Roche will apply Dyno Therapeutics’ engineered adeno-associated virus (AAV) capsid platform to develop next-generation AAV vectors for gene therapies targeting unspecified neurological diseases, through a collaboration that could generate more than $1 billion for Dyno.

The collaboration—the second for the companies—is designed to provide Roche with further access to Dyno’s platform and sequence design technologies enabling in vivo gene delivery.

Dyno and Roche launched their first collaboration in 2020, when they inked a research collaboration and license agreement for a potentially more-than-$1.8 billion partnership to develop next-generation AAV vectors for neurological disease and liver-directed gene therapies.

“Our previous collaboration with Dyno Therapeutics gives us great confidence to increase our investment in therapeutic gene delivery, to support our neurological disease portfolio,” Boris L. Zaïtra, head of Roche Corporate Business Development, said in a statement. “We are very pleased to take our partnership with Dyno Therapeutics to the next level.”

In their latest collaboration, Roche will use Dyno’s Low-Shot Efficient Accelerated Performance (LEAPSM) technology, through which Dyno applies artificial intelligence (AI) and high-throughput in vivo data collection to engineering AAV capsids for improved tissue targeting, immune-evasion, and manufacturability.

Dyno says LEAP, plus its capability to make billions of in vivo sequence-function measurements each month, enables it to rapidly optimize its capsids for therapeutic success.

At a scientific symposium held in May at the 27th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting in Baltimore, Dyno founder and CEO Eric Kelsic, PhD, shared how capsids designed with LEAP dramatically outperformed capsids designed by humans without AI-assistance, improving in vivo delivery efficiency and potentially enabling lower manufacturing costs.

The companies said Dyno will oversee the design and discovery of novel AAV capsids with improved functional properties, while Roche has agreed to conduct capsid validation studies and further preclinical, clinical, and commercialization activities for unspecified “multiple” neurological gene therapy product candidates leveraging novel Dyno capsids.

Targeting ‘difficult-to-treat’ neuro diseases

“Our combined knowledge and resources will allow us to explore new treatments for historically difficult-to-treat neurological diseases,” Zaïtra added.

Roche has agreed to pay Dyno $50 million upfront, unspecified additional payments during the research phase of the collaboration, as well as up to $1 billion-plus in potential preclinical, clinical, and sales milestone payments, plus 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,” stated Kelsic.

Kelsic joined George Church, PhD, of Harvard Medical School and four other co-founders to establish Dyno in 2018.

In addition to Roche, Dyno has a potentially more than $1.6 billion collaboration based on its platform with Astellas Pharma. The companies agreed in 2021 to partner on helping Astellas develop next-generation AAV vectors for gene therapies targeting skeletal and cardiac muscle using Dyno’s CapsidMap™ platform.

And in May 2020, Dyno secured two collaborations based on its platform: one with Novartis focused on eye diseases, and another with Sarepta Therapeutics to develop gene therapies for muscle diseases for a combined up-to-$2 billion.

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