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On 16 December 2021, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Oxbryta, intended for the treatment of hemolytic anemia due to sickle cell disease.
Federal officials Friday approved two gene therapy treatments for sickle cell disease, welcome news for thousands with the inherited blood disorder that causes terrible pain crises and early death
Treatment is the same as for patients with sickle cell disease. Patients may receive hydroxyurea to induce the protective effects of increased fetal hemoglobin production. They may also benefit from blood transfusions especially during vaso-occlusive crises.
The company was founded as Genetix Pharmaceuticals in April 1992 by MIT faculty members Philippe Leboulch and Irving London. [7]In 2001, Walter Ogier was appointed chief executive officer of Genetix Pharmaceuticals, and the company was focused on the development of LentiglobinTM for the treatment of sickle cell disease and thalassemia major (beta-thalassemia), the two most globally prevalent ...
CEO Andrew Obenshain said the latest restructuring would help support the marketing of its three gene therapies, including the recently approved sickle cell disease treatment, Lyfgenia.
Serving as the inaugural patient with sickle cell disease to undergo treatment using the revolutionary gene-editing technology CRISPR, she became one of the earliest individuals to experience CRISPR intervention. Although CRISPR had been extensively discussed and lauded, its application had primarily been confined to laboratory cell manipulation.
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