Orchard Therapeutics is established with £21m in funding to develop gene therapies for orphan diseases

Orchard’s development programmes are focused on ex-vivo autologous haematopoietic stem cell gene therapy

Orchard Therapeutics, a clinical-stage biotechnology company with operations in London, UK and Los Angeles, US, has officially launched with funding of £21m led by venture capital fund F-Prime Capital.

As part of its launch, the firm has agreed partnerships with University College London (UCL), Great Ormond Street Hospital for Children NHS Foundation Trust (GOSH), the University of Manchester, the University of California Los Angeles (UCLA) and Boston Children’s Hospital with the aim of developing gene therapies for serious and life-threatening orphan diseases.

Orchard’s management team includes founders Andrea Spezzi, Chief Medical Officer, and Nicolas Koebel, SVP Business Operations, formerly with GSK. Chief Manufacturing Officer Stewart Craig previously held executive management positions in cell and gene therapy companies for more than 20 years, and most recently was head of technical operations at Sangamo BioSciences.

'Orchard’s mission is to be nimble and focused so that we can translate results from pre-clinical and early clinical research into commercially approved medicines,' the team said.

Orchard’s development programmes are focused on ex-vivo autologous haematopoietic stem cell gene therapy

Orchard’s development programmes are focused on ex-vivo autologous haematopoietic stem cell gene therapy to restore normal gene function in primary immune deficiencies, metabolic diseases and haematological disorders.

This 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 cells (autologous) removes the need to search for a matching stem cell donor, which can take a long time.

Bobby Gaspar, Orchard’s Chief Scientific Officer and Professor of Paediatrics and Immunology at the UCL Institute of Child Health and GOSH, 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 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 lead candidate is ex-vivo autologous lentiviral stem cell gene therapy for severe combined immunodeficiency caused by adenosine deaminase deficiency (ADA-SCID).

The firm is also exploring the effects of ex-vivo autologous lentiviral stem cell gene therapy in patients with mucopolysaccharidosis type IIIA (MPS IIIA or Sanfilippo disease type A).

F-Prime Capital has extensive experience in rare diseases and gene and cell therapies, with support from UCL Business and additional participation from the UCL Technology Fund.

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