Špela Miroševič, PhD, is the Founding President of the CTNNB1 Foundation, a non-profit developing gene therapy for CTNNB1 syndrome. After her son Urban was diagnosed with the disorder in 2020, she left her career in cancer and public health research to pursue drug development for this rare condition. She has since led the Foundation's AAV9 gene therapy program from preclinical work into the clinic, raising over $5 million in philanthropic funding plus a $2 million SPARK NS grant. The first Phase I/II clinical trial was authorized in Europe in December 2025; two children have been treated so far, with about 20 more expected as the trial expands to Spain, the U.S., and Australia. She also co-leads the Dragonfly Natural History Study with Prof. Damjan Osredkar, which evaluates more than 150 children every year, and has helped change Slovenian law to enable funding for early-stage gene therapy trials.