As part of its launch, Orchard Therapeutics' has 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 and Boston Children’s Hospital for the development of these transformative gene therapies.
Orchard Therapeutics' development programmes focus on restoring normal gene function in primary immune deficiencies, metabolic diseases and haematological disorders. This pioneering technology uses a sample of the patient’s own stem cells, which are modified with a functioning copy of the missing or faulty gene before being transplanted back into the patient’s body. The use of the patient’s own (autologous) cells removes the need to search for a matching stem cell donor, which can take months or even years.
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.
Bobby Gaspar, Professor of Paediatrics and Immunology at UCL’s Institute of Child Health/GOSH and Orchard’s Chief Scientific Officer said, “Orchard’s founding scientists, also including Professors Adrian Thrasher and Waseem Qasim from UCL and GOSH, have been pioneering ex-vivo autologous haematopoietic stem cell gene therapy for the last 20 years. We have seen very promising effects in several different diseases and are hopeful that this technology will change the lives of many children with life-threatening conditions in the future.”
Orchard’s Series A financing has been led by F-Prime Capital, a leading venture capital investment fund with extensive experience in rare diseases and gene and cell therapies, with support from UCLB and additional participation from the UCL Technology Fund.
Alex Pasteur and Ben Auspitz, partners at F-Prime Capital, commented, “We are delighted to have played a central role in the formation of Orchard. We view ex-vivo gene therapy as an exciting field for investment in the creation of transformative medicines”.
Cengiz Tarhan, Managing Director of UCLB, added, “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 on this important venture.”
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 (“NIHR”) Biomedical Research Centre (“BRC”) 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.