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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 ...
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 ...
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 ...
“Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need,” the FDA’s Dr. Nicole Verdun […] The post U.S. approves two sickle cell gene ...
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 ...
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]
Regulators on Friday approved two gene therapies for sickle cell disease that doctors hope can cure the painful, inherited blood disorder that afflicts mostly Black people in the U.S. The Food and ...
Victoria Gray was the first patient ever to be treated with the gene-editing tool CRISPR for sickle-cell disease. [1]This marked the initial indication that a cure is attainable for individuals born with sickle-cell disease and another severe blood disorder, beta-thalassemia.
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