AviadoBio Ltd inks US$2.2bn gene therapy deal with Astellas Pharma Inc

In return for an equity investment of US$20m and an upfront payment of US$30m to British gene therapy specialist AviadoBio Ltd, Astellas Pharma Inc receives the option to licence the AVR-01 drug candidate that is currently in Phase I/II development in patients with frontotemporal dementia with progranulin mutations (FTD-GRN). 

Frontotemporal dementia is an often unrecognised and misdiagnosed early-onset dementia  with bad prognosis. Typically, the disorder leads to death within three to 13 years from diagnosis, and patients commonly experience a rapid decline in executive function such as attention control, working memory, problem-solving, and uncharacteristic behaviours, loss of language, apathy, and reduced mobility. 

Progranulin is an 88kDa protein and has been established as a marker for frontotemporal dementia (FTD). Approximately 5-10% of patients with FTD have low progranulin concentrations in their serum. AVB-101 is a potential one-time therapy designed to stop disease progression by delivering a functional copy of the GRN gene to restore appropriate progranulin levels to affected areas of the brain.

AVB-101 is delivered using a minimally invasive, stereotactic neurosurgical procedure directly to the part of the brain called the thalamus. The thalamus has extensive connections to other parts of the brain, including the frontal and temporal lobes, which play a critical role in FTD and the symptoms that impact patients and their families. This targeted delivery method aims to safely and effectively cross the blood-brain barrier, delivering targeted treatment directly to the brain to restore progranulin levels in the frontal and temporal cortex where it is needed most, while at the same time minimizing the dose required and thereby limiting any potential systemic exposure

Besides its US$20m equity investment and up to US$30m upfront payments for the option to license AVB-101, Astellas Pharma may pay  milestones up to US$2.18bn plus royalties to AviadoBio if Astellas exercises its option. The ongoing Phase I/II multicentre study started enrollment in April 2024.