Hunter syndrome, how to apply lessons learned when treating similar diseases | LATSS 2026

Speaker: Rob Wynn (Royal Manchester Children's Hospital) Synopsis: Bone marrow transplantation can correct rare lysosomal storage diseases, as engrafted donor-derived white blood cells supply the deficient enzyme to host tissues. Here, in one such disease - MPSII (Hunter syndrome) - we will describe genetic correction of a patient’s own blood cells, so that the patient becomes their own transplant donor. This approach is both safer and more effective than conventional transplantation as the cells are engineered to produce much higher levels of enzyme than those from a normal donor. This supraphysiological enzyme delivery results in superior disease corrections. It is also safer than conventional bone marrow transplantation as it requires less immuno-suppression. This closing talk will celebrate the dramatic results from the first three cases in a phase I/II clinical trial conducted in Manchester and will reflect on how this experience offers valuable insights for the advancement of future treatments for patients with similar genetic conditions. This talk was recorded at the LifeArc Translational Science Summit 2026. ------------------------------------- LifeArc is a self-funded medical research organisation with one clear purpose – transforming the lives of people living with rare diseases and drug-resistant infections. We aim to deliver this by conducting and funding pioneering research, and working with partners to accelerate the translation of scientific breakthroughs into new tests and treatments. Our vision is a world where people living with rare diseases and drug-resistant infections can access effective, life-changing treatments faster. Find out more at lifearc.org