UCL spinout pioneers rare disease gene therapy to treat children with severe genetic disorders

Orchard Therapeutics, a UCL spinout, is using stell cell gene therapy developed by academics to transform the lives of children with severe genetic conditions.

The company was born out of research that began at UCL in the early 1990s. 

Desperate for better treatments to help children at Great Ormond Street with rare genetic disorders, UCL’s Professors Bobby Gaspar and Adrian Thrasher began working on pioneering gene therapies. 

Professor Gaspar, from UCL’s GOSH Institute of Child Health, explains: “We were looking after kids with severe combined immunodeficiency (SCID), who either died of their disease or had to undergo treatments like bone marrow transplants, which can have significant implications.” 

Bobby and Adrian turned to the emerging science of haematopoietic stem cell gene therapy. The approach uses the patient’s own genetically modified blood stem cells to correct their defective genes, to effectively grow a new immune system.  

After successful clinical trials, the UCL team developed new treatments for two different forms of SCID and began getting referrals from patients around the world.  

The results showed such potential for correcting faulty genes in children, they realised the need to commercialise their work and make it an approved medicine. Orchard Therapeutics was set up in 2015, with help from UCL Business (UCLB), the commercialisation company of UCL and part of UCL Innovation & Enterprise.  

The company has since grown to employing nearly 200 people globally. Orchard’s first treatment was approved by the European Commission in 2020, and several other development programmes are now in clinical trials.  

David Phillips, Senior Business Manager for UCLB, said: “The team behind Orchard are doing really ground-breaking work, with the potential for very significant patient impact. So it’s very exciting to be part of bringing some of the first gene therapies to market.” 

Read the full story on the UCLB website 

Links 

Orchard Therapeutics 
Great Ormand Street Institute of Child Health 
UCLB 
REF case study: Orchard Therapeutics 
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