Sickle Cell And Beta-Thalassemia Bend To Gene Manipulation By CRISPR/Vertex And Bluebird
BY ALEX SHIMMINGS Executive Summary Data for new genetic methods to tackle severe hemoglobinopathies presented at the EHA meeting bring more confidence for bluebird’s gene therapy LentiGlobin and early suggestions of a functional cure with CRISPR gene editing with CRISPR Therapeutics/Vertex’s CTX001.
33 / August 2020
CRISPR Therapeutics AG and Vertex Pharmaceuticals Inc.’s investigational CRISPR-based gene editing therapy CTX001 looks like it could offer a new treatment strategy for beta-thalassemia and sickle cell disease, new data presented at the virtual European Hematology Association meeting suggest. The data came amid more mature results also presented at the virtual meeting for bluebird bio Inc.’s gene therapies LentiGlobin and betibeglogene autotemcelin the same indications, and together they reinforce the potential for genetic manipulation to provide lifechanging treatment for these conditions. In recent years, the two hemoglobinopathies have seen novel treatments come to the market that ameliorate their symptoms but these new genetic-based approaches seek to tackle their underlying genetic causes and offer the hope of functional cures.
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