Otsuka, ShapeTX collaborate to develop novel AAV gene therapies for ocular diseases

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Otsuka Pharmaceutical Co., Ltd. and ShapeTX 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.

According to a news release, the partners will join forces 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.

The news release noted that 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.

Moreover, the companies noted in the news release that 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, the companies noted 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.

Under the terms of the agreement, shared in the news release, 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.

According to the news release, ShapeTX also is set to gain tiered royalties on future sales of products resulting from the collaboration.

Francois Vigneault, PhD, co-founder and chief executive officer of ShapeTX, said the company has developed its AAVid platform on generative AI approaches akin to those behind Midjourney and DALL-E 2 to tackle industry challenges with gene therapy delivery.

“By incorporating diffusion models, our platform is designing novel medicines that transcend the boundaries of what is possible experimentally.,” Vigneault said in the news release. “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, PhD, executive director, head of Osaka Research Center for Drug Discovery at Otsuka Pharmaceutical, pointed out in the news release the company 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,” Sudo said in the news release. “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.”