Gerard Platenburg
Chief Scientific Officer & Co-Founder ProQR Therapeutics
Seminars
Saturday 17th January 2026
Advancing RNA Editing Therapies for CNS Disorders: Insights from a Rett Syndrome Program
1:30 pm
- Application of RNA-based editing technologies to achieve targeted modulation within the CNS, including brain distribution and editing characteristics distinct from knockdown approaches
- Translational preclinical evidence from disease models and non-human primate studies supporting progression toward clinical development
- Case study of a Rett syndrome program, highlighting the data package and strategic considerations that led to candidate nomination and IND-enabling plans
