How a Viral Vector Got the Boost It Needed to Start Defeating an Incurable Disease


Duchenne’s Muscular Dystrophy is an incurable disease that mostly affects boys. It results from a gene defect that prevents those affected from developing normal muscle structure and function. Children typically lose the ability to walk by early adolescence and may only survive into their 30s or 40s. A client was developing a new viral vector gene therapy, but they could only make one batch at a time, and this could only treat one patient. Nicole and her team worked on two paths. First, they re-engineered the growing process so it could now treat 5-10 patients per batch. Next, they gutted a site and rebuilt it with multiple bioreactor suites. All of this on a tight clinical trial deadline. Now, the client will be able to make enough material to treat hundreds of patients per year and the results are astounding. Promising clinical trials are underway and participants are developing stronger muscle tissue. Although it’s still early, the results for these Muscular Dystrophy patients are promising.