AviadoBio Advances AVB-101 Gene Therapy Program and Initiates Fourth Dose-Escalation Cohort of Phase 1/2 ASPIRE-FTD Trial
AviadoBio, a pioneering gene therapy company dedicated to developing and delivering potentially transformative medicines for neurodegenerative disorders, today announced that the company has initiated enrollment for the fourth cohort in its Phase 1/2 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 most, while at the same time minimizing 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 United States, United Kingdom, 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 favorable 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, AviadoBio 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,” said Lisa Deschamps, Chief Executive Officer, AviadoBio. “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.”
AviadoBio holds full worldwide rights to develop and commercialize AVB-101 in FTD-GRN and other potential indications, following the expiration of Astellas’ 2024 exclusive option and license agreement. Astellas remains a shareholder in AviadoBio.
About AviadoBio
AviadoBio is relentlessly chasing cures by developing and translating groundbreaking science and precision delivery into life-changing medicines across neurodegenerative diseases. Powered by a precision approach built on three integrated levels — payload, target and delivery — the company is advancing a pipeline of targeted, one-time AAV gene therapies across frontotemporal dementia with GRN mutations (FTD-GRN), retinitis pigmentosa (RP) and other retinal dystrophies, as well as Alzheimer’s disease and related tauopathies.
Founded in 2019 from pioneering research at King’s College London and the UK Dementia Research Institute, AviadoBio combines deep neuroscience expertise with proven biopharma leadership. Its capabilities include gene supplementation, optogenetics, and its proprietary vMiX™ RNA gene-silencing platform, enabling durable, disease-modifying approaches across multiple degenerative conditions. Headquartered in London, the company operates across the UK and the U.S. and is backed by leading global life sciences investors and strategic partners.
For more information, visit aviadobio.com and follow AviadoBio on X and LinkedIn.
About ASPIRE-FTD
ASPIRE-FTD is a Phase 1/2 open-label, multi-center study designed to evaluate the safety and preliminary efficacy of AVB-101 in patients with FTD-GRN. In the study, eligible patients receive a one-time administration of AVB-101 delivered as a set of MRI-guided infusions into the thalamus during a minimally invasive stereotactic neurosurgical procedure at an expert neurosurgical center. The ASPIRE-FTD clinical trial is currently open at trial sites in Belgium, Canada, Italy, the Netherlands, Poland, Spain, Sweden, the United Kingdom, and the United States.
More information about the ASPIRE-FTD study can be found at www.aspire-ftd.com or https://clinicaltrials.gov/study/NCT06064890.
About The Alzheimer’s Drug Discovery Foundation (ADDF)
Founded in 1998 by Leonard A. and Ronald S. Lauder, the Alzheimer’s Drug Discovery Foundation is dedicated to rapidly accelerating the development of drugs to prevent, treat, and cure Alzheimer’s disease. The ADDF is the only public charity solely focused on funding the development of drugs for Alzheimer’s, employing a venture philanthropy model to support research in academia and the biotech industry. The ADDF’s leadership and contributions to the field have played a pivotal role in bringing the first Alzheimer’s PET scan (Amyvid®) and blood test (PrecivityAD®) to market, as well as fueling the current robust and diverse drug pipeline. Through the generosity of its donors, the ADDF has awarded nearly $400 million to fund 792 Alzheimer’s drug development, biomarker, and prevention programs in 21 countries. To learn more, please visit: http://www.alzdiscovery.org/.
About The Association for Frontotemporal Degeneration (AFTD)
Online at theaftd.org, AFTD is the largest national nonprofit devoted to providing resources to help families affected by FTD today, and advancing research to foster accurate diagnosis, treatments, and a cure. Our volunteer-founded organization – driven by thousands of volunteers and donors – reflects a community’s profound determination to #endFTD.
View source version on businesswire.com: https://www.businesswire.com/news/home/20260330432799/en/
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Abstract
AviadoBio Advances AVB-101 Gene Therapy Program and Initiates Fourth Dose-Escalation Cohort of Phase 1/2 ASPIRE-FTD Trial
