Orchard Therapeutics' 2019: Pipeline progress, breaking ground on its $90M manufacturing site

Orchard Therapeutics started 2018 with two clinical-stage assets and a preclinical pipeline that was, in CEO Mark Rothera’s words, “not that large.” Now, after picking up GlaxoSmithKline’s rare disease gene therapy unit and becoming a commercial-stage company, Orchard is looking to bring its treatment for “bubble boy syndrome” to the U.S. and ramp up its clinical work. Orchard acquired the GSK assets in exchange for a 19.9% stake in Orchard and undisclosed milestones and royalties. These included the EMA-approved Strimvelis, which treats ADA-SCID, a rare genetic disorder that causes immunodeficiency, as well as late-stage candidates for metachromatic leukodystrophy (MLD) and Wiskott-Aldrich syndrome (WAS). The company, based in Boston and London, now has five clinical-stage gene therapies and 10 preclinical programs.