Shape and Roche Collaborate to advance AAV-based RNA Editing for Neuroscience and Rare Diseases

Shape Therapeutics and Roche entered into a multi-target strategic collaboration and license agreement in which Shape will apply its proprietary RNA editing platform and potentially leverage its technology platform for next-generation tissue-specific adeno-associated viruses (AAVs) for the development of gene therapy for certain targets in the areas of Alzheimer’s disease, Parkinson’s disease, and rare diseases.

ShapeTX will conduct preclinical research to identify and deliver development candidates discovered by its AI-powered platforms RNAfix and, potentially, AAVid. Roche will be responsible for the development and worldwide commercialization of any potential products resulting from the collaboration.

“Our mission at ShapeTX is to unlock the next breakthrough in RNA technologies in the gene therapy space across a wide range of therapeutic areas,” said Francois Vigneault, co-founder and CEO of ShapeTX. “The relationship with Roche quickly centered on a common desire to tackle some of the world’s most challenging diseases by accelerating the development of breakthrough technologies towards the clinic.”

The ShapeTX gene therapy platform is comprised of RNAskip, RNAfix, and RNAswap payload technologies, next-generation tissue-specific AAVid delivery technology, and SquareBio, a solution for scalable gene therapy manufacturing based on industrialization of human stable cell lines. At the core of these technologies is the ShapeTX AI engine.

Under the terms of the agreement, ShapeTX is eligible to receive an initial payment as well as development, regulatory and sales milestone payments, potentially exceeding $3 billion in aggregate value. ShapeTX is also eligible to receive tiered royalties on future sales of products resulting from the collaboration.

“We are excited by the disruptive potential of ShapeTX’s RNA-editing approach based on nature’s own mechanism for specific base editing,” said James Sabry, head of Roche Pharma Partnering. “This new collaboration is also perfectly aligned with our broader efforts across the Roche Group to unlock the full potential of gene therapy.”

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

Author: Rare Daily Staff