Despite high costs, gene therapy manufacturing has thus far been able to keep pace with demand, but that could change, as treatments for hemophilia and sickle cell disease (SCD) enter the later stages of development and evaluation.
Gene therapies, including viral vector and chimeric antigen receptor T-cell therapies, have gained regulatory approval in the U.S. and Europe to treat small patient populations. However, in the U.S., 20,000 people have hemophilia and 100,000 have SCD – increasing demand. A lack of manufacturing capacity could raise prices for and limit access to the treatments. One issue is related to the nature of gene therapy itself, which creates a unique treatment based on each patient’s individual DNA. “It’s not like making penicillin,” John Gray, PhD, senior vice president and chief scientific officer at Audentes Therapeutics, told STAT News. “We don’t even have the technology to take two batches side by side and see if they’re the same product.”
Providers also are concerned about moving gene therapy from clinical trials to real-world clinical practice. Treatment with gene therapy so far has relied on academic research centers or contractors to produce the treatments. Minor tweaks to the process often require U.S. Food and Drug Administration approval, necessitating additional trials and further delaying production.
At Bluebird Bio, which hopes to manufacture treatments for SCD and beta thalassemia, Chief Technology Manufacturing Officer Derek Adams, PhD, acknowledged the magnitude of the challenge. “We’re going to need to see some significant innovation in the manufacturing space for the entire industry to be able to start meeting peak demand – that is, a large number of diseases with patient populations the size of sickle cell and hemophilia,” Dr. Adams told STAT News.
Source: STAT News, November 5, 2018.