The future is now: Orchard’s potentially curative gene therapies

Orchard Therapeutics, a biotech with roots in the heart of London, is among leaders of a genetic revolution.

“When the human genome was mapped nearly 20 years ago, the notion that this genetic blueprint could advance therapies capable of finding and possibly fixing the genes responsible for some of the world’s most devastating diseases was an idea of the future,” says Bobby Gaspar, professor of paediatrics and immunology at the UCL Great Ormond Street Institute of Child Health.

Giovanni and Cecilia Price hold a photo of their sister, Liviana, who died at an early age from MLD, a diagnosis they share

Professor Gaspar, who has led multiple successful gene therapy clinical trials in immune deficiencies and other rare genetic diseases, is chief scientific officer at Orchard. A global entity with offices in London, Boston and the San Francisco Bay area, Orchard has the European-approved, commercial gene therapy product, Strimvelis®, together with six clinical-stage gene therapy products in development and a robust pre-clinical pipeline, all aimed at potentially curing a range of rare diseases with a single administration of gene therapy.

Orchard’s gene therapy approach is highly individualised and utilises gene-modified blood stem cells to target the underlying cause of disease. Blood stem cells are taken from the patient, genetically modified outside of the body by inserting a functional copy of a missing or faulty gene, and then transplanted back in the patient to potentially cure the disease through a single administration.

Mark Rothera, a seasoned biotech executive who has spent most of his career bringing treatments to patients with rare diseases, took the helm at Orchard in September 2017 after spending nearly 30 years in the industry and has grown the company from 35 employees to more than 200. Mr Rothera and his team took the company public last October and continue to add new programmes to the company’s growing portfolio, each of which leverages Orchard’s gene therapy approach.

“Everything I’ve launched previously has focused on slowing disease progression and trying to improve quality of life, but at Orchard we have the potential opportunity to take a child on a path to dying at five years of age, and with a single administration, provide them with a more normal life,” he says.

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