Translating both a cure and a commercialization pathway: insights into a breakthrough gene therapy company’s journey

This article examines the commercialization pathway of Orchard Therapeutics (Orchard), tracing its evolution from an academic innovation hub to a biotech enterprise with an EMA- and US FDA-approved medicine. We analyze critical inflection points, from the 2018 acquisition of GSK’s rare disease gene therapy portfolio to Orchard’s acquisition by Kyowa Kirin and the FDA approval of Lenmeldy in 2024. Drawing on extensive interviews with key decision makers, we show how Orchard addressed regulatory, manufacturing, and reimbursement hurdles by forging external production partnerships, crafting innovative pricing models, and shaping newborn screening policy. The case offers transferable lessons for scaling operations, aligning incentives, and accelerating market access.