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Lovotibeglogene autotemcel, sold under the brand name Lyfgenia, is a lentiviral gene therapy used for the treatment of sickle cell disease. [1] [3] [4] [5]The most common side effects include stomatitis (mouth sores of the lips, mouth, and throat), low levels of platelets, white blood cells, and red blood cells, and febrile neutropenia (fever and low white blood cell count), consistent with ...
Exagamglogene autotemcel, sold under the brand name Casgevy, is a gene therapy used for the treatment of sickle cell disease [3] [5] and transfusion-dependent beta thalassemia. [3] It was developed by Vertex Pharmaceuticals and CRISPR Therapeutics .
What we do know is that the manufacturers will sell Casgevy and Lyfgenia to wholesalers in the US — which sell products to pharmacies and medical facilities at a markup — for $2.2 million and ...
In 2023, both exagamglogene autotemcel (Casgevy) and lovotibeglogene autotemcel (Lyfgenia) were approved for the treatment of sickle cell disease. [ 12 ] [ 130 ] [ 14 ] Kendric Cromer in October 2024 became the first commercial case in the USA to receive gene therapy and was discharged from Children's National Hospital . [ 131 ]
Casgevy, developed by partners Vertex Pharmaceuticals and CRISPR Therapeutics, and bluebird bio's Lyfgenia were approved for people aged 12 years and older. Sickle cell disease (SCD) is a painful ...
Casgevy works by editing the DNA in a patient’s stem cells — which are responsible for making the body’s blood cells — so that they no longer produce sickle-shaped cells.
Exagamglogene autotemcel (Casgevy): treatment for sickle cell disease. [11] Gendicine: treatment for head and neck squamous cell carcinoma; Idecabtagene vicleucel (Abecma): treatment for multiple myeloma [12] Lovotibeglogene autotemcel (Lyfgenia): treatment for sickle cell disease. [11] Nadofaragene firadenovec (Adstiladrin): treatment for ...
[173] [174] In December 2023, the US Food and Drug Administration (FDA) approved the first cell-based gene therapies for treating sickle cell disease, Casgevy and Lyfgenia. Casgevy is the first FDA approved gene therapy to use the CRISPR-Cas9 technology and works by modifying a patient's hematopoietic stem cells. [175]