AviadoBio CEO Lisa Deschamps and Astellas senior vice president Richard Wilson discussed the ASPIRE-FTD clinical trial for FTD-GRN, challenges with the development and potential distribution of the AVB-101 gene therapy, and the motivations for their work in an interview with Cell & Gene.

Collaboration Helps Overcome Challenges with Gene Therapy Development

While acknowledging that gene therapies can have long development timelines, fraught with unforeseen and complex challenges, Deschamps told interviewer Erin Harris that what drives AviadoBio is their hope to make a fundamental difference in the lives of families facing FTD-GRN.

“I’ve seen firsthand the transformative effect gene therapy can have in rare disease, where entire treatment paradigms are changed,” Deschamps said. “We are motivated by the challenge of finding a path forward for people with FTD, one of the most common forms of dementia in those under the age of 65. FTD is frequently misdiagnosed and underrecognized, and there are no approved disease-modifying treatments.”

In October 2024, AviadoBio announced its exclusive agreement with Astellas, in which the latter, larger company provides significant resources to further the development of AVB-101. Astellas possesses the infrastructure to manufacture and distribute AVB-101 globally, and has experience navigating the regulatory processes of different countries, which will be essential as the trial opens new sites in various countries.

“At AviadoBio, we say we’re relentlessly chasing cures. It’s essential to do that alongside a partner who believes in the same mission,” Deschamps said. “From our perspective, it’s more than science; what drew us together was a shared belief in delivering breakthroughs for patients with no other options.”

Wilson, who is also Astellas’ primary focus lead for genetic regulation, added, “We know the work AviadoBio is doing is a strong fit, expanding our work in gene therapy in line with our long-term strategic view – and in an area where AviadoBio brings expertise and capabilities with their highly differentiated approach in an increasingly competitive space.”.

AviadoBio and Astellas Motivated by Lived Experiences of Families Affected by FTD

People living with FTD need biopharma companies to remain committed to finding effective treatments, Wilson said. He highlighted the experiences that those touched by FTD have shared with him: “A few months ago, I had the privilege of joining AviadoBio for the Association for Frontotemporal Degeneration’s Hope Rising Benefit. It was powerful to listen to so many stories of lives changed by the diagnosis of FTD, as well as the resilience and courage that followed.”

Setbacks can occur during clinical trials, lengthening the development of even promising interventions. Asked what lessons AviadoBio has learned during the development of AVB-101, Deschamps underscored the importance of staying true to the company’s mission of developing life-changing medicines for people facing neurological disorders.

Wilson sounded a similar note. “[I]t is exciting that we’ve seen a critical, industry-wide shift in this direction, and real action in practice,” he said. “However, challenges around how we best put patients at the heart of trials still exist, from slow enrollment to drop-out rates, and complex protocols that don’t always meet patients’ needs. Increased and earlier patient involvement through a continuous ‘feedback loop’ approach could help address these challenges.”

Events like Hope Rising and the Research Roundtable are just some ways that AFTD supports collaboration and networking between companies researching treatments and diagnostic tools for FTD. Learn more about AFTD’s support for FTD research on our For Researchers page.

Are you interested in reading more about ongoing clinical trials for FTD? Vesper Bio’s trial for a sortilin inhibitor, a potential disease-modifying treatment for FTD-GRN, reached an enrollment milestone earlier this year. Coya Therapeutics reported positive interim data for its trial for a pair of drugs that address regulatory T cell dysfunction in FTD and other dementias.