Matching articles for "Casgevy"
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.
Casgevy and Lyfgenia: Two Gene Therapies for Sickle Cell Disease
The Medical Letter on Drugs and Therapeutics • January 22, 2024; (Issue 1694)
Two cell-based gene therapies – exagamglogene
autotemcel (Casgevy – Vertex) and lovotibeglogene
autotemcel (Lyfgenia – Bluebird Bio) – have been
approved by the FDA for treatment of sickle...
Two cell-based gene therapies – exagamglogene
autotemcel (Casgevy – Vertex) and lovotibeglogene
autotemcel (Lyfgenia – Bluebird Bio) – have been
approved by the FDA for treatment of sickle cell
disease in patients ≥12 years old with recurrent vaso-occlusive
crises. They are the first gene therapies to
be approved in the US for use in sickle cell disease;
Casgevy is the first treatment to be approved in the
US that uses CRISPR/Cas9 gene-editing technology.