Orchard Therapeutics, a clinical-stage biotechnology company with operations in London and the U.S., has officially launched with a £21-million Series A financing led by F-Prime Capital. As part of its launch, the company has announced formal 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 for the development of transformative gene therapies for serious and life-threatening orphan diseases.
Orchard’s development programs exploit the potential of ex-vivo autologous hematopoietic stem cell gene therapy to restore normal gene function in primary immune deficiencies, metabolic diseases and hematological 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 cells (autologous) removes the need to search for a matching stem cell donor, which can take months or even years.
Bobby Gaspar, Orchard’s chief scientific officer and professor of Pediatrics 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 hematopoietic 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). Interim clinical data from this program show significant immune reconstitution and 100% survival in 32 patients treated at GOSH and UCLA, as of March 2016.
Orchard 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).
Orchard employs a collaborative development model for its research programs, working closely with clinicians and researchers at leading academic centers. Professor David Williams, chief of Hematology/Oncology at Boston Children’s Hospital and president of Dana-Farber/Boston Children’s Cancer and Blood Disorders Center, said, “Orchard builds on highly successful academic collaborations that have been in place for more than a decade. Each institution brings specific disease expertise and a significant experience in developing and carrying out gene therapy trials using autologous hematopoietic stem cells.”
Orchard’s management team includes senior pharmaceutical leaders with extensive experience in gene and cell therapy, including founders Andrea Spezzi, chief medical officer, and Nicolas Koebel, SVP Business Operations, who prior to joining Orchard were involved in the clinical development and market access planning at 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 Scientific Advisory Board is comprised of world-leading researchers in gene therapy and rare diseases, including Professor Alessandra Biffi (Dana Faber/Boston Children’s Hospital), Dr. Brian Bigger (The University of Manchester), Professor Bobby Gaspar (UCL/GOSH), Dr. Simon Jones (Central Manchester University Hospitals NHS Foundation Trust), Professor Donald Kohn (UCLA), Professor Fulvio Mavilio (Genethon), Professor Waseem Qasim and Professor Adrian Thrasher (UCL/GOSH), Professor David Williams (Boston Children’s Hospital) and Professor Robert Wynn (Central Manchester University Hospitals NHS Foundation Trust).