Roche deal latest in AAV gene therapy partnership plan for Dyno

By Jane Byrne

- Last updated on GMT

© GettyImages/sittithat tangwitthayaphum
© GettyImages/sittithat tangwitthayaphum

Related tags AAV platform Gene therapy Nervous system capsids

US company, Dyno Therapeutics, has inked a deal with Roche to develop next-generation AAV gene therapy vectors for central nervous system (CNS) diseases and liver-directed therapies.

Under the terms of the deal, Dyno will be responsible for the design of novel adeno-associated virus (AAV) capsids with improved functional properties for gene therapy, while Roche and Spark  Therapeutics, an independent company within the Roche Group, will take on all the preclinical, clinical and commercialization activities for gene therapy product candidates using those capsids.

The Cambridge, Massachusetts-based Dyno will receive an undisclosed upfront cash payment with potential for future milestone payments that could exceed US$1.8bn.

By designing capsids that confer improved functional properties to AAV vectors, Dyno said its proprietary CapsidMap platform overcomes the limitations of today’s gene therapies on the market and in development. The technology can help expanding the range of diseases treatable with gene therapies, said the developer.

“CapsidMap is an AI and machine-learning powered approach to comprehensively map the AAV capsid space and discover novel synthetic capsids with transformative delivery properties for many diseases.

“At its core 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. 

“We are excited to be on the leading edge of this field and making an important contribution to advancing the field of gene therapies to reach more patients,“​ Dyno CEO Eric Kelsic told BioPharma-Reporter.

In terms of how this deal complements the work Dyno is doing with Novartis and Sarepta, two partnerships based on the same technology it announced ​in May this year, he explained:

“Each of our partners has focused on specific disease areas in relation to our work with them developing next-generation gene therapy vectors: Novartis is focused on ocular diseases, Sarepta on muscle diseases, and Roche on CNS diseases and liver-directed therapies.”

All of those collaborative partnerships in tandem with the work going on internally at Dyno are helping to enhance the CapsidMap platform, said the CEO.

Interest in technology piqued early 

The biotech’s technology platform is based on work done at Harvard Medical School.

When asked how the company has been able to secure such high-level partnerships in the gene therapy field so quickly, with Dyno only founded in late 2018, Kelsic said one reason is the startup is focused on solving a challenging problem key to achieving gene therapy's full potential.

“We have [also] invented a compelling and differentiated solution to this problem by combining powerful new technologies like AI and next-gen DNA synthesis, and we have recruited fantastic scientific and business teams passionate about our mission to transform gene therapy, who have been hard at work, and who know how to tell this story.”

There was significant interest in Dyno's technology from many gene therapy developers even prior to the formation of the company, he said, given the fact its team communicated early about it at a number of scientific meetings.

“That interest has only increased with the progress we've made since then. We plan to continue to form more partnering deals. Many gene therapy companies are seeking new capsids because they are limited by the small number of naturally occurring AAV vectors to create their gene therapies. With CapsidMap, Dyno designs novel capsids with new and optimized properties, enabling gene therapy developers to create gene therapies targeted to more tissues and to treat more diseases.”

Dyno has financed its business, to date, through seed financing, which included US$9m secured at launch in late 2018, as well as from the funding from partnerships. 

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