Astellas and AviadoBio Announce Exclusive Gene Therapy AVB-101 Deal for Frontotemporal Dementia

AviadoBio Ltd. and Astellas Pharma Inc. have entered into an exclusive option and license agreement for AVB-101, an investigational gene therapy currently in Phase 1/2 development aimed at treating frontotemporal dementia with progranulin mutations (FTD-GRN). This collaboration highlights a significant move in addressing early-onset dementia, which is a critical yet often misdiagnosed condition affecting individuals under 65 years old. FTD-GRN patients typically suffer from rapid deterioration in cognitive functions, uncharacteristic behavior changes, loss of language, apathy, and decreased mobility, with a life expectancy of three to 13 years post-diagnosis.

Under this agreement, Astellas has secured the option to obtain a worldwide exclusive license for AVB-101, not only for FTD-GRN but also for other potential applications. The financial terms include a $20 million equity investment from Astellas and an additional up to $30 million in upfront payments to AviadoBio. Should Astellas decide to exercise its option, AviadoBio stands to receive up to $2.18 billion in license fees and milestone payments, along with royalties.

Lisa Deschamps, CEO of AviadoBio, expressed optimism about the partnership, emphasizing that the collaboration could address the significant unmet needs in treating frontotemporal dementia. Deschamps pointed out that the ASPIRE-FTD trial for AVB-101 has already begun dosing its first cohort of patients. She believes that combining AviadoBio's expertise in gene therapy and delivery with Astellas' global capabilities in development and commercialization could speed up the availability of this investigational treatment, offering hope to families and individuals battling FTD-GRN and other neurodegenerative diseases.

Adam Pearson, Chief Strategy Officer at Astellas, echoed this sentiment, underscoring the innovative nature of AVB-101 and its potential role in the next generation of gene therapy products. He stated that genetic regulation is a core component of Astellas' primary strategy and that this agreement aligns with their mission to provide solutions for patients with debilitating conditions.

AviadoBio is committed to developing life-changing medicines for those with neurological conditions, leveraging its deep understanding of brain science and proprietary gene therapy platforms. Founded on pioneering research from King's College London and the UK Dementia Research Institute, AviadoBio benefits from a leadership team experienced in gene therapy development, delivery, and commercialization. The company is well-positioned to bring transformative treatments to patients, driven by a relentless pursuit of cures for neurodegenerative diseases.

AviadoBio's funding comes from notable investors, including New Enterprise Associates, Monograph Capital, F-Prime Capital, Johnson & Johnson Innovation - JJDC, Inc., SV Health Investor's Dementia Discovery Fund, Advent Life Sciences, EQT Life Sciences, and LifeArc Ventures.

Astellas Pharma Inc. operates in over 70 countries and focuses on creating new drugs to meet high unmet medical needs. They aim to combine their pharmaceutical expertise with cutting-edge technology through partnerships, seeking to transform innovative science into valuable health solutions for patients.

Astellas Gene Therapies, a Center of Excellence within Astellas, is dedicated to developing genetic medicines for rare diseases affecting the eye, central nervous system, and neuromuscular system. With a robust gene therapy drug discovery engine and manufacturing capabilities, Astellas Gene Therapies aims to advance their programs towards clinical investigation, based out of South San Francisco, with additional facilities in Sanford, North Carolina, and Tsukuba, Japan.