ASPIRE-FTD Phase 1/2 Clinical Trial Opens in the U.S. at The Ohio State University Wexner Medical Center

July 3, 2024
  • ASPIRE-FTD is now recruiting at The Ohio State University Wexner Medical Center
  • The study is evaluating AVB-101, an investigational gene therapy designed to stop disease progression for people with frontotemporal dementia with GRN mutations

 LONDON July 1, 2024 — AviadoBio, a pioneering gene therapy company dedicated to developing and delivering potentially transformative medicines for neurodegenerative disorders, today announced that its Phase 1/2 ASPIRE-FTD clinical trial is now open and recruiting patients in the United States at The Ohio State University. ASPIRE-FTD is evaluating AviadoBio’s investigational gene therapy, AVB-101, in people with frontotemporal dementia (FTD) with progranulin (GRN) gene mutations (FTD-GRN). The study is also recruiting in Europe with study sites in the Netherlands, Poland, and Spain, with additional sites expected to open in multiple countries. 

FTD is a devastating form of early-onset dementia that typically leads to death within three to 10 years from diagnosis.1,2 People with FTD commonly experience personality changes, behavioral disturbance, loss of language, apathy, and reduced mobility.3 It is a leading cause of dementia in people under the age of 65 and is often misdiagnosed.4 People with FTD who have disease-causing GRN mutations produce a reduced amount of progranulin protein. AVB-101 is a potential one-time therapy designed to stop disease progression by delivering a functional copy of the GRN gene to restore appropriate progranulin levels to affected areas of the brain. 

“People living with FTD-GRN have no available disease-modifying treatments and the impact of this disorder is profound for both patients and their families,” said neurosurgeon Dr. James “Brad” Elder, professor of neurological surgery at The Ohio State University Wexner Medical Center. “There is an urgent need for collaboration between researchers, clinicians, patients, advocates, and families to courageously explore new approaches for FTD, including innovative and targeted delivery approaches. This study will allow us to investigate how highly targeted GRN supplementation could be a potential breakthrough treatment.” 

“Although delivery of progranulin protein via gene therapy is known to be possible, effective brain delivery remains a challenge primarily due to the anatomy of the brain,” said David Cooper, M.D., Chief Medical Officer of AviadoBio. “Our goal is to use intrathalamic delivery to facilitate the biodistribution of PGRN protein to areas of the cortex affected by FTD. The therapy has already shown great promise in preclinical studies, and we now look forward to taking this important next step in its clinical development.” 

 AVB-101 is delivered as a one-time-only treatment using a minimally invasive, stereotactic neurosurgical procedure directly to the part of the brain called the thalamus. The thalamus has extensive connections throughout the brain, including the frontal and temporal lobes, which play a critical role in FTD. This targeted delivery method aims to safely and effectively cross the blood-brain barrier, delivering the investigational gene therapy directly to the brain to restore GRN levels in the frontal and temporal cortex where it is needed most, while at the same time reducing the dose required and the potential systemic exposure. 

“Thousands of people are diagnosed with FTD each year. The relatively early onset of FTD and often strongly hereditary nature can make it all the more challenging for patients and their families,” said Lisa Deschamps, AviadoBio’s Chief Executive Officer. “We are grateful to the patients and families with FTD-GRN involved in ASPIRE-FTD, as well as The Ohio State University researchers for their dedication to investigating AVB-101’s great potential in stopping disease progression and offering hope to patients and their loved ones.” 

More information about the ASPIRE-FTD study can be found at or

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2. Riedl L et al. Neuropsychiatr Dis Treat. 2014;10:297–310
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4. Hendriks S, Peetoom K, Bakker C, et al. Global Prevalence of Young-Onset Dementia: A Systematic Review and Meta-analysis. JAMA Neurol. 2021;78(9):1080–1090. doi:10.1001/jamaneurol.2021.216
5. Hogan DB et al. Can J Neurol Sci. 2016;43 (Suppl 1):S96–S109
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7. Kansal K et al. Dement Geriatr Cogn Disord. 2016;41:109–122
8. Galvin JE et al. Neurology. 89(20):2049–2056
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10. Onyike CU and Diehl-Schmid J. Int Rev Psychiatry. 2013;25(2):130–137
11. Kuang, L., et. al. Frontotemporal dementia non-sense mutation of progranulin rescued by aminoglycosides. Human Molecular Genetics 2020;29(4):624-634

Notes to Editors

ASPIRE-FTD is an open-label, multi-center, Phase 1/2 dose-escalation study designed to evaluate the safety and preliminary efficacy of AVB-101 in patients with FTD-GRN. In the study, patients will receive a single administration of AVB-101 delivered as a one-time infusion into the thalamus via a stereotactic neurosurgical procedure at expert neurosurgical centers throughout Europe and the United States.

An investigational gene therapy, AVB-101 contains a correct (non-mutated) version of the GRN gene. It is designed to restore levels of progranulin in the brain, potentially slowing or stopping the progression of FTD-GRN. AVB-101 will be delivered as a one-time infusion directly into the brain via a minimally invasive surgical procedure, performed by a study neurosurgeon at a specialist neurosurgical center.

FTD is a devastating form of early-onset dementia that typically leads to death within seven to 13 years of symptom onset and three to 10 years from diagnosis.1,2 People with FTD commonly experience personality changes, behavioral disturbance, loss of language, apathy, and reduced mobility.3
FTD is a leading cause of dementia in people under the age of 654 with an estimated prevalence at any one time of up to 4.6 cases per 1,000 people.5 FTD typically strikes younger than Alzheimer’s disease and the majority of FTD cases occur between 45 and 68 years of age.6,7 Given the early onset, FTD can have a substantially greater impact on work, family, and finances than Alzheimer’s disease.8 Genetic FTD cases account for about one-third of cases and are associated with autosomal dominant mutations in three genes, including the GRN (progranulin) gene.9 Approximately 11,000 people in the U.S. and EU5 are living with FTD-GRN with approximately 2,200 new cases per year.10,11 Some FTD cases may be misidentified, and diagnostic delay is common. As disease education, genetics knowledge, and research and treatment options grow, these numbers are expected to increase.

At AviadoBio, we are relentlessly chasing cures by translating groundbreaking science and precision delivery into life-changing medicines for people living with frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). With our deep understanding of the brain and suite of proprietary gene therapy platforms and delivery technologies, AviadoBio is working to overcome the challenges of delivering the right drug to the right place. Its innovative, neuroanatomy-led approach is designed to maximize the therapeutic potential of gene therapy to halt or potentially reverse neurodegenerative diseases. AviadoBio was founded on pioneering research from King’s College London and the UK Dementia Research Institute and has a leadership team with extensive gene therapy development, delivery, and commercialization
experience which uniquely positions the company for success in bringing transformative medicines to patients.
AviadoBio’s investors include New Enterprise Associates (NEA), Monograph Capital, F-Prime Capital, Johnson & Johnson Innovation – JJDC, Inc. (JJDC), SV Health Investor’s Dementia Discovery Fund (DDF), Advent Life Sciences, EQT Life Sciences (Dementia Fund), and LifeArc.
For more information, please visit and follow us on X @AviadoBio and LinkedIn at AviadoBio.