Hospital to take unprecedented step after drug firm pulled out despite successful trial of treatment
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.
About Author
More Stories
Virologist Wendy Barclay: ‘Wild avian viruses are mixing up their genetics all the time. It’s like viral sex on steroids’
Microsoft unveils chip it says could bring quantum computing within years
Trump administration retreats in fight against Russian cyber threats