Dr. He Jiankui dragged everyone into a brave new world when he edited human embryo genomes to be resistant to HIV and those embryos were implanted and carried to term birth of twin girls. This was about as big an explosion in science as has ever occurred.
The news drowned out another development from October that was also a milestone for biotech/pharma: a clinical hold on the first CRISPR-based genome surgery based trial was removed by the FDA.
This trial tested a more elegantly designed therapy: ex vivo genome surgery to render a thalassemic or sickle cell anemic patient’s blood cells capable of producing the normal hemoglobin protein, followed by transplant of those improved cells back into the patient.
The pioneering trial was fated for a clinical hold due to its trailblazing status, but removal of the clinical hold clearly signals the FDAs desire to usher in the new era of these drugs.