UK researchers developing stem cell gene therapy for fatal childhood disease

Scientists in Manchester, UK, who have developed a stem cell gene therapy to reverse a fatal childhood illness, have agreed to work with a new therapeutics company to test it in a human trial.

University of Manchester and Central Manchester University Hospital NHS Foundation Trust (CMFT) researchers have developed the pioneering approach for Sanfilippo disease (also known as mucopolysaccharidosis type III or MPS III) – a genetic condition for which there is currently no effective treatment. The most common of the four types of Sanfilippo (type A) affects around 100 children in the UK, or one in 89,000 births, and it is this type that is targeted by the new treatment.

Licensing deal with Orchard Therapeutics