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¹û¶³Ó°Ôº and F-Prime launch Orchard Therapeutics to treat rare childhood diseases

3 May 2016

¹û¶³Ó°Ôº Business, the technology transfer company of ¹û¶³Ó°Ôº, and venture capital firm F-Prime Capital Partners have launched Orchard Therapeutics, a biotechnology company specialising in gene therapies for life threatening rare childhood diseases.

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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 ¹û¶³Ó°Ôº, Great Ormond Street Hospital for Children NHS Foundation Trust (GOSH), University of California Los Angeles (¹û¶³Ó°ÔºA), 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 ¹û¶³Ó°Ôº/GOSH and ¹û¶³Ó°ÔºA 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 ¹û¶³Ó°ÔºB and additional participation from the ¹û¶³Ó°Ôº Technology Fund.

Cengiz Tarhan, Managing Director of ¹û¶³Ó°ÔºB, said:

"We are very excited to see ¹û¶³Ó°Ôº'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 ¹û¶³Ó°Ôº and its partners and we are delighted to be able to work with F-Prime Capital to launch Orchard."

The work at the ¹û¶³Ó°Ôº Institute of Child Health has been developed through the laboratories of Professors Bobby Gaspar, Adrian Thrasher and Waseem Qasim in collaboration with GOSH, ¹û¶³Ó°ÔºA 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 ¹û¶³Ó°Ôº, the Great Ormond Street Hospital Children's Charity, the Medical Research Council, the Wellcome Trust and the Histiocytosis Research Trust.Ìý

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