IDefine

We are excited to announce a new research collaboration with UT Southwestern Medical Center aimed at developing a potential to treat (KS).

Led by Steven Gray, Ph.D., professor at UT Southwestern and director of the UTSW Gene Therapy Program, this two-year research effort will explore a possible treatment approach using advanced gene therapy technology designed to target the central nervous system. To learn more, check out our press release here: https://www.prnewswire.com/news-releases/idefine-and-ut-southwestern-announce-research-collaboration-to-advance-gene-therapy-for-rare-disease-kleefstra-syndrome-302789121.html?tc=eml_cleartime

This collaboration represents an important step forward for our community and reflects the growing momentum behind research that could lead to a first therapy for KS. By combining Dr. Gray and the UT Southwestern team’s scientific expertise with our commitment to accelerating meaningful research, this work has the potential to help shape treatment possibilities for individuals and families impacted by KS.

We are incredibly grateful to every family and advocate across the KS community whose ongoing support, encouragement, and dedication continue to make this progress possible. While there is still important work ahead, we are hopeful about what this research may mean for the future.