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

▤ Summary

IDefine and UT Southwestern have formed a two-year research collaboration to advance a potential gene therapy for Kleefstra syndrome. The program will test the feasibility and safety of replacing the EHMT1 gene using next-generation CNS-targeted delivery technology. IDefine will contribute about $310,000 to support the pre-clinical work through April 2028. The effort aims to move closer to a first treatment for a rare neurodevelopmental disorder that currently has no approved therapies.

▦ Linked Companies