Astellas and AviadoBio forge $2bn dementia gene therapy partnership

Astellas Pharma and AviadoBio have forged an exclusive option and license agreement valued at over $2 billion, centered on an investigational gene therapy for dementia. The focus of this collaboration is AviadoBio’s AVB-101, a candidate currently in phase 1/2 trials aimed at patients suffering from frontotemporal dementia (FTD) with progranulin mutations.

Under the terms of the agreement, Astellas receives the option to secure a global exclusive license for the development and commercialization rights to AVB-101 for FTD with progranulin mutations, as well as other potential applications. In return, Astellas will invest $20 million in equity in AviadoBio and provide up to $30 million in upfront payments. If Astellas exercises its option, AviadoBio stands to gain up to $2.18 billion in license fees and milestone payments, in addition to royalties.

Frontotemporal dementia (FTD), accounting for fewer than one in 30 dementia cases, is a type of early-onset dementia with symptoms typically emerging between ages 45 and 64. This condition often leads to death within three to 13 years after diagnosis. Patients with FTD frequently experience significant personality changes, unusual behaviors, language loss, apathy, and decreased mobility.

AVB-101 is designed as a one-time treatment administered through a minimally invasive procedure directly to the thalamus in the brain, an area with extensive neural connections. This approach aims to restore progranulin levels in the frontal and temporal cortex, potentially altering the disease's progression.

Adam Pearson, Astellas’ chief strategy officer, expressed optimism about the collaboration, stating, “We look forward to working with AviadoBio’s team as we broaden our gene therapy pipeline to benefit more individuals living with debilitating neurodegenerative diseases. AVB-101 represents a groundbreaking approach to treating FTD with progranulin mutations, and this agreement could make it a part of the next wave of gene therapy products.”

Lisa Deschamps, AviadoBio’s chief executive officer, also highlighted the strategic importance of this partnership, commenting, “This collaboration combines our promising gene therapy candidate for FTD with progranulin mutations and our delivery expertise with Astellas’ global development and commercialization capabilities in gene therapies. Together, we can expedite delivering this investigational medicine to families worldwide who urgently need treatment options for FTD with progranulin mutations and other neurological disorders.”

The alliance between Astellas Pharma and AviadoBio underscores a mutual commitment to advancing innovative treatments for neurodegenerative diseases. By leveraging AviadoBio’s cutting-edge gene therapy research and Astellas’ robust development and commercialization infrastructure, the partnership aims to make significant strides in addressing the unmet needs of patients with FTD and potentially other related conditions.