Researchers from the University of Southern California’s Keck School of Medicine are receiving up to $6.8 million for a two-year research project to develop new computational models and support tools that could accelerate access to cell and gene therapies for children with rare diseases.
The team will develop a new framework that combines detailed data about the biological features of each therapy and how patients respond to them. By using AI to study these connections, the project aims to better understand how specific features of a therapy relate to patient outcomes.
The research is funded by the Advanced Research Projects Agency for Health (ARPA-H) UNIfying Cell Therapy Outcome prediction and Regulatory Navigation (UNICORN) project, led by ARPA-H Program Manager Daria Fedyukina, Ph.D.
Ultimately, the aim — based on research recently published in Nature Medicine — is to build a blueprint for developing effective bespoke gene therapies.
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