Gene therapy’s make or buy choice
How gene therapy companies are navigating manufacturing decisions
Whether or not to bring manufacturing in-house for gene therapy companies is becoming a critical choice, as contract manufacturers struggle to keep up with demand. The trade-off for the upfront investment risk is control over development timelines and the rapidly evolving science of process development.
A spate of gene therapies could hit the market in the next few years, following Spark Therapeutics Inc.’s Luxturna voretigene neparvovec, which last year became the first of the modality to gain FDA approval. At least one additional candidate is in registration -- Novartis AG’s Zolgensma onasemnogene abeparvovec -- and 14 others are in Phase III testing, according to BioCentury’s BCIQ database.
The surge is straining contract manufacturers, leading to long queues that threaten development timelines. Academic production centers are seeing similar backups (see “Academia’s Manufacturing Problem”).
At the same time, process development science for gene therapies is advancing swiftly, making it appealing for some drug developers to perform the work internally and secure manufacturing-related IP.
Because most gene therapy companies are targeting small patient populations, often on accelerated regulatory timelines, they need small quantities of product quickly, which the CMOs are not set up to accommodate.
While antibody or small molecule start-ups typically view the upfront costs of in-house production as prohibitive or too risky, for gene therapy developers