Gene therapy trial starts in early-onset dementia

MRI of the brain

AviadoBio has launched its Phase I/II ASPIRE-FTD clinical trial at the Ohio State University in the US.

ASPIRE-FTD is evaluating 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 form of early-onset dementia that typically leads to death within three to 10 years from diagnosis.

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 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.”

Targeted, minimally-invasive delivery

AVB-101 is delivered as a one-time-only treatment using a minimally invasive, stereotactic neurosurgical procedure directly to the thalamus.

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.

“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, Chief Medical Officer of AviadoBio. “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.”

Diana Spencer, Senior Digital Content Editor, DDW

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