Astellas and Dyno Partner to Develop Next-Generation AAV Gene Therapy Vectors for Skeletal and Cardiac Muscle

After suffering a series of setbacks in the development of its gene therapies acquired through a 2019 buyout of Audentes in 2019, Astellas Pharma has signed an option and license agreement with Dyno Therapeutics to develop next-generation adeno-associated virus (AAV) vectors for gene therapy directed to skeletal and cardiac muscle using Dyno’s CapsidMap platform.

Photo: Eric Kelsic, co-founder and CEO of Dyno Therapeutics

Dyno’s CapsidMap platform represents a transformative approach applying in vivo experimental data and machine learning to create novel AAV capsids – the cell-targeting protein shells of viral vectors – designed to optimize tissue targeting and immune-evading properties, in addition to improving packaging capacity and manufacturability. Unlike traditional approaches, CapsidMap is well-suited to simultaneously optimize capsids for delivery across multiple organs, with the goal of enabling more effective whole-body treatment for many diseases.

“Through our efforts in gene therapy and the Astellas Gene Therapies Center of Excellence, Astellas strives to identify, develop and deliver transformative gene-based therapies for patients with genetic diseases who currently have few or no effective treatment options. Our principal focus is on developing adeno-associated virus delivered therapies for the treatment of well-defined serious diseases,” said Naoki Okamura, chief strategy, financial, and business officer at Astellas.

Under the terms of the agreement, Dyno will design novel AAV capsids with improved functional properties for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities, including manufacturing, of gene therapy product candidates using the novel capsids. Dyno will receive an $18 million upfront payment and be eligible to receive additional payments during the research phase of the collaboration as well as clinical and sales milestone payments and royalties for any resulting products. The aggregate potential value of future milestone and royalty payments to Dyno exceeds $235 million per product and more than $1.6 billion in total value.

“Dyno and Astellas each bring unique strengths to this collaboration, together enabling more rapid creation of new therapies for patients with great unmet need,” said Eric Kelsic, co-founder and CEO of Dyno Therapeutics. “This partnership demonstrates the flexibility of Dyno’s platform to precisely design the delivery properties of gene therapy vectors towards multi-organ and disease-specific profiles, applying the scientific insights we are rapidly learning across all our partnered and internal vector engineering programs using CapsidMap.”

Author: Rare Daily Staff