Advancing RNA Editing Therapeutics for Rett Syndrome: Targeting the Root Cause with CNS-Directed Oligonucleotides
Time: 8:00 am
day: Day 2
Details:
- Introducing ProQR’s RNA editing platform and its potential to correct disease causing mutations in MECP2, the primary gene implicated in Rett Syndrome
- Highlighting preclinical data demonstrating targeted editing in the brain using CNS-delivery conjugates and oligonucleotide-based strategies
- Discuss the translational path, including biomarker strategies and candidate selection from non-human primate (NHP) studies, to inform clinical entry
- Outline the near-term development timeline with clinical candidate nominations expected this year and next, and reflect on parallel efforts in liver-directed RNA editing
