January 14, 2022
1 min read

Acadia, Stoke to develop treatments for rare CNS diseases

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Acadia Pharmaceuticals Inc. and Stoke Therapeutics Inc. announced they will collaborate to develop novel RNA-based medicines as potential treatments for severe and rare genetic neurodevelopmental central nervous system diseases.

The biotechnology companies will focus on SYNGAP1 syndrome, Rett syndrome and an undisclosed neurodevelopmental target of mutual interest.

“Stoke’s RNA-based approach to upregulating healthy proteins offers very exciting new possibilities for the treatment of rare, neurodevelopmental diseases like Rett syndrome,” Steve Davis, CEO of Acadia, said in a press release. “Combining Stoke’s capabilities with Acadia’s extensive expertise in neuroscience drug development and commercialization enables us to push harder and faster in exploring some of the new frontiers in rare central nervous system disorders. We are excited to have the opportunity to further build our Rett syndrome franchise and pursue treatments in SYNGAP1 syndrome and other neurodevelopmental disorders.”

The companies will have joint global research, development and commercialization responsibilities for SYNGAP1. Stoke will lead research and preclinical development activities, and Acadia will lead clinical development and commercialization activities for Rett syndrome and the undisclosed neurodevelopmental program.

“Rett syndrome and SYNGAP1 syndrome are two severe, intractable diseases of the central nervous system, and both are associated with developmental delays, motor function loss, autism, seizures and other comorbidities that impact quality of life for patients and their families,” Edward M. Kaye, MD, CEO of Stoke Therapeutics, said in the release. “Acadia shares our deep commitment to improving outcomes for people living with neurodevelopmental disorders. We believe their late-stage development and commercialization capabilities, in addition to their neuroscience expertise, complement our discovery research efforts and clinical learnings from our work in Dravet syndrome.”