20 active clinical trial sites spanning the United States, United Kingdom, Canada, and several European countries.
Early clinical data have shown dose-dependent increases in cerebrospinal fluid (CSF) progranulin, an important biomarker for therapeutic activity in FTD-GRN. The therapy has also demonstrated a favorable safety profile, with no serious adverse events related to AVB-101 reported to date and no need for prophylactic or reactive immunosuppression.
Additional clinical data from the ASPIRE-FTD trial are expected to be presented at upcoming scientific meetings, including the Alzheimer’s Association International Conference (AAIC) in July 2026 and the International Society for Frontotemporal Dementias (ISFTD) meeting in October 2026.
Supporting the continued development of AVB-101, AviadoBio has also secured a strategic investment through the Treat FTD Fund, backed by the Alzheimer’s Drug Discovery Foundation (ADDF) and the Association for Frontotemporal Degeneration (AFTD). The fund focuses on advancing clinical research aimed at developing new treatments and biomarkers for FTD.
In addition, AviadoBio has regained full global rights to the AVB-101 program, following the expiration of Astellas’ exclusive option and license agreement in 2024. Astellas remains a shareholder in the company.
Frontotemporal dementia is a progressive neurodegenerative disease that primarily affects the frontal and temporal regions of the brain, leading to behavioral, cognitive, and language impairments. Currently, there are no disease-modifying treatments available, highlighting the urgent need for new therapeutic strategies.
If successful, AVB-101 could represent a promising AAV-based gene therapy approach for neurodegenerative disease, aiming to restore progranulin levels and potentially slow or modify disease progression in patients with FTD-GRN.
