Sickle cell pipeline: not all gene therapies made the same
Permanently restoring hemoglobin is the goal, but companies have different ideas about how to achieve it
A functional cure has become the chief goal of drug development for sickle cell disease, and companies have started clinical trials of half a dozen therapies that could achieve that goal. All involve genetically modifying a patient’s hematopoietic stem cells ex vivo, but the nature of the genetic manipulations and the technologies used to create them vary widely.
This week’s announcement by Graphite Bio Inc. (NASDAQ:GRPH) that it is terminating a Phase I/II trial of the only candidate in the pipeline intended to correct the mutation that causes sickle cell disease, thereby restoring normal expression of adult hemoglobin, leaves the pipeline almost entirely focused on approaches designed to compensate for the mutation by inducing re-expression of fetal hemoglobin. ...