Vertex and CRISPR Therapeutics submit BLAs to FDA for exa-cel
Pharmaceutical Technology
APRIL 4, 2023
Formerly known as CTX001, exa-cel is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy. We want to thank the clinical trial participants and the sickle cell and beta thalassemia communities, as well as the physicians, nurses, coordinators, caregivers and friends who support them.”
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