Otsuka Pharmaceutical Co., Ltd. (Otsuka) and ShapeTX, a programmable medicine company that uses AI and RNA to end genetic diseases, announced today a multi-target collaboration to develop intravitreally-delivered adeno-associated viruses (AAVs) for ocular diseases, with the option to add additional targets and tissue types.
The firms will work together to explore novel therapeutic options for people suffering with critical eye disorders using ShapeTX‘s AAVidTM capsid discovery platform and transgene engineering technology, as well as Otsuka’s experience in genetic payload design and ophthalmology.
Terms of the Agreement between Otsuka and ShapeTX :
According to the terms of the agreement, ShapeTX will receive an initial payment from Otsuka and will be eligible for research, regulatory, and sales milestone payments totaling up to $1.5 billion. ShapeTX may also be eligible for tiered royalties on future product sales stemming from the agreement.
ShapeTX‘s AI-driven AAVid platform:
ShapeTX’s AI-driven AAVid platform combines huge throughput screening of billions of distinct AAV variants with machine learning to uncover novel AAV capsids for in vivo direct-to-NHP selection to maximise clinical translation.
AAVid capsids are engineered to achieve accurate target tropism while detuning for off-target biodistribution, lowering the needed dose and related clinical safety hazards. ShapeTX will also use its transgenic engineering expertise to optimise payloads provided by Otsuka for therapeutic levels of gene expression in targeted cell types as part of the agreement.
“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.”
About Otsuka:
Otsuka Pharmaceutical Co., Ltd. is a global healthcare firm whose corporate philosophy is “Otsuka-people creating new products for better health around the world.” Otsuka conducts research, develops, manufactures, and markets new goods, with an emphasis on pharmaceutical solutions that address unmet medical needs and nutraceutical products that promote daily health.
In pharmaceuticals, Otsuka is a leader in the challenging fields of mental, renal, and cardiovascular health, as well as oncology and numerous under-addressed diseases such as tuberculosis, a major global public health issue. These commitments demonstrate how Otsuka is a “big venture” firm at heart, infusing all it undertakes with a youthful spirit of inventiveness.
About ShapeTX:
ShapeTX is a leader in developing programmable RNA therapies to treat disease’s genetic origins. ShapeTX is pioneering advances in RNA editing, next-generation AAVs, and gene therapy manufacturing by combining innovations in AI and RNA technology to generate and analyse hundreds of billions of therapeutic possibilities. The ShapeTX platform enables pharmaceutical innovators to create medicines for a wide range of diseases, including uncommon genetic disorders and devastating conditions like Alzheimer’s and Parkinson’s.
