Breakthrough Treatment for Genetic Dementia in the UK Lab

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AviadoBio’s breakthrough therapy hopes to stop progress of FTD, which is usually diagnosed in people under 65

Located in east London's Docklands, AviadoBio is developing a groundbreaking therapy to combat a form of dementia affecting millions globally. While there is currently no cure for dementia, the team at AviadoBio is hopeful that their clinical trials can halt the progression of a specific genetic type of frontotemporal dementia (FTD).

FTD primarily impacts the front and sides of the brain and is distinct from Alzheimer's disease as it does not initially present with memory loss. Instead, it is characterized by a gradual decline in language skills and shifts in personality and behavior.

Typically diagnosed in individuals aged 45 to 65, FTD can also affect younger adults in their 20s and 30s. With an estimated 20,000 to 40,000 individuals in the UK and 1 to 2 million worldwide living with FTD, the need for effective treatment is critical.

AviadoBio's gene therapy, targeting a type of FTD known as FTD-GRN, aims to address mutations in a gene resulting in a deficiency of progranulin (GRN), a vital protein for brain cell health. The company, in collaboration with Astellas, is conducting clinical trials in multiple countries, including the UK, US, Poland, Spain, Sweden, and the Netherlands.

Early results from the trial are promising, with the first data expected to be published next year. This therapy offers hope for individuals like Julia Crawford, who battled FTD caused by mutations in the C9orf72 gene. Her daughter, Jessica, is now part of a global study on FTD families and has firsthand experience with the devastating impact of the disease.

AviadoBio's lead product, AVB-101, involves a unique procedure where a functional gene copy is infused directly into the brain to restore progranulin levels. This one-time treatment does not require immunosuppressant drugs post-surgery, offering a potential breakthrough in dementia care.

While other medications are in development globally, AviadoBio's approach stands out for its targeted treatment of the thalamus, a crucial brain region. The company's presence in Canary Wharf's life science cluster underscores the UK's leadership in dementia research, with initiatives like the GENFI study contributing to significant advancements in the field.

With the potential to transform the landscape of dementia treatment, AviadoBio's innovative therapy offers hope for individuals and families affected by this debilitating condition.



Source: The Guardian
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