A landmark gene-editing treatment for sickle cell disease moves closer to reality

https://www.npr.org/sections/health-shots/2023/10/31/ [login to see] /a-landmark-gene-editing-treatment-for-sickle-cell-disease-moves-closer-to-realit
The Food and Drug Administration may soon approve a therapy that uses the gene-editing technique called CRISPR to treat sickle cell disease. It would mark the first time gene editing moves from the lab into clinical use.

A committee of advisers to the FDA meets Tuesday to review the scientific evidence for the treatment, including whether sufficient research has been done to assess its long-term safety. The agency has until Dec. 8 to make a decision about the therapy, known as "exa-cel."

In an analysis posted by the FDA Friday, agency scientists conclude exa-cel is highly effective at preventing episodes of excruciating pain that plague sickle cell disease patients. The treatment worked in 29 of 30 patients followed for at least 18 months and doesn't appear to cause any serious short-term safety concerns, the FDA concluded.