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Skip to contentSkip to site index Search & Section Navigation Section Navigation SEARCH Health SUBSCRIBE FOR $1/WEEKLog in Friday, December 8, 2023 Today’s Paper SUBSCRIBE FOR $1/WEEK SICKLE CELL DISEASE * F.D.A. Approves 2 Therapies * Understand CRISPR * Anxiety and Hope for a Cure * Failures of Care Advertisement SKIP ADVERTISEMENT Supported by SKIP ADVERTISEMENT F.D.A. APPROVES SICKLE CELL TREATMENTS, INCLUDING ONE THAT USES CRISPR People with the genetic disease have new opportunities to eliminate their symptoms, but the treatments come with obstacles that limit their reach. * Share full article * * * 44 * Read in app A vial of Vertex Pharmaceuticals’s CRISPR Cas-9 gene therapy for sickle cell disease.Credit...Vertex Pharmaceuticals By Gina Kolata Gina Kolata has reported on gene therapy for nearly 30 years and on sickle cell disease since 2018. Dec. 8, 2023Updated 1:57 p.m. ET On Friday, the Food and Drug Administration approved the first gene editing therapy ever to be used in humans, for sickle cell disease, a debilitating blood disorder caused by a single mutated gene. The agency also approved a second treatment using conventional gene therapy for sickle cell that does not use gene editing. For the 100,000 Americans with the disease, most of them Black, the approvals offer hope for finally living without an affliction that causes excruciating pain, organ damage and strokes. While patients, their families and their doctors welcome the F.D.A.’s approvals, getting either therapy will be difficult, and expensive. Advertisement SKIP ADVERTISEMENT “It is practically a miracle that this is even possible,” said Dr. Stephan Grupp, chief of the cellular therapy and transplant section at Children’s Hospital of Philadelphia. Dr. Grupp, who consults for Vertex, said his medical center was hoping to begin treating sickle cell patients next year. But, he added, “I am very realistic about how hard this is.” The obstacles to treatment are myriad: an extremely limited number of medical centers authorized to provide it; the requirement that each patient’s cells be edited or have a gene added individually; procedures that are so onerous that not everyone can tolerate them; and a multimillion-dollar price tag and potential insurance obstacles. Subscribe to The Times to read as many articles as you like. Gina Kolata reports on diseases and treatments, how treatments are discovered and tested, and how they affect people. More about Gina Kolata Read 44 Comments * Share full article * * * 44 * Read in app Advertisement SKIP ADVERTISEMENT COMMENTS 44 F.D.A. Approves Sickle Cell Treatments, Including One That Uses CRISPRSkip to Comments Share your thoughts. The Times needs your voice. We welcome your on-topic commentary, criticism and expertise. Comments are moderated for civility. SITE INDEX SITE INFORMATION NAVIGATION * © 2023 The New York Times Company * NYTCo * Contact Us * Accessibility * Work with us * Advertise * T Brand Studio * Your Ad Choices * Privacy Policy * Terms of Service * Terms of Sale * Site Map * Canada * International * Help * Subscriptions Enjoy unlimited access to all of The Times. See subscription options