The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are glimpses of forward momentum as Vertex and Genetix Bio provide updates.
This story was originally published on BioPharma Dive. To receive daily news and insights, subscribe to our free daily BioPharma Dive newsletter. In the years since the approval of Imbruvica for ...
CRISPR gene therapy Casgevy can nearly eliminate sickle cell crises, but difficult stem‑cell collection is delaying access ...
Rilzabrutinib is an investigational, oral reversible Bruton tyrosine kinase inhibitor. The Food and Drug Administration (FDA) has granted Orphan Drug designation to rilzabrutinib for the treatment of ...
Please provide your email address to receive an email when new articles are posted on . ASH issued sickle cell disease research priorities that address seven key focus areas. Topics include end-organ ...
Given the remarkable advances seen with gene therapies for sickle cell disease (SCD), it may seem counterintuitive to suggest the space may be in need of more therapies that have already been ...
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Breakthrough gene therapy for sickle cell disease remains out of reach in Africa
As Uganda rolls out mandatory nationwide screening of newborns for sickle cell disease this month, a gene therapy celebrated ...
This electron microscope image provided by the National Institutes of Health in 2016 shows a blood cell altered by sickle cell disease, top. About two weeks ago, Ediomi Utuk-Lowery got an unexpected ...
Opportunities in the Sickle Cell Disease market include advancing gene-editing therapies like EDIT-301, expanding VOC-reducing treatments such as inclacumab, and exploring novel drug candidates like ...
Meghana Keshavan covers biotech and contributes to The Readout newsletter. SAN DIEGO — Pfizer’s abrupt withdrawal of its sickle cell drug Oxybryta over safety concerns was called “tone deaf” during a ...
This electron microscope image provided by the National Institutes of Health in 2016 shows a blood cell altered by sickle cell disease, top. (National Center for Advancing Translational Sciences ...
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