Research 🇬🇧 Directly KS related

IDefine and UT Southwestern Announce Research Collaboration to Advance Gene Therapy for Rare Disease Kleefstra Syndrome

person IDefine - The Kleefstra Syndrome Foundation

calendar_today Jun 3, 2026

IDefine and UT Southwestern launched a two-year, $310,000 preclinical research collaboration led by Dr. Steven Gray to evaluate EHMT1 gene replacement therapy for Kleefstra syndrome, focusing on CNS-targeted delivery, feasibility, safety, and future treatment development.