A European Medicines Agency committee has issued a positive opinion on the gene-editing therapy Casgevy (exagamglogene autotemcel) in treating sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The Committee for Medicinal Products for Human Use (CHMP) recommendation will be considered by the European Commission in making a final…
New regulatory decisions in the U.S. and the U.K. have made Casgevy (exagamglogene autotemcel) the first gene-editing therapy to be approved for adults and adolescents with sickle cell disease (SCD) who experience painful vaso-occlusive crises (VOCs). With these recent approvals, the therapy from Vertex Pharmaceuticals and…
Most sickle cell disease (SCD) patients treated with the gene therapy Lyfgenia (lovotibeglogene autotemcel) in clinical trials were free from vaso-occlusive events (VOEs) after a median of three years of follow-up. That’s according to new data from Lyfgenia’s developer, Bluebird Bio, at the 65th American Society of Hematology…
Note: This story was updated Dec. 12, 2023, to clarify that Lyfgenia treats vaso-occlusive events, including painful vaso-occlusive crises (VOCs). The U.S. Food and Drug Administration (FDA) has approved the gene-editing therapy exagamglogene autotemcel (exa-cel) — now known as Casgevy — for treating sickle cell disease (SCD). The therapy…
Note: This story was updated Dec. 12, 2023, to clarify that Lyfgenia treats vaso-occlusive events (VOEs), including painful vaso-occlusive crises (VOCs). The U.S. Food and Drug Administration (FDA) has approved lovotibeglogene autotemcel, a gene therapy from Bluebird Bio known as lovo-cel, for treating sickle cell disease (SCD) patients ages…
A Phase 3 clinical trial evaluating whether adding red blood cell exchange to standard-of-care improves outcomes for adults with high-risk sickle cell disease (SCD) is still recruiting at sites across the U.S., as well as in France and Brazil. The study, called Sickle Cell Disease and CardiovAscular Risk Red…
Health regulators in the Kingdom of Bahrain have approved the gene-editing therapy Casgevy (exagamglogene autotemcel) to treat people with sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). This decision makes Bahrain, a small island nation in the Persian Gulf, the second country to approve Casgevy. The therapy was…
Skin grafts can ease pain associated with leg ulcers and improve wound healing in people with sickle cell disease (SCD), according to a study in France. The procedure consists of the surgical collection of a piece of healthy skin on the body that is then transplanted to the injured…
A clinical project designed to test whether masitinib, AB Science’s experimental oral therapy for sickle cell disease (SCD), can resolve complications associated with the inherited disorder has been awarded €9.2 million (about $10 million) in public funding in France. The funding comes as part of a larger investment of €160…
The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has conditionally approved the gene-editing therapy exa-cel (exagamglogene autotemcel), under the brand name Casgevy, for people with sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The authorization covers adults and children, ages 12 and older, with SCD and experiencing…
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