UCL and F-Prime launch Orchard Therapeutics to treat rare childhood diseases

Orchard Therapeutics has secured £21 million investment led by F-Prime to support the development of gene therapy treatments for a range of diseases.

As part of its launch, Orchard announced formal partnerships with UCL, Great Ormond Street Hospital for Children NHS Foundation Trust (GOSH), University of California Los Angeles (UCLA), The University of Manchester, Boston Children's Hospital for the development of these transformative gene therapies.

Orchard's lead programme is for the treatment of the rare and fatal disorder called severe combined immunodeficiency caused by adenosine deaminase deficiency (ADA-SCID), sometimes referred to as "bubble baby" syndrome in the press. Children with the disorder must be protected from any infections due to their highly fragile immune system.

In a first-in-man clinical study, global leaders in the field at UCL/GOSH and UCLA have shown significant immune reconstitution and 100% survival in 32 patients treated, as of March 2016.

Orchard's financing has been led by F-Prime Capital, with support from UCLB and additional participation from the UCL Technology Fund.

Cengiz Tarhan, Managing Director of UCLB, said:

"We are very excited to see UCL's breakthrough treatments move forward in a commercial environment in a way that will benefit patients globally. The formation of Orchard represents the culmination of decades of research at UCL and its partners and we are delighted to be able to work with F-Prime Capital to launch Orchard."

The work at the UCL Institute of Child Health has been developed through the laboratories of Professors Bobby Gaspar, Adrian Thrasher and Waseem Qasim in collaboration with GOSH, UCLA and King's College London, with funding and support from the National Institute for Health Research Biomedical Research Centre at Great Ormond Street Hospital for Children NHS Trust and University College London, the Great Ormond Street Hospital Children's Charity, the Medical Research Council, the Wellcome Trust and the Histiocytosis Research Trust. 

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