Dyno Therapeutics forms partnership with Roche to advance AAV gene therapy vectors for neurological diseases

Dyno Therapeutics, a genetic technologies company applying artificial intelligence (AI) to enable in vivo gene delivery, has announced its second research collaboration with Roche to develop next-generation adeno-associated virus (AAV) vectors for gene therapies targeting neurological diseases.

Dyno and Roche previously announced a research collaboration and license agreement for neurological diseases and liver-directed therapies in October 2020. Under the terms of this new collaboration, Dyno Therapeutics provides Roche further access to the company’s field-leading platform and sequence design technologies enabling in vivo gene delivery.

Existing gene therapies have primarily used a small number of naturally occurring AAV vectors limited by low delivery efficiency as well as problems with pre-existing immunity and manufacturability. To overcome these challenges, Dyno has pioneered the application of AI and high-throughput in vivo data collection to engineering AAV capsids for improved tissue targeting, immune-evasion and manufacturability.

Dyno’s Low-Shot Efficient Accelerated Performance (LEAPSM) technology, paired with the capability to make billions of in vivo sequence-function measurements each month, enables optimization of capsids for therapeutic success at an accelerated rate. As a platform built to solve the most critical challenges facing gene therapy developers, Dyno’s industry-leading gene delivery solutions enable Dyno partners to create ever better gene therapies.

Under the terms of the new agreement, Dyno is responsible for the design and discovery of novel AAV capsids with improved functional properties. Roche in turn is responsible for conducting capsid validation studies and further preclinical, clinical, and commercialization activities for multiple neurological gene therapy product candidates leveraging novel Dyno capsids. Roche will pay Dyno US$50 million upfront, along with additional payments during the research phase of the collaboration, plus potential preclinical, clinical, and sales milestone payments totaling over US$1 billion, and royalties on net sales of commercial products.

“This new collaboration with Roche, focused on developing next-generation gene therapies for underserved patients with life-altering neurological diseases, is a testament to the outstanding progress made by Dyno’s platform and to the commitment we make to partners in all of our projects,” said Eric Kelsic, Ph.D., Founder and Chief Executive Officer of Dyno. “Our approach combines extensive in vivo data with the world’s most advanced AI models for sequence-function prediction, empowering Dyno capsid engineers with industry-leading capabilities for solving the longstanding challenge of therapeutic gene delivery. Dyno’s platform brings the entire field closer to realizing a future where all gene therapies are safe, effective, and widely accessible to all patients who need them.”

“We are very pleased to take our partnership with Dyno Therapeutics to the next level. Our combined knowledge and resources will allow us to explore new treatments for historically difficult-to-treat neurological diseases,” said Boris L. Zaïtra, Head of Roche Corporate Business Development. “We are dedicated to making significant advancements in this field, and part of this is partnering with companies like Dyno Therapeutics. Our previous collaboration with Dyno Therapeutics gives us great confidence to increase our investment in therapeutic gene delivery, to support our neurological disease portfolio.”

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