USC Gets $6.8M to Speed Gene Therapy for Rare Diseases

Children with rare diseases often face an impossible waiting game for treatments that could save their lives, but researchers at USC just received up to $6.8 million to change that.

Scientists at the Keck School of Medicine are building an AI-powered system that could dramatically speed up how gene and cell therapies reach kids who need them most. The project, called UNICORN, tackles one of medicine's toughest challenges: creating personalized treatments when you can't study thousands of patients at once.

Gene and cell therapies work by engineering a patient's own cells to fight diseases like childhood cancer and genetic disorders at the cellular level. But unlike regular drugs manufactured in huge batches, these therapies are created one patient at a time in highly controlled labs, making them expensive and difficult to study through traditional clinical trials.

Dr. Mohamed Abou-el-Enein, who leads the project, and his team are combining advanced cell analysis technology with machine learning to spot biological patterns that predict which treatments will work best. By studying detailed data about how therapies are made and how patients respond, the AI can learn from every single child treated, even when the patient groups are small.

The researchers will collect samples and data from pediatric patients at multiple U.S. medical centers, analyzing everything from CAR T cell therapies that train immune cells to attack blood cancers to treatments that repair genetic defects directly. Each child's treatment adds valuable information that makes the model smarter for the next patient.

The Ripple Effect

This approach doesn't just help individual kids. It's creating a blueprint that other researchers can use to develop small-batch therapies more efficiently. Every successful treatment teaches the system something new, building a growing knowledge base for rare disease research.

The project represents a fundamental shift in how medicine approaches orphan diseases, those affecting so few people that traditional drug development doesn't make economic sense. By making better predictions with less data, UNICORN could open doors for treatments that might never have made it through conventional testing.

The team is building on technology developed right at USC, including an electronic system called Bluecord that tracks samples and integrates data securely across participating centers. This standardization means insights from one hospital can strengthen predictions at another, creating a network effect that benefits everyone.

For families watching their children battle rare diseases, time isn't just money. The framework aims to provide the evidence regulators need to approve safe, effective therapies without requiring the massive patient numbers that simply don't exist for rare conditions.

What started as a tool to improve one type of cancer treatment is now becoming a living, learning system designed to bring hope to the rarest patients of all.