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...

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 uses your own blood stem cells to help your body make working red …

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-thalassemia) who need regular blood transfusions

CASGEVY indicated for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso occlusive crises (VOCs).

CASGEVY is indicated for the treatment of sickle cell disease (SCD) in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). For autologous use only. For one-time,...

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, thereby significantly enhancing the quality of life of individuals with SCD.

Apr 24, 2024 · Clinical trials at CHOP and other sites have shown that successful gene editing can prevent cells from developing the distinctive crescent shape apparent in sickle cell disease and have eliminated pain episodes in almost all patients. CASGEVY was the first FDA-approved therapy developed with CRISPR technology.

Dec 8, 2023 · 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 from Vertex Pharmaceuticals and CRISPR Therapeutics is indicated for patients ages 12 and older who have recurrent vaso-occlusive crises (VOCs).

CASGEVY™ (exagamglogene autotemcel) is a one-time gene therapy treatment for people age 12 years or older with sickle cell disease or beta thalassemia. It uses a gene editing technology called CRISPR/Cas9 to modify patients’ own blood-forming stem cells.

Dec 19, 2023 · With Casgevy, an edit (or “cut”) is made in a particular gene to reactivate the production of fetal hemoglobin, which dilutes the faulty red blood cells caused by sickle cell disease (more on that below).