Otsuka Pharmaceutical Co., Ltd. and Shape, a programmable medicine company using AI and RNA to target genetic diseases, partnered to develop intravitreally delivered adeno-associated viruses (AAVs) for ocular diseases, with options to add additional targets and tissue types. The companies will apply Shape’s AAVid capsid discovery platform and transgene engineering technology with Otsuka’s expertise in genetic payload design and ophthalmology to develop novel treatments for serious eye diseases.

Shape’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, Shape 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.

Shape will receive an initial payment from Otsuka and is eligible to receive development, regulatory, and sales milestone payments potentially exceeding $1.5 billion. Shape is also eligible to receive royalties on sales of products resulting from the collaboration.

“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,” said Francois Vigneault, co-founder, and CEO of Shape. “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.”

Toshiki Sudo, executive director, head of Osaka Research Center for Drug Discovery at Otsuka Pharmaceutical, 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 Shape aims to enable delivery of vectorized 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.”