Can drug makers drop manufacturing costs enough to make gene therapies more feasible for curing patients with devastating diseases? [Sponsored by Pfizer]
For the 320 million people worldwide who suffer from genetic diseases like cystic fibrosis and hemophilia, effective therapies have been hard to come by. The medicines that are available may treat symptoms and alleviate pain, rather than tackling the root cause of disease.
The U.S. Food & Drug Administration has already approved five gene therapies that use a virus as a delivery vehicle. As of February 2022, 25 of these viral-vector-based therapies have entered late-stage efficacy testing and another 120 have begun Phase 2 trials to gather safety data, so the number of approved therapies is expected to grow.As manufacturers since Henry Ford have discovered, things are more economical to make when they’re standardized and when production is streamlined.
For more than two decades, they have done so at lab-scale volumes—often less than a liter—by carefully adjusting the buffer compounds in the cell culture broth, as well as its temperature, atmosphere, pH and other additives. But the same growing conditions have failed almost universally when producing hundreds or even thousands of liters in large bioreactors for clinical trials, and often the supply of clinical-grade rAAV has often failed to meet the demand, Mallela says.
The company has invested $800 million since 2017 in a manufacturing hub near North Carolina’s Research Triangle Park. The hub includes an R&D facility for preclinical testing of new medicines, a nearby pilot plant that produces medicine for late-stage clinical trials, and, just down the road in Sanford, North Carolina, the first manufacturing plant in the world for the commercial production of gene therapies.
The challenges of commercialization Eventually, Hjelmquist and his team created a robust, consistent process. But scaling to commercial production meant even bigger batches and more challenges, and required more innovation, says Ann Czar, who heads facility operations and compliance at the Sanford facility.
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