Sickle Cell And Beta-Thalassemia Bend To Gene Manipulation By CRISPR/Vertex And Bluebird
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.
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The Swiss major envisions developing an accessible in vivo gene therapy for SCD that could potentially be administered once directly to the patient without the need to modify cells in a lab.
With gene therapies for sickle cell disease looming and two new expensive therapies of to a slow start, access challenges could prevent a large portion of patients from receiving treatment, a report from the US National Academies of Sciences, Engineering and Medicine concludes. The report urges development of new payment models, offering five ideas.
Public Company Edition: Drug developers continue to take advantage of investors’ preference for biotechnology investments, with CRISPR, Acceleron and BioNTech raising massive sums. The wealth isn’t spreading to everyone, however, as DBV Technologies is restructuring and cutting jobs.