Astellas and Dyno Therapeutics Announce $1.6 Billion Collaboration to Develop Next-Generation AAV Gene Therapy Vectors for Skeletal and Cardiac Muscle Diseases

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Recently, Astellas announced a licensing agreement with Dyno Therapeutics to use Dyno's CaspidMap artificial intelligence platform to develop next-generation adeno-associated virus (AAV) vectors for skeletal muscle and cardiac muscle targeted gene therapy.

Under the terms of the agreement, Dyno will design novel AAV capsids with improved functional properties for gene therapy, and Astellas will be responsible for preclinical, clinical, and commercialization activities, including manufacturing, of gene therapies developed using the novel AAV capsids. Dyno will receive an upfront payment of $18 million and is eligible for additional payments during the research phase of the collaboration, as well as clinical and sales milestone payments and royalties on any resulting products. Dyno is eligible to receive potential milestone and royalty payments of over $235 million for each product developed using the novel AAV capsids, with the total value of the agreement exceeding $1.6 billion.

Following the establishment of its Gene Therapy Center of Excellence to strengthen its gene therapy division after acquiring Audentes Therapeutics for $3 billion last year, Astellas has now partnered with Dyno Therapeutics. This collaboration will combine Dyno's AI-driven AAV vector engineering capabilities with Astellas' global leadership in AAV-based gene therapy pipeline assets.

Currently, due to safety concerns, AAV vectors face some limitations and underwent inspection by the FDA Advisory Committee in the United States this fall. During this two-day review, the FDA Advisory Committee conducted safety reviews of Novartis' SMA gene therapy Zolgensma and Spark Therapeutics' gene therapy for inherited retinal diseases, Luxturna.

In addition to safety issues, AAV also faces challenges such as manufacturing difficulties, limited payload capacity, and a narrow range of targetable tissues. Gene therapies using AAV also carry the risk of being ineffective in individuals with pre-existing immunity to the AAV virus.

Dyno Therapeutics believes that the company is capable of solving the aforementioned problems. Its CapsidMap artificial intelligence platform represents a transformative approach, utilizing in vivo experimental data and machine learning to create novel AAV capsids (the cell-targeting protein shells of viral vectors), optimizing tissue targeting and immune escape, while enhancing packaging capacity and manufacturability. Unlike traditional methods, CapsidMap is a unique platform particularly suited for simultaneously optimizing capsids to achieve delivery across multiple organs, aiming for more effective systemic treatments for a variety of diseases.

Previously, Dyno Therapeutics, with its unique CapsidMap AI platform, reached a $2 billion agreement with Novartis and Sarepta Therapeutics, as well as an $1.8 billion agreement with Roche and its subsidiary Spark Therapeutics. Through these partnerships, the collaborators aim to develop next-generation AAV gene therapy vectors, with the goal of expanding the utility of gene therapies in ocular diseases, muscular disorders, central nervous system conditions, and liver diseases.

Reference Source: Astellas becomes latest Dyno gene therapy partner with $1.6B biobucks pact to join Roche, Novartis, Sarepta