AviadoBio has initiated enrollment for the fourth cohort in its phase-I/II ASPIRE-FTD clinical trial evaluating AVB-101, its investigational gene therapy being developed for people living with frontotemporal dementia with GRN mutations (FTD-GRN).

AVB-101 is a differentiated and precise approach to restoring progranulin in the brain of FTD-GRN patients. AVB-101 is delivered using a minimally invasive, stereotactic neurosurgical procedure directly to the part of the brain called the thalamus. The thalamus has extensive connections to other parts of the brain, including the frontal and temporal lobes, which play a critical role in FTD and the symptoms that impact patients and their families. This targeted delivery method aims to safely and effectively cross the blood-brain barrier, delivering targeted treatment directly to the brain to restore progranulin levels in the frontal and temporal cortex where it is needed the most, while at the same time minimising the dose required and thereby limiting any potential systemic exposure.

To date, the ASPIRE-FTD trial has completed three dose-escalation cohorts with the dosing of 12 patients from five countries across three continents. ASPIRE-FTD now has 20 active trial sites across the US, UK, Canada, and in six European countries.

Early data demonstrate dose-dependent elevations in the key biomarker of cerebrospinal fluid (CSF) progranulin. The overall safety profile of AVB-101 remains favourable with no need for prophylactic or reactive immunosuppression. To date, there have been no serious adverse events related to AVB-101. AviadoBio plans to present additional data at the Alzheimer's Association International Conference (AAIC) meeting in July 2026 and the International Society for Frontotemporal Dementias (ISFTD) meeting in October 2026.

Through the Treat FTD Fund, the company recently received an investment from The Alzheimer’s Drug Discovery Foundation (ADDF) and The Association for Frontotemporal Degeneration (AFTD) to support continued development of AVB-101 in the ASPIRE-FTD clinical trial. The Treat FTD Fund, launched in 2016 by the ADDF and the AFTD, invests in clinical studies aimed at developing novel treatments or biomarkers for FTD disorders.

“We are focused on continuing to advance AVB-101 building on the encouraging data we’ve seen so far. We are thrilled to now have the ADDF and the AFTD’s support on this important mission as we progress the ASPIRE-FTD trial. We are deeply committed to developing new options for patients and families affected by FTD, where significant unmet need remains,” said Lisa Deschamps, Chief Executive Officer, AviadoBio.

The company holds full worldwide rights to develop and commercialise AVB-101 in FTD-GRN and other potential indications, following the expiration of Astellas’ 2024 exclusive option and licence agreement. Astellas remains a shareholder in AviadoBio.