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From Flask to Vat: The Challenges of Scaling Up Gene Therapy


Over the past two decades, gene therapy has been incubating at a very small scale in academic research labs. Like a chef fine-tuning a new recipe, scientists have been working in single batches that would only be sufficient to treat one or two patients at a time. 

But now, as these treatments are beginning to show great promise for rare diseases such as Duchenne muscular dystrophy (DMD) and hemophilia, researchers are facing the next challenge: scaling up to produce gene therapy for clinical trial patients and then eventually at the commercial level, to reach thousands of real-world patients.  

From Flask To Vat: The Challenges Of Scaling Up Gene Therapy