Gerard Platenburg

Company: ProQR

Job title: Chief Scientific Officer

Seminars:

Advancing RNA Editing Therapeutics for Rett Syndrome: Targeting the Root Cause with CNS-Directed Oligonucleotides 8:00 am

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…Read more

day: Day 2