USC team develops new AI model to advance rare disease therapies

USC team develops new AI model to advance rare disease therapies UNICORN project uses AI to analyze therapies and improve outcomes Written by Andrea Lobo, PhD | April 8, 2026   Print   Preferred Source on Google Researchers at the Keck School of Medicine, University of Southern California, are receiving up to $6.8 million in funding to develop an artificial intelligence (AI)-based computational model to help advance gene and cell therapies for children with rare diseases, including conditions such as aromatic L-amino acid decarboxylase (AADC) deficiency. Funded by the Advanced Research Projects Agency for Health (ARPA-H), the two-year project, called “UNICORN: UNIfying Cell Therapy Outcome prediction and Regulatory Navigation,” aims to understand how specific biological characteristics of therapies are linked to patient outcomes. Recommended Reading March 18, 2026 News by Marisa Wexler, MS Genetic testing may help tailor medications for rare disease patients AI project aims to improve how rare disease therapies are developed “This project reimagines how we develop therapies for rare diseases,” Mohamed Abou-el-Enein, MD, PhD, principal investigator of the new project and executive director of the USC/Children’s Hospital Los Angeles Cell Therapy Program, said in a university news story. “By working closely with colleagues across the field, we are building a scientific foundation that helps translate complex biological data into clearer evidence and guides the creation of mor...