Matching articles for "betibeglogene autotemcel"
In Brief: Casgevy for Beta Thalassemia
The Medical Letter on Drugs and Therapeutics • May 13, 2024; (Issue 1702)
Exagamglogene autotemcel (Casgevy – Vertex), a cell-based
gene therapy recently approved for treatment
of sickle cell disease1, has now been approved by
the FDA for treatment of patients ≥12 years old...
Exagamglogene autotemcel (Casgevy – Vertex), a cell-based
gene therapy recently approved for treatment
of sickle cell disease1, has now been approved by
the FDA for treatment of patients ≥12 years old with
transfusion-dependent beta thalassemia. Casgevy is
the first gene therapy that uses CRISPR/Cas9 gene-editing
technology to be approved in the US for any
disorder. Betibeglogene autotemcel (Zynteglo), an
autologous lentiviral vector cell-based gene therapy,
was approved in the US in 2022 for treatment of
transfusion-dependent beta thalassemia.