Research 🇬🇧 Directly KS related

IDefine Joins Multi-Foundation Research Collaboration with Johns Hopkins to Advance Novel RNA Therapeutic Platform

person IDefine

calendar_today Jul 28, 2026

IDefine has joined a research collaboration led by COMBINEDBrain and Johns Hopkins University to test a new RNA-based therapeutic platform for Kleefstra syndrome and four other rare neurodevelopmental disorders. The approach uses “mRNA boosters” to increase gene expression from the remaining functional gene copy in haploinsufficiency disorders. Researchers will evaluate the technology in patient-derived cellular models, aiming to advance a shared treatment strategy across multiple conditions.