FDA Approves First Gene Therapies to Treat Patients with Sickle Cell ...
Casgevy, a cell-based gene therapy, is approved for the treatment of sickle cell disease in patients 12 years of age and older with recurrent vaso-occlusive crises. Casgevy is the first...
NHS England to offer Casgevy for sickle cell disease patients
21 hours ago · The process targets the BBCL11A gene to increase foetal haemoglobin production, helping red blood cells function more effectively and reducing complications …
Patient Information | CASGEVY® (exagamglogene autotemcel)
CASGEVY is a one-time* gene therapy that may help people 12 years and older with sickle cell disease (SCD) and frequent vaso-occlusive crises (VOCs) live severe VOC-free. CASGEVY …
CASGEVY® (exagamglogene autotemcel) | Patient Website
CASGEVY is a one-time therapy used to treat people aged 12 years and older with: sickle cell disease (SCD) who have frequent vaso-occlusive crises or VOCs. beta thalassemia (β …
CASGEVY | FDA - U.S. Food and Drug Administration
CASGEVY indicated for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso occlusive crises (VOCs).
Revolutionary breakthrough: FDA approves CASGEVY, the first …
Casgevy, the first FDA-approved CRISPR/Cas9 gene therapy for sickle cell disease (SCD), offers a curative approach and eliminates the need for recurrent transfusions and transplants, …
World-first gene-editing treatment for blood disorders including sickle …
23 hours ago · Casgevy is designed to work by precisely editing the genes in a patient’s bone marrow stem cells to enable the production of a functioning haemoglobin. ... 28 out of 29 sickle …
Researchers Publish Final Results of Key Clinical Trial for Gene ...
Apr 24, 2024 · In a landmark study, an international consortium led by researchers at CHOP published the final results of a key clinical trial of the gene therapy CASGEVY for the treatment …
FDA approves cure for sickle cell disease, the first treatment to …
Both treatments work by genetically modifying a patient’s own stem cells. Until now, the only known cure for sickle cell disease was a bone marrow transplant from a donor, which carries …
Casgevy: UK approves gene-editing drug for sickle cell
Nov 16, 2023 · The treatment for sickle cell disease and beta thalassemia is the first to be licensed using the gene-editing tool known as Crispr, for which its discoverers were awarded …