NHS trust bids to take license for abandoned gene therapy

An NHS trust has taken the pioneering step of applying to hold the marketing authorisation for a gene therapy for a rare disease that was abandoned by a biotech developer for commercial reasons.

Great Ormond Street Hospital (GOSH) in London is piloting the approach with a lentiviral gene therapy for the rare genetic disorder ADA-SCID, which causes children to be born without a properly functioning immune system. ADA-SCID affects up to three children every year and leaves them very vulnerable, meaning even minor infections can become life-threatening.

The gene therapy was developed at GOSH, UCL Great Ormond Street Institute of Child Health, and the University of California Los Angeles, and was licensed to biotech company Orchard Therapeutics.

The company abandoned the programme in 2021, however, saying it had run into technical issues that undermined its ability to take it forward, despite encouraging results in a trial involving 50 subjects published in the New England Journal of Medicine.

GOSH is being supported in its endeavour by GOSH Charity and non-profit medical research organisation and charity LifeArc, as well as contract research organisation Parexel. The partners say this is the first move of its kind in the UK, and could allow patients to benefit from therapies that have been shown to work, but have no commercial sponsor.

The hospital wants to explore if it can take on the licences for these treatments to make them accessible to children at GOSH, as well as to other clinical centres via collaborative NHS agreements.

“For rare and ultra-rare diseases, it’s a sad fact that there is little commercial reason for companies to develop costly treatments with very small markets,” commented Professor Claire Booth, a specialist in gene therapy and paediatric immunology at GOSH.

“It’s simply not good enough that we have treatments that we know work, but we can’t get them to our patients,” she added, noting that GOSH has the facilities in place to manufacture them and the collaborations in place to allow them to be used more widely. “We must do something radical to tackle this.”

The model, if successful, could be applied to other treatments, according to the partners, who point out that there are around 3.5 million people in the UK who live with a rare disease.

The project has been welcomed by Maria, the mother of healthy seven-year-old daughter Sarah who was fortunate to be included in the clinical trial of the ADA-SCID gene therapy after being diagnosed at just a few weeks old.

“We have always felt quite lucky that Sarah got the treatment when she did, but it was only through the research programme that she got it,” said Maria.

“To think that we now know the gene therapy can work and families still can’t get it seems senseless,” she added. “We hope that this new project at GOSH will mean that more families like us can get transformative treatments for rare diseases without uncertainty.”