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In December, the FDA approved the first two cell-based gene therapies for treating sickle cell disease: Casgevy and Lyfgenia. And, like any emerging medical technology, the initial use of the ...
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 ...
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 ...
The FDA on Friday also approved a second treatment for sickle cell disease, called Lyfgenia, a gene therapy from drugmaker Bluebird Bio. ... How Casgevy works. In patients with sickle cell disease ...
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 ]
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 .
The US Food and Drug Administration on Friday approved two gene-based treatments for sickle cell disease, including the first therapy that uses the gene-editing technique CRISPR, opening a new era ...
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 ...
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