The US-based biotechnology company will receive an upfront payment from Otsuka and is eligible to receive additional development, regulatory, and sales milestone payments, totalling more than $1.5bn, along with tiered royalties on future sales

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Otsuka, Shape Therapeutics enter into AAV therapeutics partnership. (Credit: Michal Jarmoluk from Pixabay)

Japan-based Otsuka Pharmaceutical has teamed up with US-based drugmaker Shape Therapeutics to develop intravitreally delivered adeno-associated virus (AAV) therapeutics for ocular diseases.

The collaboration aims to develop novel treatment options for people living with serious eye diseases, with options for additional targets and tissue types.

Shape Therapeutics will leverage its AAVid capsid discovery platform and transgene engineering technology, and Otsuka its expertise in genetic payload design and ophthalmology.

In exchange, Shape Therapeutics will receive an upfront payment from Otsuka and is eligible to receive additional development, regulatory, and sales milestone payments, totalling more than $1.5bn.

The US-based biotechnology company is also eligible to receive tiered royalties on the future sales of products emerging from the collaboration.

Otsuka Pharmaceutical executive director and Osaka Research Center for Drug Discovery head Toshiki Sudo said: “Otsuka has drug discovery expertise in a broad range of therapeutic areas including central nervous systems (CNS), nephrology and ophthalmology.

“Our recent research activities have led to identification of target molecules and antibodies for specific ocular diseases of interest with high unmet medical needs.

“Our collaboration with ShapeTX aims to enable delivery of vectorised antibody drugs in combination with AAV, in order to target specific disease cell types in the eye and provide a once-in-a-lifetime and curative administration with stable lifetime expression.”

Shape Therapeutics developed its AI-driven AAVid platform by combining high-throughput screening of billions of unique AAV variants and machine learning techniques.

The platform is intended to identify novel AAV capsids for direct-to-NHP in-vivo selection, to enhance clinical translation.

AAVid capsids are used for precise delivery of the medication to the target tissue, to reduce the required dose and related clinical safety risks.

Shape Therapeutics will also apply its transgene engineering technology to optimise payloads provided by Otsuka for therapeutic levels of gene expression in targeted cell types.

Shape Therapeutics co-founder and CEO Francois Vigneault said: “We’ve built our AAVid platform on generative AI approaches akin to those behind Midjourney and DALL-E 2 to tackle industry challenges with gene therapy delivery.

“By incorporating diffusion models, our platform is designing novel medicines that transcend the boundaries of what is possible experimentally.

“Our collaboration with Otsuka marks an exciting chapter in our journey as we extend the reach and impact of our technologies to help as many patients as possible.”

Last week, Otsuka signed an agreement to acquire Mindset Pharma, a Canada-based drug discovery and development company, for a total consideration of CAD80m ($59.2m).