ASPIRE-FTD Phase 1/2 Clinical Trial Begins at The Ohio State University Wexner Medical Center

Tuesday, July 02, 2024

AviadoBio, a leading gene therapy company specializing in treatments for neurodegenerative disorders, has initiated its Phase 1/2 ASPIRE-FTD clinical trial at The Ohio State University in the United States.

This trial aims to evaluate AVB-101, an investigational gene therapy, in individuals suffering from frontotemporal dementia (FTD) caused by progranulin (GRN) gene mutations (FTD-GRN).

Recruitment is also underway in Europe, with study sites in the Netherlands, Poland, and Spain, and plans for additional global sites.

FTD is a severe form of early-onset dementia that often leads to death within three to 10 years of diagnosis. It is characterized by personality changes, behavioral disturbances, language loss, apathy, and decreased mobility.

FTD is a leading cause of dementia in people under 65 and is frequently misdiagnosed. Individuals with FTD-GRN mutations produce insufficient progranulin protein, which AVB-101 aims to address by delivering a functional GRN gene copy to restore progranulin levels in affected brain regions.

A neurosurgeon and professor at The Ohio State University Wexner Medical Center, emphasized the urgent need for innovative treatments for FTD-GRN. AVB-101 uses a targeted delivery approach through intrathalamic administration, utilizing the extensive brain connections of the thalamus to distribute progranulin protein to the cortex.

This minimally invasive procedure aims to effectively cross the blood-brain barrier, optimizing therapy delivery while minimizing systemic exposure.

The therapy's promising preclinical results and the pivotal role of the ASPIRE-FTD trial in advancing clinical development.

FTD-GRN patients, families, and researchers for their commitment to exploring AVB-101's potential in halting disease progression and offering hope to affected individuals and their loved ones.


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