The fatality underscores the risks of one-time, genetic treatments for blood disorders like sickle cell and beta thalassemia, including those already marketed by Bluebird Bio and Vertex.
Presented additional data from the Phase 1 clinical trial ENTR-601-44-101 and new preclinical data supporting ENTR-601-45 for DMD at 2024 World Muscle Society Annual Congress – – On track to submit gl ...