Astellas Pharma, recognizing the urgent need for treatments in neurodegenerative disorders, has made a significant financial commitment to AviadoBio, a London-based biotech startup. The collaboration involves a $50 million investment focused on a pioneering gene therapy for frontotemporal dementia (FTD), a form of dementia that currently lacks any FDA-approved medications. This financial package includes a $20 million equity stake and up to $30 million in upfront payments.
AviadoBio’s key program, AVB-101, is at the heart of this deal. Currently in early-stage clinical trials, AVB-101 aims to treat a version of FTD triggered by mutations in the gene responsible for producing the progranulin protein, which plays a critical role in regulating immune activity within the brain. FTD associated with progranulin deficiency (FTD-PGN) causes rapid cognitive decline and typically results in death within three to 13 years of diagnosis. The therapy delivers a functional copy of the gene directly into the thalamus, where it is distributed to key cells via an engineered virus commonly used in gene therapy applications.
The science behind AVB-101 is built on research from King’s College London, with AviadoBio having emerged with robust support, backed by $80 million from a Series A funding round in 2021. Preclinical studies in mice have shown promising results, such as the restoration of normal progranulin levels and a reduction in disease markers. The treatment has recently progressed to human trials, with a Phase 1/2 study beginning in early 2023 aiming to enroll nine participants.
AviadoBio is not alone in targeting progranulin levels to treat FTD-PGN. Competitors include Passage Bio with its PBFT02 gene therapy and Alector’s AL100, an antibody drug currently in Phase 3 testing under a collaboration with GSK.
Gene therapy is a strategic focus for Astellas, following its acquisition of Audentes Therapeutics in 2020. This acquisition expanded Astellas’s pipeline with several gene therapies for rare inherited disorders, although some faced regulatory challenges and clinical holds due to safety concerns. Despite these setbacks, Astellas continues to explore the potential of gene therapy across various medical fields, including neuromuscular disorders, central nervous system conditions, and ophthalmology.
This deal with AviadoBio adds to Astellas’s experiences with similar agreements, demonstrating its ongoing strategy to secure innovative therapies through strategic investments and partnerships. Past collaborations include an equity investment in Taysha Gene Therapies with options for rare neurodegenerative disorders therapies, and licensing agreements for gene therapies with Kate Therapeutics and 4D Molecular Therapeutics.
The Astellas-AviadoBio collaboration could potentially unlock up to $2.18 billion for AviadoBio in license fees and milestone payments, plus subsequent royalties, should Astellas proceed beyond their current option to fully license AVB-101. This partnership stands to bolster AviadoBio’s resources significantly and enhance Astellas’s portfolio of gene therapies, particularly for tackling the challenging field of neurodegenerative diseases.
This collaboration is aimed not only at advancing a potentially first-in-class treatment for FTD but also at consolidating Astellas’s position in the gene therapy market. With each company bringing its unique strengths to the table—AviadoBio’s innovative gene therapy platform and Astellas’s global development and commercialization capabilities—the partnership holds promise for addressing the profound unmet medical needs of the FTD patient community.
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