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Vertex and CRISPR Therapeutics submit BLAs to FDA for exa-cel

Pharmaceutical Technology

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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Strategies to Successfully Manage Complex Cell Therapy Clinical Trials

XTalks

β€œAt Medpace, we go further by having a specialist regulatory technical team support our clinical trials, particularly for cell-therapy studies.” Medpace has IP experience with both autologous and allogeneic cellular products, ex vivo and in vivo gene transfer, and a number of different gene-editing mechanisms. If so, why?