FDA approval of gene therapies for sickle cell disease: Q&A with NHLBI Director Dr. Gary Gibbons and NHLBI’s Division of Blood Diseases and Resources Director Dr. Julie Panepinto

The U.S. Food and Drug Administration approved two new gene therapies to treat sickle cell disease — one a gene-editing and the other a gene-addition approach. Nearly 100,000 people in the United States — and millions worldwide — have been diagnosed with this painful, life-threatening genetic blood disease. To help explain the historic importance of this medical milestone, we sat down with Gary Gibbons, M.D., director of the National Heart, Lung, and Blood Institute (NHLBI), and Julie Panepinto, M.D., director of the NHLBI’s Division of Blood Diseases and Resources (DBDR).

Genetic therapies aim to treat or cure conditions by adding new DNA or changing existing DNA. Decades of basic research on sickle cell disease by scientists—including NIH-supported researchers— and selfless efforts by clinical trial participants have helped lay the groundwork for these novel genetic approaches. Researchers, patients, clinicians, and advocacy groups expect these new FDA-approved approaches to help people with sickle cell disease live longer, less painful, and more productive lives. “We have made some exciting research advances over the years and are ready to collect on our scientific investments in sickle cell research,” Gibbons said. “However, we must remember that these advances need to go hand-in-hand with scalable innovations that will ensure equitable access to life-altering care and that we must continue to engage in additional research endeavors that will minimize or eliminate potential risks that might be associated with these therapies.”

Q: Two companies, Bluebird Bio and Vertex Pharmaceuticals, just received FDA approval of their gene therapy protocols. How will FDA approval of these approaches impact the field? How will it impact patients?

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