Otsuka and Shape Therapeutics (ShapeTX) collaborate to develop intravitreally-delivered adeno-associated viruses (AAVs) for ocular diseases.
Otsuka Pharmaceutical Co., Ltd. and ShapeTX, the programmable medicine company using AI and RNA to end genetic diseases, announce a multi-target collaboration to develop intravitreally-delivered adeno-associated viruses (AAVs) for ocular diseases, with options to add additional targets and tissue types. The companies will collaborate to apply ShapeTX’s AAVid capsid discovery platform and transgene engineering technology along with Otsuka’s expertise in genetic payload design and ophthalmology to develop novel treatment options for people living with serious eye diseases
ShapeTX’s AI-driven AAVid platform combines massive 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 are designed for precise target tropism while detuning for off-target biodistribution, thereby reducing the required dose and associated clinical safety risks. As part of the collaboration, ShapeTX will also apply the company’s transgene engineering technology to optimize payloads provided by Otsuka for therapeutic levels of gene expression in targeted cell types.
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