ASPIRE-FTD Phase 1/2 Clinical Trial Launches at Ohio State's Wexner Medical Center

AviadoBio, a leading gene therapy company focused on neurodegenerative diseases, has announced that its ASPIRE-FTD clinical trial is now open for recruitment at The Ohio State University Wexner Medical Center in the United States. This Phase 1/2 trial is designed to evaluate AVB-101, an investigational gene therapy aimed at halting the progression of frontotemporal dementia (FTD) in people with progranulin (GRN) gene mutations (FTD-GRN). The study is also ongoing in European countries, including the Netherlands, Poland, and Spain, with plans to expand to additional locations.

Frontotemporal dementia is a severe form of early-onset dementia often resulting in death within three to ten years post-diagnosis. This condition leads to personality changes, behavioral issues, language loss, apathy, and reduced mobility. It is a leading cause of dementia in individuals under 65 and is frequently misdiagnosed. FTD-GRN patients produce insufficient progranulin protein due to GRN mutations. AVB-101 aims to address this by delivering a functional copy of the GRN gene to restore proper progranulin levels in the brain.

Neurosurgeon Dr. James “Brad” Elder from The Ohio State University emphasized the urgent need for new treatment options for FTD-GRN, highlighting the importance of collaboration among researchers, clinicians, patients, and their families. He noted that the trial’s targeted delivery of GRN supplementation could potentially revolutionize treatment for this disorder.

David Cooper, M.D., Chief Medical Officer of AviadoBio, discussed the challenges of progranulin delivery to the brain due to its complex anatomy. He explained that the therapy would be administered intrathalamically to ensure the PGRN protein reaches the brain’s cortex areas affected by FTD. Previous preclinical studies have shown promising results, and the clinical trial aims to build on this foundation.

AVB-101 is administered as a one-time treatment through a minimally invasive stereotactic neurosurgical procedure targeting the thalamus. The thalamus is essential for its extensive brain connections, including the frontal and temporal lobes, which are crucial in FTD. This delivery method aims to cross the blood-brain barrier effectively, delivering the gene therapy where it is most needed while minimizing systemic exposure.

Lisa Deschamps, CEO of AviadoBio, acknowledged the significant impact of FTD’s early onset and hereditary nature on patients and their families. She expressed gratitude to the patients and families participating in the ASPIRE-FTD trial, as well as the dedicated researchers at The Ohio State University for their commitment to exploring AVB-101’s potential.

The ASPIRE-FTD study is an open-label, multi-center, Phase 1/2 dose-escalation trial to assess the safety and preliminary efficacy of AVB-101 in FTD-GRN patients. Participants will receive a single administration of AVB-101 into the thalamus at specialized neurosurgical centers in Europe and the United States.

AVB-101, an investigational gene therapy, includes a correct version of the GRN gene to restore progranulin levels in the brain, potentially slowing or stopping FTD-GRN progression. It is delivered as a one-time infusion via a minimally invasive surgical procedure.

Frontotemporal dementia (FTD) usually leads to death within three to ten years of diagnosis, causing personality changes, behavioral disturbances, language loss, apathy, and reduced mobility. It is a leading cause of dementia in those under 65. Approximately 11,000 people in the U.S. and EU5 live with FTD-GRN, with around 2,200 new cases annually. Genetic mutations, including those in the GRN gene, account for about a third of FTD cases.

AviadoBio is at the forefront of developing gene therapies for neurodegenerative diseases, leveraging its deep understanding of the brain and proprietary delivery technologies. The company aims to transform the treatment landscape for conditions like FTD and amyotrophic lateral sclerosis (ALS) through its innovative approaches.