Google
Trusted Resources: News & Events
Latest announcements and gatherings
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?
+myBinderRelated Content
-
Hatoon Ezzat, MBBS, FRCPC, ABIMDr. Hatoon Ezzat received her medical de...
-
Acute Vaso-Occlusive pain associated with the onset of menstruation is distinct from dysmenorrhea in sickle cell dis...Acute vaso-occlusive pain episodes are t...
-
Coronavirus Preparedness for People With Chronic Diseaseshttps://www.youtube.com/watch?v=G_JC3vUT...
-
Sickle cell disease is associated with higher mortality among patients hospitalized with ischemic bowel diseaseBackground: Sickle cell disease (SCD) i...
-
Opioid Abuse Backlash Could Hurt Sickle Cell Patients’ Medication Needs, Some FearWith drug overdoses now the leading caus...
-
Shengdar Q. Tsai, PhDDr. Shengdar Q. Tsai is an assistant mem...
-
2019 Cure Sickle Cell Initiative Annual Forum: Accelerating Scientific PrioritiesTuesday, November 19 - Thursday, Novembe...
To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More Information
The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.
Powered by
SomeBodyToTalkTo connects people affected by rare diseases with trusted resources, supportive communities, and opportunities to share their experiences.