Otsuka, ShapeTX to develop gene therapies for eye diseases

Otsuka Pharmaceutical has entered a multi-target partnership with ShapeTX for the development of new adeno-associated viruses (AAV) gene therapies to treat ocular diseases.

The parties will focus on developing intravitreally delivered AAVs.

The AAVid capsid discovery platform and transgene engineering technology of ShapeTX will be combined with the capabilities of Otsuka in genetic payload design and ophthalmology.

ShapeTX is entitled to receive an initial undisclosed payment from Otsuka.

Otsuka will also make developmental, regulatory and sales-based milestone payments totalling more than $1.5bn.

Based on potential sales of products developed by the partnership, ShapeTX will also receive tiered royalty payments.

ShapeTX’s artificial intelligence (AI)-powered AAVid platform merges throughput screening of numerous AAV variants and machine learning to detect new AAV capsids for direct-to-NHP in vivo selection to enhance clinical translation. 

ShapeTX will also use its transgene engineering technology to optimise payloads of Otsuka for therapeutic gene expression levels in directed cell types.

The collaboration offers options to include other targets and tissue types in the future.

Otsuka Pharmaceutical Osaka Research Center for Drug Discovery executive director and head Toshiki Sudo stated: “Our recent research activities have led to the identification of target molecules and antibodies for specific ocular diseases of interest with high unmet medical needs.

“Our collaboration with ShapeTX aims to enable the 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.

“This holds the potential to become dramatically beneficial to patients who have suffered from specific chronic ocular diseases.”

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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