UK-based gene therapy firm AviadoBio has partnered with Japanese pharmaceutical company Astellas in a deal potentially worth $2.18 billion to develop AVB-101, a gene therapy for a genetic form of frontotemporal dementia (FTD). AviadoBio will receive an upfront investment of $30 million to extend human clinical trials.
Targeting GRN Mutation in Frontotemporal Dementia
Frontotemporal dementia is a devastating early-onset dementia, leading to death within 3 to 13 years of diagnosis. It is characterized by a rapid decline in executive function, uncharacteristic behaviors, loss of language, apathy, and reduced mobility. The GRN mutation is responsible for an estimated 5% to 10% of FTD cases. AVB-101 delivers a modified virus directly into the brain to rescue cells damaged by the loss of the GRN gene, using the natural neuronal network to distribute the missing protein throughout the brain.
Clinical Development and Future Potential
AVB-101 has shown promising results in cellular and animal studies, successfully reversing pathology. An early human clinical trial is underway, and three patients have been treated. According to Professor Chris Shaw, co-founder of AviadoBio, it is too early to see beneficial results, but the aim is to halt frontotemporal dementia in its tracks.
Gene Therapy and Neurological Diseases
Professor Shaw highlighted the potential of this gene supplement approach to be applied across a broad range of neurological diseases. He noted that crossing the blood-brain barrier remains a significant hurdle for delivering gene therapies to damaged brain cells. New viral vectors are being developed to cross the blood-brain barrier and supplement or silence genes in the majority of neurons in the brain and spinal cord, potentially transforming gene therapy for neurological disorders if proven safe.
