Researchers from five research institutes have banded together to move an innovative gene therapy out of academia and into the commercial arena. Orchard Therapeutics was launched in May by a group of collaborators that reads like a who's who in gene therapy—among them Adrian Thrasher of Great Ormond, London, David Williams of Boston Children's Hospital, Don Kohn of University of California in Los Angeles and University College London's Bobby Gaspar, who will serve as the company's CSO. The company raised $33 million series A financing from F-Prime Capital of Cambridge, Massachusetts,with support from University College London Business and Technology Fund.

Orchard's focus is on developing ex vivo autologous lentiviral stem cell gene therapies, an area in which the founding scientists and Nicolas Koebel, the company's senior vice president for business operations, are well-versed. Koebel was part of the group at GlaxoSmithKline (GSK; Brentford, UK) that forged a collaboration with San Raffaele Telethon Institute for Gene Therapy (TIGET) in Milan to develop a gene therapy for the rare severe immune deficiency ADA-SCID, characterized by a deficiency in adenosine deaminase. This therapy was the first ex vivo package presented to the European Medicines Agency, and in April, GSK and collaborators learned that it had received the go-ahead from the Committee for Medicinal Products for Human Use of the European Medicines Agency (see feature page 600).

Orchard's founding scientists have been working together since at least 2005, when they formed the Transatlantic Gene Therapy Consortium to tackle the safety problems encountered with the first generation of retroviral gene therapy vectors. This grassroots effort, which combined the expertise from a number of centers in treating rare diseases with different gene therapy platforms, was highly successful, according to Williams. In starting Orchard, the idea was to bring a biotech structure to that expertise, says Williams. “We felt the focus was correct, the timing was correct,” he says. The company's role now is to get approval, negotiate reimbursement and to commercialize the therapy to make it available to patients, says Koebel. Getting reimbursement will be key for the small biotech, but Koebel is confident that when the time comes they will be able to demonstrate that the technology delivers transformative clinical benefit to patients. “Generally our stand and probably for the industry [should be] to move the discussion from a price discussion to a discussion of the value,” he says. The company has an ADA-SCID program in clinical trials, with 32 patients treated so far, and has a preclinical program in mucopolysaccharidosis type III, which will be ready for the clinic later this year.