Otsuka Partners with ShapeTX to Develop AAV Gene Therapies for Ocular Diseases

Otsuka Pharmaceutical and ShapeTX have announced a comprehensive collaboration aimed at developing intravitreally-delivered adeno-associated viruses (AAVs) for the treatment of ocular diseases, with the flexibility to explore additional targets and tissue types in the future.

The collaboration will harness ShapeTX's AAVidTM capsid discovery platform and transgene engineering technology, complemented by Otsuka's expertise in genetic payload design and ophthalmology, to create innovative therapies for individuals grappling with severe eye conditions.

ShapeTX's AAVid platform, powered by artificial intelligence, leverages extensive screening of billions of unique AAV variants and machine learning. This approach enables the identification of novel AAV capsids optimized for direct-to-NHP in vivo selection, thus enhancing clinical translation. AAVid capsids are meticulously engineered for precise target tropism while minimizing off-target biodistribution, ultimately reducing the required dosage and associated clinical safety concerns.

As part of this collaboration, ShapeTX will also apply their transgene engineering technology to fine-tune the payloads provided by Otsuka, ensuring therapeutic levels of gene expression in specific cell types.

According to the terms of the agreement, ShapeTX will receive an initial payment from Otsuka and remains eligible for development, regulatory, and sales milestone payments that have the potential to exceed a cumulative value of $1.5 billion. Additionally, ShapeTX stands to receive tiered royalties on future sales of products resulting from this groundbreaking 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, PhD, co-founder and chief executive officer of ShapeTX. "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, Ph.D., executive director, head of Osaka Research Center for Drug Discovery at Otsuka Pharmaceutical commented, "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 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."