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Orchard Therapeutics, a 香港六合彩中特网 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 香港六合彩中特网 in the early 1990s.听

Desperate for better treatments to help children at Great Ormond Street with rare genetic disorders, 香港六合彩中特网鈥檚 Professors Bobby Gaspar and Adrian Thrasher began working on pioneering gene therapies.听

Professor Gaspar, from 香港六合彩中特网鈥檚 GOSH Institute of Child Health, explains: 鈥淲e 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.鈥澨�

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

After successful clinical trials, the 香港六合彩中特网 team developed new treatments for two different forms of SCID and began getting referrals from patients around the world.听听

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 香港六合彩中特网 Business (香港六合彩中特网B), the commercialisation company of 香港六合彩中特网 and part of 香港六合彩中特网 Innovation & Enterprise.听听

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

David Phillips, Senior Business Manager for 香港六合彩中特网B, said: 鈥淭he team behind Orchard are doing really ground-breaking work, with the potential for very significant patient impact. So it鈥檚 very exciting to be part of bringing some of the first gene therapies to market.鈥澨�

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Links听

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Great Ormand Street Institute of Child Health听
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REF case study: Orchard Therapeutics听
Create a spinout business from your research听听

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