UK Dementia Breakthrough: Halting Genetic Disease
- A British university spinout, AviadoBio, is pioneering a potential breakthrough in treating frontotemporal dementia (FTD), a devastating condition affecting millions globally.
- Professor James Rowe, a consultant neurologist at addenbrooke’s Hospital, cambridge, described the treatment as possibly "transformative," offering the possibility of longer and more normal lives for patients.
- The therapy, AVB-101, involves a neurosurgeon infusing a functional copy of the progranulin gene directly into the brain via a thin cannula.
Scientists are making strides against frontotemporal dementia (FTD) with innovative gene therapy, offering a beacon of hope. AviadoBio is leading the charge, developing a groundbreaking treatment for FTD-GRN, a genetic form of this debilitating disease. Clinical trials are underway across the UK,US,and europe,targeting the root cause by restoring progranulin levels. This protein is essential for brain health. The therapy could revolutionize treatment. The condition affects millions globally. News Directory 3 understands the urgency, and we’re following this story closely. Discover how this novel approach can change the future of FTD treatment beginning next year.
Gene Therapy Offers Hope for Frontotemporal dementia Treatment
Updated June 13, 2025
A British university spinout, AviadoBio, is pioneering a potential breakthrough in treating frontotemporal dementia (FTD), a devastating condition affecting millions globally. Their gene therapy targets FTD-GRN, a genetic subtype of the disease.
Professor James Rowe, a consultant neurologist at addenbrooke’s Hospital, cambridge, described the treatment as possibly “transformative,” offering the possibility of longer and more normal lives for patients. FTD, unlike Alzheimer’s, primarily impacts the front and sides of the brain, leading to personality changes, behavioral issues, and language difficulties.
The therapy, AVB-101, involves a neurosurgeon infusing a functional copy of the progranulin gene directly into the brain via a thin cannula. This aims to restore progranulin levels, crucial for healthy brain cells. The procedure takes about 90 minutes, guided by MRI, and requires no subsequent immunosuppressant drugs.
AviadoBio, a company of 60 employees, has partnered with astellas, a Japanese pharmaceutical firm, to advance the therapy. Clinical trials are currently enrolling patients in the United kingdom, the United States, Poland, Spain, Sweden, and the netherlands. The first infusion occurred in Warsaw in March 2024. Patients will be monitored for up to five years, with initial data expected next year.
The company’s chief executive officer, Lisa deschamps, said the goal is to supplement the GRN gene and restore progranulin levels to reduce the neurodegeneration effect of frontotemporal dementia.
Jessica Crawford,whose mother died from FTD,underwent IVF with pre-implantation genetic diagnosis to ensure her son did not inherit the gene mutation. She also participates in GENFI, a global study of families with FTD.
“It may be one of the first dementias to have a definitive treatment, a cure if you like, a really transformative treatment that allows people to live much longer and much more normal lives,” said Prof James Rowe, Addenbrooke’s Hospital.
what’s next
AviadoBio anticipates publishing initial data from its clinical trials next year, offering a glimpse into the potential of this gene therapy to combat frontotemporal dementia and improve the lives of those affected by this devastating condition.