Otsuka and ShapeTX Forge Dynamic Partnership to Pioneer AAV Gene Therapies in Ophthalmology with AI

TL;DR:

  • Otsuka Pharmaceutical and ShapeTX join forces for multi-target collaboration in ocular disease treatments.
  • AAVid capsid discovery platform and transgene engineering technology play pivotal roles in the partnership.
  • AI-driven AAVid platform identifies novel AAV capsids, optimizing clinical translation.
  • AAVid capsids were designed for precise target tropism and reduced clinical safety risks.
  • ShapeTX to fine-tune gene expression levels in targeted cell types.
  • ShapeTX receives substantial initial payments and potential milestone payments exceeding $1.5 billion.
  • ShapeTX stands to gain tiered royalties from future product sales.

Main AI News:

In an unprecedented collaboration, Otsuka Pharmaceutical Co., Ltd. and ShapeTX are poised to revolutionize the field of ocular disease treatments. Their multi-target endeavor focuses on the development of adeno-associated viruses (AAVs) for intravitreal delivery, with the possibility of incorporating additional targets and tissue types down the line.

This strategic partnership leverages ShapeTX’s cutting-edge AAVid capsid discovery platform and transgene engineering technology, synergizing with Otsuka’s unparalleled genetic payload design expertise and extensive background in ophthalmology. The primary goal? To pioneer innovative therapeutic options for individuals grappling with severe eye conditions.

ShapeTX’s AAVid platform, powered by artificial intelligence, stands as a game-changer. It combines high-throughput screening of countless unique AAV variants with machine learning to unearth novel AAV capsids, allowing for direct-to-NHP in vivo selection and optimizing clinical translation.

Furthermore, the collaboration’s vision extends beyond mere discovery. AAVid capsids are meticulously tailored for precise target tropism while minimizing off-target biodistribution—a crucial move that reduces the necessary dosage and associated clinical safety risks. As part of the partnership, ShapeTX will also employ its transgene engineering technology to fine-tune the payloads provided by Otsuka, ensuring therapeutic levels of gene expression in targeted cell types.

The agreement’s terms, as disclosed in the news release, dictate that ShapeTX will receive an initial payment from Otsuka and could potentially accrue development, regulatory, and sales milestone payments exceeding an astonishing $1.5 billion in total.

Moreover, ShapeTX is positioned to receive tiered royalties on future product sales stemming from this groundbreaking collaboration.

Francois Vigneault, PhD, co-founder and CEO of ShapeTX, affirmed the company’s commitment to pushing the boundaries of gene therapy delivery. “Our AAVid platform, inspired by generative AI techniques akin to those driving Midjourney and DALL-E 2, is pioneering the development of medicines previously deemed experimentally impossible,” Vigneault stated in the press 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.”

Dr. Toshiki Sudo, Executive Director and Head of the Osaka Research Center for Drug Discovery at Otsuka Pharmaceutical, emphasized the company’s extensive drug discovery prowess across various therapeutic domains, including central nervous systems (CNS), nephrology, and ophthalmology. “Our recent research endeavors have led to the identification of target molecules and antibodies tailored to specific ocular diseases with pressing unmet medical needs,” Sudo noted. “Our collaboration with ShapeTX aims to enable the delivery of vectorized antibody drugs in conjunction with AAV, with the ultimate goal of targeting specific disease cell types in the eye and offering a curative administration with lifelong stability. This breakthrough holds tremendous promise for patients grappling with specific, chronic ocular diseases.”

Conclusion:

This groundbreaking partnership between Otsuka and ShapeTX signifies a significant leap forward in the development of advanced gene therapies for ocular diseases. The utilization of AI-driven technology and precise targeting strategies holds promise not only for patients suffering from chronic eye conditions but also for the broader gene therapy market, where innovative approaches are becoming increasingly essential for addressing unmet medical needs. This collaboration sets a high bar for future advancements in ocular therapeutics and underscores the growing significance of AI-driven solutions in the pharmaceutical industry.

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