In London’s Docklands, British biotech company AviadoBio is developing a promising gene therapy to treat FTD. The Guardian wrote about the company’s progress on June 13.

AviadoBio’s lead therapy, AVB-101, targets the specific genetic form FTD-GRN, caused by mutations that create a deficiency in progranulin, a protein essential for healthy brain cells. The treatment represents a remarkable feat of precision medicine: during a 90-minute MRI-guided procedure, neurosurgeons use a tube as thin as angel hair pasta to deliver a functional copy of the progranulin gene directly to affected brain areas.

What makes this approach particularly promising is its simplicity and permanence. It’s a one-time treatment requiring no ongoing immunosuppressive drugs. As AviadoBio CEO Lisa Deschamps explained to the Guardian, persons with FTD are born with roughly half the progranulin levels of healthy individuals, and the therapy aims to restore normal protein levels to reduce neurodegeneration.

Prof. James Rowe, a consultant neurologist at Cambridge’s Addenbrooke’s hospital involved in the U.K. trial, believes this could be game-changing: “It may be one of the first dementias to have a really transformative treatment that allows people to live much longer and much more normal lives.”

Jessica Crawford’s Journey

The human cost of FTD is powerfully illustrated through Jessica Crawford’s experience. The 33-year-old from East Yorkshire watched her mother Julia’s devastating decline after being diagnosed with FTD in 2019. Initially misdiagnosed as depressive, in 2014 Julia’s once-sociable personality changed dramatically—she stopped going out and became obsessively repetitive, playing Candy Crush endlessly and rewatching TV shows.

Julia first lost the ability to speak, then communicate entirely, becoming so confused she once put raw chicken in a sandwich. Jessica became her full-time caregiver in 2020, but Julia’s condition worsened, requiring placement in a care facility. She died in 2022.

Discovering she carries the same gene mutation, but seeking to have children, Jessica and her husband made the decision to use IVF with pre-implantation genetic screening. Of five embryos, four carried the mutation, and the fifth resulted in the birth of her healthy son. Remarkably, she donated the affected embryos to science and currently participates in GENFI, a global FTD research study, continuing to contribute to the fight against the disease that took her mother.

Clinical Progress and Future Outlook

AviadoBio has already treated six research participants in Poland and the U.S. as part of its international clinical trial, which spans the U.K., U.S., Poland, Spain, Sweden, and the Netherlands. Participants are being monitored for up to five years, and the company expects to publish initial results next year.

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.

The 60-person company recently secured an exclusive licensing agreement with Japanese pharmaceutical company Astellas, providing the resources and expertise needed to advance this promising therapy. AviadoBio’s approach stands out among competing treatments, as their gene therapy uniquely targets the thalamus—the brain’s “relay station.”

This innovative London-based company represents tangible hope for families like Jessica’s.