Biopharmaceutical company AviadoBio announced on July 1 that its ASPIRE-FTD phase 1/2 clinical trial has opened its first trial site in the United States, at Ohio State University’s Wexner Medical Center. The company is evaluating the experimental gene therapy AVB-101 as an intervention for FTD caused by a variant of the GRN gene.

The GRN gene carries instructions for the progranulin protein, which plays a crucial role in cell survival and in regulating inflammation. FTD-GRN is caused by the genetic variant inhibiting progranulin production, which leads to the abnormal accumulation of a protein (such as TDP-43) in the brain. The gene therapy AVB-101 aims to provide a functional GRN gene copy to restore progranulin production.

J. Bradley Elder, MD, professor of neurological surgery at Wexner, discusses the importance of the clinical trial:

“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. 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. AVB-101 is a one-time treatment delivered through minimally invasive surgery that allows gene therapy to bypass the protective blood-brain barrier safely. The procedure provides the treatment directly to the thalamus, a crucial structure in the brain’s center that has extensive connections to neighboring areas.”

In November 2023, the US Food and Drug Administration cleared ASPIRE-FTD to open US-based sites, approving the company’s Investigational New Drug Application for AVB-101. In April 2024, AviadoBio announced that the first European participant was dosed with AVB-101. ASPIRE-FTD’s site at Wexner Medical Center is now recruiting participants who carry a risk-creating GRN variant.

AviadoBio CEO Lisa Deschamps is enthusiastic about the trial. “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,” she says.

To learn more about the clinical trial, visit ASPIRE-FTD’s website or the trial’s ClinicalTrials.gov page.

The participation of people with FTD, care partners, and family members in studies like ASPIRE-FTD is critical in driving research toward a cure. Learn more about participation opportunities on our Studies Seeking Participants page. If you have questions about joining a study, contact AFTD’s HelpLine at 1-866-507-7222 or info@theaftd.org.