Intricate Supply Chain Complicates Gene Therapy Manufacturing

By Cathy Yarbrough, Contributing Editor
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After three decades of disappointments and clinical setbacks, gene therapy is finally delivering on its promise to alleviate – and possibly even cure – a long list of acquired and hereditary diseases. In 2017, the FDA approved three therapies that use vectors derived from harmless viruses to deliver functional copies of genes to patients’ cells. FDA Commissioner Scott Gottlieb, M.D., hailed gene therapy as “a whole new scientific paradigm for the treatment of serious diseases.”

The newly-approved gene therapies include two CAR T cell treatments. The third gene therapy to gain the FDA’s approval last year treats previously irreversible blindness in children and adults with inherited retinal disease (IRD). (Editor’s note: for more information about the three groundbreaking therapies, please see sidebar “FDA-Approved Gene Therapies.”)