Casgevy (exagamglogene autotemcel) is now approved in the U.S. to treat children as young as 2 with sickle cell disease (SCD) and recurrent vaso-occlusive crises (VOCs). The U.S. Food and Drug Administration (FDA) initially approved the Vertex Pharmaceuticals’ gene-editing therapy in 2023 for SCD patients ages 12 and older who…
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A low dose of Vascarta‘s VAS-101, a curcumin-based gel being developed for sickle cell disease (SCD), was well tolerated and showed early signs of reducing red blood cell sickling, pain, and fatigue when administered under the tongue in a small Phase 1 study. The Phase 1 trial was…
Children with sickle cell anemia, the most common and severe form of sickle cell disease (SCD), who undergo stem cell transplant experience better long-term cognitive function after a decade than those given standard treatments like hydroxyurea or blood transfusions, according to a 10-year follow-up study. Transplant recipients also…
Brothers Cameron, Cayden, and Carmani Boozer have partnered with NMDP to encourage more young adults to join its blood stem cell donor registry and help people with sickle cell disease (SCD), blood cancers, and other serious blood disorders access potentially curative transplants. NMDP, formerly known as the National…
Agios Pharmaceuticals’ medication mitapivat rapidly increased levels of the oxygen-carrying protein hemoglobin in patients ages 16 and older with sickle cell disease (SCD), while also being linked to a lower blood transfusion burden over 52 weeks. These are new data from the Phase 3 portion of the global…
The U.S. Food and Drug Administration is reviewing an application from Vertex Pharmaceuticals seeking approval of Casgevy (exagamglogene autotemcel) for children with sickle cell disease (SCD) as young as 5 years old. The gene-editing therapy is currently approved in the U.S. and elsewhere for people ages 12 and older with…
A patient with severe sickle cell disease (SCD) remained free of two key SCD symptoms — including painful vaso-occlusive crises — for more than a year after receiving a single dose of CS-206, Correctsequence Therapeutics‘ investigational gene-editing therapy, new data show. The woman represents the first patient…
Stem cell transplant from a partially matched donor is more cost-effective than gene therapy to treat sickle cell disease (SCD), according to a study. In order for currently available gene therapies to deliver cost effectiveness on par with stem cell transplant, their cost would need to come down…
Fulcrum Therapeutics said it is discontinuing development of pociredir, its experimental oral treatment for sickle cell disease (SCD), after the U.S. Food and Drug Administration (FDA) raised concerns that the therapy may increase the risk of certain blood cancers. The concerns stem from the unexpectedly high rate of…
Higher blood levels of erythropoietin (EPO), a hormone that stimulates the production of red blood cells, increase the risk of blood clot-related, or thrombotic, events in people with sickle cell disease (SCD), according to a study involving more than 500 patients. Researchers also discovered a relationship between the production…
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