ARTICLE | Product Development
Additional durability data build confidence for gene editing, gene therapies in hemoglobinopathies
Rounding up EHA data from Agios, bluebird, CRISPR-Vertex and Editas
June 13, 2020 12:28 AM UTC
It’s all about durability for gene therapies and gene editing treatments in hemoglobinopathies, and new data suggest programs from partners CRISPR Therapeutics and Vertex as well as bluebird are on track to deliver.
In addition to the progress with new modalities, Agios Pharmaceuticals Inc. (NASDAQ:AGIO) presented data supporting advancement of its PKR agonist mitapivat in α and β thalassemia and sickle cell disease as it also announced receiving $255 million in non-dilutive funding via a deal with Royalty Pharma. ...