Otsuka Collaborates with ShapeTX for Development of AAV Gene Therapies

Otsuka Pharmaceutical and ShapeTX announced on Sept.7, 2023 a multi-target collaboration to develop intravitreally delivered adeno-associated viruses (AAVs) for ocular diseases, with the possiblity to add additional targets and tissue types. According to the press release, the companies are collaborating to apply ShapeTX’s AAVid capsid discovery platform and transgene engineering technology in addition to Otsuka’s expertise in genetic payload design and ophthalmology to develop novel treatment options for eye diseases.

The 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. Further, according to the release, AAVid capsids are designed for precise target tropism while detuning for off-target biodistribution, which reduces the required dose and associated clinical safety risks. In addition, ShapeTXTX will 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 new agreement, ShapeTXTX will receive an initial payment from Otsuka and is eligible to receive development, regulatory, and sales milestone payments that will potentially exceed $1.5 billion in aggregate value. ShapeTXTX is also eligible to receive tiered royalties on future sales of products resulting from the collaboration, according to the press release.

“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 ShapeTXTX, in a press release. “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.”

Source: ShapeTX