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Exagamglogene autotemcel is the first cell-based gene therapy treatment utilizing CRISPR/Cas9 gene editing technology to be approved by the US Food and Drug Administration (FDA). [13] The most common side effects include low levels of platelets and white blood cells, mouth sores, nausea, musculoskeletal pain, abdominal pain, vomiting, febrile ...
The FDA on Friday also approved a second treatment for sickle cell disease, called Lyfgenia, a gene therapy from drugmaker Bluebird Bio. Both treatments work by genetically modifying a patient’s ...
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 ...
Exa-cel is a drug therapy for the treatment of the rare blood disorders beta thalassemia and sickle cell disease developed jointly with Vertex Pharmaceuticals, [7] that received FDA approval for clinical use in December 2023. [5]
(Reuters) -The U.S. Food and Drug Administration (FDA) on Friday approved two gene therapies for sickle cell disease, making one of them the first treatment in the United States based on the Nobel ...
The FDA is one step closer to approving a cure for sickle cell disease that uses CRISPR gene editing. It would be the first approve drug to use gene editing. FDA moves closer to sickle cell cure ...
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 ...
A breakthrough treatment for sickle cell patients could soon become the first gene-editing treatment to be approved by the Food and Drug Administration (FDA). Sickle cell disease (SCD) is an ...
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