Overview
- Three-year-old Oliver Chu of California became the first patient to receive the Manchester-developed, one-off stem-cell gene therapy at Royal Manchester Children’s Hospital.
- The procedure harvests the child’s stem cells, inserts a working IDS gene via a viral vector, and reinfuses corrected cells designed to produce the missing enzyme throughout the body and into the brain.
- Follow-up tests show very high enzyme levels—reported as hundreds of times normal—and Oliver has paused weekly Elaprase infusions as his speech, mobility, and cognitive measures improve.
- Researchers emphasize early results require caution, with five boys enrolled from the US, Europe, and Australia who will be followed for a minimum of two years.
- The program was built over more than a decade at the University of Manchester, advanced with Great Ormond Street Hospital and academic partners, and sustained by LifeArc funding after a prior commercial setback.