A new sickle cell anemia treatment that would be the first to use the CRISPR gene-splicing tool could be the first such medication on the market as it gains another step of approval from the FDA, NBC News reports. Called exa-cel, the treatment eliminates the need for donor cells and instead “edits” the DNA of the patient directly, eliminating the damaged section of the cells’ DNA to essential eliminate the cells’ malfunction.
And FDA advisor board undertook an all-day review of the data related to exa-cel testing. Instead of reviewing the safety and efficacy, the panel is at the stage of looking at secondary effects from “off-target” gene editing. “There seems to be a lot of uncertainty, a lot of unknowns, about what these off-target changes might mean,” said committee member Lisa Lee, an epidemiologist and the director of scholarly integrity and research compliance at Virginia Polytechnic Institute and State University. “Are those unknowns more harmful than not allowing this to go forward?”