Otsuka and ShapeTX Collaborate to Develop Novel AAV Gene Therapies for Ocular Diseases

Rare Daily Staff

Japanese pharmaceutical Otsuka and programmable medicine biotech ShapeTX have entered into a multi-target collaboration to develop intravitreally-delivered, adeno-associated viruses 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 dosage 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.

“By incorporating diffusion models, our platform is designing novel medicines that transcend the boundaries of what is possible experimentally,” said Francois Vigneault, co-founder and CEO of ShapeTX.

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.

“Our recent research activities have led to identification of target molecules and antibodies for specific ocular diseases of interest with high unmet medical needs,” said Toshiki Sudo, executive director, head of Osaka Research Center for Drug Discovery at Otsuka Pharmaceutical. “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.”

Photo: Francois Vigneault, co-founder and CEO of ShapeTX