Dyno Therapeutics Enters Collaboration and License Agreement With Roche to Develop Next-Generation AAV Gene Therapy Vectors for CNS Diseases and Liver-Directed Therapies

• Collaboration combines Dyno’s AI-powered AAV vector capabilities with Roche and Spark Therapeutics’ leading-edge gene therapy capabilities
• Dyno is eligible to receive milestone payments that may exceed $1.8 billion

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Dyno Therapeutics, a biotech company applying artificial intelligence (AI) to gene therapy, today announced a collaboration and license agreement with Roche (SIX: RO, ROG; OTCQX: RHHBY) to apply Dyno’s CapsidMap™ platform for the development of next-generation adeno-associated virus (AAV) vectors for gene therapies for central nervous system (CNS) diseases and liver-directed therapies for the portfolio of both Roche and Spark Therapeutics, a member of the Roche Group.

Dyno’s proprietary CapsidMap™ platform represents a transformative approach to the identification of novel AAV capsids – the cell-targeting protein shell of viral vectors – optimizing tissue targeting and immune-evading properties, in addition to improving packaging capacity and manufacturability.

Under the terms of the agreement, Dyno will be responsible for the design of novel AAV capsids with improved functional properties for gene therapy, while Roche and Spark Therapeutics will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the novel capsids. Dyno will receive an undisclosed upfront payment and if successful, is 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 payments to Dyno may exceed $1.8 billion.

“This new partnership represents Dyno’s largest collaboration to date, and we are excited to work with Roche and Spark Therapeutics to expand the frontier of gene therapies for the central nervous system and liver. The Dyno, Roche and Spark teams share a bold vision for gene therapy and believe that enhancing vectors that deliver these therapies is key to developing new treatments for patients in need,” stated Dyno’s CEO and co-founder Eric Kelsic, Ph.D. “Partnering is a fundamental element of Dyno’s business strategy, and the continuing interest by leading gene therapy developers is accelerating our growth plans and positive impact on patients.”

“We strongly believe in the potential of gene therapy and are excited to bring together experts from Roche, Spark and Dyno to develop next-generation gene therapies. Dyno’s innovative AI-powered approach to designing optimized AAV vectors will further complement and build on our progress in gene therapy. We look forward to leveraging Dyno’s technology to develop new, innovative treatments for patients across CNS and liver-directed therapies,” said James Sabry, Head of Roche Pharma Partnering.

About CapsidMap™ for Designing AAV Gene Therapies

By designing capsids that confer improved functional properties to Adeno-Associated Virus (AAV) vectors, Dyno’s proprietary CapsidMap™ platform overcomes the limitations of today’s gene therapies on the market and in development. Today’s treatments are primarily confined to a small number of naturally occurring AAV vectors that are limited by delivery, immunity, packaging size, and manufacturing challenges. CapsidMap uses artificial intelligence (AI) technology for the design of novel capsids, the cell-targeting protein shell of viral vectors. The CapsidMap platform applies leading-edge DNA library synthesis and next generation DNA sequencing to measure in vivo gene delivery properties in high throughput. At the core of CapsidMap are advanced search algorithms leveraging machine learning and Dyno’s massive quantities of experimental data, that together build a comprehensive map of sequence space and thereby accelerate the discovery and optimization of synthetic AAV capsids.

About Dyno Therapeutics

Dyno Therapeutics is a pioneer in applying artificial intelligence (AI) and quantitative high-throughput in vivo experiments to gene therapy. The company’s proprietary CapsidMap™ platform rapidly discovers and systematically optimizes Adeno-Associated Virus (AAV) capsid vectors that significantly outperform current approaches for in vivo gene delivery, thereby expanding the range of diseases treatable with gene therapies. Dyno was founded in 2018 by experienced biotech entrepreneurs and leading scientists in the fields of gene therapy and machine learning. The company is located in Cambridge, Massachusetts. Visit www.dynotx.com for additional information.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201014005248/en/