October 6, 2016
By Alex Keown, BioSpace.com Breaking News Staff
LONDON – Startup biotech company Orchard Therapeutics has opened up shop in the crowded Bay Area, setting up its North American subsidiary in Foster City—a quick expansion for a company that has only been in existence a few short months.
The U.K.-based company, which is developing a platform of ex-vivo autologous gene therapies in serious and life-threatening orphan diseases, has already struck partnerships with the University of California at Los Angeles and Boston Children’s Hospital. The company, which launched in May with about $33 million in Series A funding, said it also has collaborations with “leading manufacturing partners specialized in gene and cell therapy.” In addition to the partnerships with the U.S. institutions, Orchard also has collaborative partnerships with University College London, Great Ormond Street Hospital for Children NHS Foundation Trust (GOSH) and the University of Manchester.
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 percent survival in 32 patients treated at GOSH and UCLA as of March 2016. As of the end of September, Orchard said the number of patients treated increased to 39 patients.
Orchard’s drug candidate is expected to challenge Strimvelis, GlaxoSmithKline ’s gene therapy drug recently approved in Europe to treat ADA-SCID. Children born with ADA-SCID do not develop a healthy immune system so they cannot fight off everyday infections, which results in severe and life-threatening illness. Without prompt treatment, the disorder often proves fatal within the child’s first year of life. ADA-SCID is estimated to occur in approximately 15 patients per year in Europe.
Foster City, which is home to Gilead Sciences , will serve as the headquarters for Orchard’s North American subsidiary, Orchard Therapeutics North America. The U.S. site will provide the central technical base for laboratory operations associated with the development and validation of manufacturing processes and analytical methods for the company’s products, the company said.
“We are building a world-class team of experts in the development, manufacture, testing and delivery of cell-based gene therapy products. This facility represents a next key step in establishing a central technical capability to support the global clinical development and commercialization of Orchard’s products,” Stewart Craig, Orchard’s chief manufacturing officer said in a statement.
Alexander Pasteur, the interim chief executive officer of Orchard, said the company’s early goal is to become a category leader in ex-vivo autologous gene therapy for rare conditions. In a statement released Wednesday, Pasteur said the opening of a facility in the United States is a key step in building the platform that “can deliver Orchard’s transformative therapies to patients wherever they are.”
