Otsuka and ShapeTX Collaborate to Develop AAV Gene Therapies for Ocular Diseases

Otsuka Pharmaceutical and ShapeTX, a programmable medicine company using artificial intelligence (AI) and RNA to combat genetic diseases, announced a multitarget collaboration to develop intravitreally delivered adeno-associated viruses (AAVs) for ocular diseases, with options to add additional targets and tissue types. The companies will apply ShapeTX's AAVid capsid discovery platform and transgene engineering technology to Otsuka’s genetic payload design and ophthalmology products.

Under the terms of the agreement, ShapeTX will receive an initial payment from Otsuka and is eligible to receive development, regulatory, and sales milestone payments potentially exceeding $1.5 billion in aggregate value. ShapeTX is also eligible to receive tiered royalties on future sales of products resulting from the collaboration, advised the companies.

According to the companies, ShapeTX's AI-driven AAVid platform combines throughput screening of billions of unique AAV variants and machine learning to identify novel AAV capsids for direct-to-NHP in vivo selection to maximize clinical translation. AAVid capsids, which are designed for precise target tropism while detuning for off-target biodistribution, reducing the required dose and associated clinical safety risks, noted the companies.

As part of the collaboration, ShapeTX will also apply its transgene engineering technology to optimize payloads provided by Otsuka for therapeutic levels of gene expression in targeted cell types.