UK-based gene therapy firm AviadoBio has partnered with Japanese pharmaceutical company Astellas in a landmark deal potentially worth USD$2.18 billion. The collaboration aims to advance the clinical development of AVB-101, a gene therapy targeting a genetic form of frontotemporal dementia (FTD). AviadoBio will receive an upfront investment of USD$30 million to extend human clinical trials.
Targeting GRN Mutation in Frontotemporal Dementia
Frontotemporal dementia is a devastating early-onset dementia, often misdiagnosed, leading to death within 3-13 years of diagnosis. It is characterized by a rapid decline in executive function, uncharacteristic behaviors, loss of language, apathy, and reduced mobility. The AVB-101 gene therapy focuses on addressing cases of FTD caused by mutations in the GRN gene, which account for an estimated 5-10% of all FTD cases.
Professor Chris Shaw, co-founder of AviadoBio and the inaugural Hugh Green Translational Chair in Neuroscience at the University of Auckland’s Centre for Brain Research, stated, “We are finally delivering on a promise made to our patients – that genetic discoveries would lead to transformative genetic therapies.”
AVB-101: A Novel Gene Therapy Approach
AVB-101 utilizes a modified virus to deliver a functional copy of the GRN gene directly into the brain. This approach aims to supplement the missing protein and rescue cells damaged by the GRN gene loss. The therapy leverages the brain's natural neuronal network to distribute the protein throughout the brain.
Early Clinical Trial and Future Prospects
AVB-101 has demonstrated promising results in cellular and animal studies, successfully reversing pathology associated with GRN mutations. An early human clinical trial is currently underway, with three patients already treated. While it is too early to assess definitive benefits, the primary goal is to halt the progression of frontotemporal dementia.
Professor Shaw emphasized the broader potential of this gene supplement approach, stating, “In some ways, this is a very niche therapy, but this gene supplement approach has the potential to be applied across a broad range of neurological diseases. There is a whole pipeline of other therapies that could be brought to clinical trial on the back of this.” He also noted the challenges of crossing the blood-brain barrier for effective gene therapy delivery, highlighting ongoing efforts to develop viral vectors capable of efficiently targeting neurons throughout the brain and spinal cord.
