Research and Innovation

Gene therapy treatment manufactured at GOSH and ICH used to treat world's first patient with life limiting genetic condition, MPSIIIA

A two-year old patient at the Royal Manchester Children’s Hospital (RMCH) is the first in the world to receive a pioneering new gene therapy treatment for the rare and life-limiting genetic condition, Mucopolysaccharidosis IIIA (MPSIIIA). The treatment was manufactured at Great Ormond Street Hospital (GOSH) and the UCL Great Ormond Street Institute of Child Health (ICH) after being developed by RMCH and the University of Manchester.