Pacs2 Research Foundation

The non-profit organization dedicated to advance therapeutics development and improve lives of children affected by ultra rare neurodevelopmental disorder, epileptic encephalopathy, PACS2 syndrome. We fund and coordinate scientific research run globally. We plan first clinical trials for 2026. We are parents of little Lena living with PACS2 syndrome- Established in May 2022, we have already made available for scientific community various cell and animal models. Currently, we are actively coordinating eight projects that span the spectrum from basic science to drug repurposing and exploration of RNA editing. Interdisciplinary & international collaboration is at the core of our approach, and we are proud to partner with institutions such as: Oxford University (UK), Northwestern University (US), Captor Therapeutics, Warsaw Medical University (Poland), Medical University of Bialystok (Poland), 2 Institutes of Polish Academy of Science: The Nencki Institute of Experimental Biology and The Institute of Biochemistry and Biophysics, Charles River Laboratories and Perlara PBC. Having established connections with approximately 60-70 families out of the 100 diagnosed patients, we are now also planning a natural history study. For a comprehensive overview, visit