What We’re Reading: Sickle Cell Disease and Exa-cel Gene Therapy


Ahead of the FDA’s decision (expected Dec. 8, 2023) on Vertex Pharmaceuticals’ gene therapy exagamglogene autotemcel (exa-cel) for severe sickle cell disease (SCD), several recent stories have addressed some of the implications for patients.

Exa-cel is a one-time therapy that uses a patient’s own hematopoietic stem cells that are edited to produce high levels of fetal hemoglobin in red blood cells. Patients receiving this therapy have their own cells edited using the CRISPR/Cas9 technology. The edited cells, exa-cel, will then be infused back into the patient as part of an autologous hematopoietic stem cell transplant.

Related: FDA Sets PDUFA Dates for First CRISPR Gene Edited Therapy

An article in MIT Technology Review profiles a patient with sickle cell disease who participated in the trials for exa-cel. He details the sometimes grueling process, include the 17 weeks total he spent in the hospital. But he says the therapy has changed his life and how symptoms disappeared virtually overnight.

Stat profiles another patient in the trial, a young woman in college. Two years after treatment, she is free of sickle cell and now longer in pain. But the chemotherapy used to condition the body to receive the new gene-edit cells could leave her infertile.

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