Great Ormond Street hoping to license gene therapy for ‘bubble baby’ syndrome

^{Photo by Kind and Curious from Unsplash}

When Great Ormond Street hospital (Gosh) published the results of its gene therapy trial for “bubble baby” syndrome it was hailed as a medical breakthrough. The treatment had a more than 95% success rate for treating the life-threatening disorder in which children have no immune system. But less than a year later, the therapy had been dropped by the pharmaceutical company that planned to bring it to market.

Now, Gosh is taking the unprecedented step of attempting to license the therapy itself on a non-profit basis and without industry involvement, in order to make it more widely available to babies and children worldwide.

“We’ve been developing the treatments, seeing the amazing results in trial and then we hit this roadblock,” said Prof Claire Booth, of Gosh and University College London, who was a principal investigator on the trial. “We’re seeing more and more companies pulling out of the field. So we needed to find a way to get it to patients.”

The move comes amid concern about the affordability of gene therapies...