Otsuka collaborates with ShapeTX to develop novel AAV gene therapies for ocular diseases

Otsuka Pharmaceutical Co., Ltd. (Otsuka) and ShapeTX, the programmable medicine company using AI and RNA to end genetic diseases, today 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. The companies will collaborate 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.

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. 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, 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, 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. ShapeTX is also eligible to receive tiered royalties on future sales of products resulting from the collaboration.

“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 Pharmaceutical

Otsuka Pharmaceutical Co., Ltd. is a global healthcare company with the corporate philosophy, Otsuka–people creating new products for better health worldwide. Otsuka researches, develops, manufactures, and markets innovative products, with a focus on pharmaceutical products to meet unmet medical needs and nutraceutical products for the maintenance of everyday health.

In pharmaceuticals, Otsuka is a leader in the challenging areas of mental, renal, and cardiovascular health and has additional research programs in oncology and on several under-addressed diseases including tuberculosis, a significant global public health issue. These commitments illustrate how Otsuka is a “big venture” company at heart, applying a youthful spirit of creativity in everything it does.

About ShapeTX

ShapeTX is pioneering programmable RNA medicines to repair the genetic causes of disease. By merging innovations in AI and RNA technology to generate and analyze hundreds of billions of therapeutic possibilities, ShapeTX is developing breakthroughs in RNA editing, next-generation AAVs, and gene therapy manufacturing. The ShapeTX platform enables pharma innovators to design treatments across a wide range of diseases, including rare genetic disorders as well as debilitating conditions, such as Alzheimer’s, Parkinson’s, and many more.

PackGene is a CRO & CDMO technology company that specializes in packaging recombinant adeno-associated virus (rAAV) vectors. Since its establishment in 2014, PackGene has been a leader in the AAV vector CRO service field, providing tens of thousands of custom batches of AAV samples to customers in over 20 countries. PackGene offers a one-stop CMC solution for the early development, pre-clinical development, clinical trials, and drug approval of rAAV vector drugs for cell and gene therapy (CGT) companies that is fast, cost-effective, high-quality, and scalable. Additionally, the company provides compliant services for the GMP-scale production of AAVs and plasmids for pharmaceutical companies, utilizing five technology platforms, including the π-Alpha™ 293 cell AAV high-yield platform and the π-Omega™ plasmid high-yield platform. PackGene’s mission is to make gene therapy affordable and accelerate the launch of innovative gene drugs. The company aims to simplify the challenging aspects of gene therapy development and industrialization processes and provide stable, efficient, and economical rAAV Fast Services to accelerate gene and cell therapy development efforts from discovery phase to commercialization.

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