Gene therapy company AviadoBio announced the completion of the second cohort of its phase 1/2 clinical trial of a potential genetic FTD treatment at the 2025 AFTD Annual Education Conference in May. The trial is currently recruiting participants in the U.S. and Europe for a third cohort.

Earlier Studies Show AVB-101 Is Well Tolerated in People with FTD-GRN

In FTD caused by inherited mutations of the GRN gene, production of the neuroprotective protein progranulin is impaired. Among other things, this can lead to the abnormal accumulation of the protein TDP-43 and, subsequently, neurodegeneration.

As explained by AviadoBio’s chief medical officer David Cooper, MD, in a 2024 interview with Neurology Live, the goal of the ASPIRE-FTD clinical trial is to restore progranulin production in the brain, hopefully slowing or stopping FTD-GRN. Administered through a one-time surgical procedure, the experimental gene therapy AVB-101 reintroduces a working copy of GRN to kickstart progranulin production. The surgery delivers the working gene directly to the thalamus, bypassing the protective blood-brain barrier that might otherwise halt AVB-101’s progress.

In the first cohort of the current trial, researchers found that AVB-101 was generally well-tolerated with no serious adverse events.

At AFTD’s 2025 Annual Education Conference in Colorado, AviadoBio announced that the second cohort had been dosed. Additionally, the company noted that it expected to publish early biomarker data in 2026.

“Dosing our sixth patient with AVB-101 marks an important and encouraging step forward in our research pathway,” said Dr. Cooper. “In the first cohort, we did not see any clinically significant safety findings through follow-up of up to 52 weeks and did not require any immunosuppression prophylactically or reactively.”

AviadoBio also announced that it intends to begin dosing a third cohort later this year, with recruitment underway for trial sites in the U.S., U.K.,  Spain, Poland, Sweden, and Netherlands. The trial is recruiting people between 30-75 years old who carry a pathogenic GRN mutation and who show signs of either behavioral variant FTD or primary progressive aphasia.

For more information on ASPIRE-FTD, visit the clinical trial’s webpage or clinicaltrials.gov page.

Are you interested in participating in clinical trials like ASPIRE-FTD? Signing up with the FTD Disorders Registry can help you keep up to date on participation opportunities and provide you with a platform to share your lived experiences to guide researchers.