Gene Therapies for Neurological Diseases Will Be Focus of New Shape Therapeutics, Roche Collaboration


The 2 companies will focus their strategic partnership on the development of gene therapies for rare diseases, as well.

Shape Therapeutics announced that it is collaborating with Roche to develop novel adeno-associated virus (AAV)- based RNA editing technology for the treatment of neurological diseases such as Alzheimer disease (AD), Parkinson disease (PD), as well as rare disease targets.1

“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, PhD, co-founder and chief executive officer, ShapeTX, in a statement.1 “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 partnership will see Shape using its RNA editing platform RNAfix and its AAVid technology platform for next-generation tissue-specific AAVs to conduct preclinical research, with the goal of identifying and delivering candidates for gene therapies. Roche will develop and commercialize any gene therapy products developed.

“We are excited by the disruptive potential of ShapeTX’s RNA-editing approach based on nature’s own mechanism for specific base editing. This new collaboration is also perfectly aligned with our broader efforts across the Roche Group to unlock the full potential of gene therapy,” added James Sabry, head, Roche Pharma Partnering, to the statement.1 “We look forward to working with ShapeTX to create novel treatment options for neuroscience and rare disease indications.”

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Shape, using its novel ShapeTX AI engine, is targeting therapeutic development in neurology, including AD and PD, ophthalmology, including Stargardt disease, retinitis pigmentosa, and age-related macular degeneration, neuromuscular disorders such as Duchenne muscular dystrophy, and other rare diseases such as cystic fibrosis.

Other similar collaborations have occurred recently, with ViGeneron and Biogen announcing in January 2021 that they would co-develop AAV-based gene therapies for eye disease.2 That partnership plans to use ViGeneron’s proprietary vgAAV technology and novel engineered AAV capsids to efficiently transduce retinal cells via intravitreal injections.

That partnership will see ViGeneron optimizing and validating in vitro therapeutic candidates for an undisclosed target to treat inherited eye disease. Biogen has the right to add an additional reserved target within 2 years after the effective date. The companies will work together on the in vivo proof of concept and Biogen will be responsible for all further development and commercialization of the selected therapeutic candidates.

“This collaboration exemplifies our strategy to develop in-house programs for selected retinal targets, while maximizing our proprietary technology platforms with additional collaboration programs for other targets in ophthalmology and further indications,” Caroline Man Xu, MD, co-founder and chief executive officer, ViGeneron, said in a statement at that time.2 “ViGeneron’s recognized expertise in retinal gene therapy together with Biogen’s leading research, drug development and commercialization experience is a powerful combination that we believe will allow us to deliver more novel gene therapies to patients in need.”

1. Shape Therapeutics enters into a strategic research collaboration with Roche to advance breakthrough AAV-based RNA editing technology for neuroscience and rare disease indications. News release. Shape Therapeutics. August 24, 2021.
2. ViGeneron signs global collaboration agreement for ophthalmic gene therapy development. News release. ViGeneron. January 5, 2021. Assessed August 25, 2021.
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