The treatment, manufactured by Vertex, is the first-ever approved therapy that utilizes CRISPR-based gene editing technology to treat eligible patients.
Stem cell therapy and bone marrow transplants are currently the only pathways to cure sickle cell disease and beta thalassemia, however, they involve a lot of risks.
The Innovative Genomics Institute (IGI) has established an 'Affordability Task Force' to tackle the issue of expanding access to these novel therapies.
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