Otsuka and Shape Join Forces in a Visionary Quest: $1.5 Billion Deal to Pioneer Eye AAV Gene Therapies

Otsuka and Shape Join Forces in a $1.5 Billion Vision to Revolutionize Ocular Gene Therapies

In an electrifying development, Japan’s Otsuka Pharmaceuticals unveiled a transformative collaboration with RNA editing innovator, Shape Therapeutics, to pioneer intravitreal adeno-associated virus (AAV) gene therapies for ocular diseases.

While precise financial details remain cloaked, the partnership’s potential aggregate value exceeds a staggering $1.5 billion, encompassing an upfront payment from Otsuka and potential future payments linked to development, regulatory milestones, and sales achievements. Seattle-based Shape Therapeutics will also earn tiered royalties on future sales of any product born from this groundbreaking partnership.

In return for their investment, Otsuka gains coveted access to Shape’s AI-powered AAVid capsid discovery platform and state-of-the-art transgene engineering technology. Remarkably, this collaboration empowers Otsuka to expand its horizons by targeting additional disease cell types within the eye.

AAVid, propelled by the magic of artificial intelligence, engineers capsids with unprecedented precision, minimizing off-target biodistribution. The result? Lower gene therapy doses and an enhanced safety profile—a breakthrough poised to redefine the gene therapy landscape.

Francois Vigneault, Shape’s CEO, praised AAVid’s AI capabilities for pushing the boundaries of gene therapy delivery, transforming possibilities into realities.

This pioneering partnership also harnesses Shape’s transgene engineering prowess to optimize therapeutic payload expression levels—a vital step in the journey towards curative administrations.

Otsuka’s strategic pivot towards ophthalmology is evident, particularly following challenges in its neurology business, marked by back-to-back Phase III setbacks in July 2023. Otsuka’s investigational TAAR1 agonist ulotaront, developed in collaboration with Sumitomo Pharma, fell short of expectations in schizophrenia trials.

Thursday brought another Phase III disappointment as a fixed-dose trial featuring Otsuka’s brexpiprazole, in combination with sertraline, failed to significantly alleviate symptom severity in post-traumatic stress disorder patients, compared to placebo. However, the Otsuka-Shape alliance signals a new chapter, filled with promise and the potential to reshape the future of ocular therapies.

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