Restoring Splicing Function in DM1 by Selectively Targeting Toxic CUGRepeat RNA

• Selectively targeting toxic CUG-repeat RNA in DM1
• Disrupting RNA foci to release MBNL1
• Linking target engagement to restored splicing function
• Building confidence in potency, selectivity and disease relevance

Speakers:

Domi Stickens

Chief Scientific Officer

Arrakis Therapeutics Inc.