(p. 6) Scientists like me can now visualize an ideal scenario for the future of CRISPR medicines: When a 3-month-old starts to develop antibiotic-resistant infections, her primary care doctor orders a DNA test, and 48 hours later, the faulty gene that is preventing the development of a normal immune system is identified. “Not a problem. We will refer your child for corrective CRISPR therapy,” says the physician to the devastated parents.
. . .
(p. 7) To make CRISPR cures a reality, the biomedical community needs to start with regulation. For treatments developed for genetic diseases that affect tens of thousands of people (or, say, if a company tries to take on heart disease, which affects millions), the Food and Drug Administration has a well-established, yearslong review process. But the F.D.A. needs to consider a new regulatory process that could create a more streamlined path for bringing much-needed CRISPR medicine tailored to patients with a one-of-a-kind genetic typo. There is precedent for this: Starting in the late 1990s, the F.D.A. facilitated regulatory pathways for innovation of a then-new class of genomic medicines for cancer — CAR-T therapy — which is now widely used clinically. The same can be done for CRISPR.
For the full commentary, see:
(Note: ellipsis added.)
(Note: the online version of the commentary has the date Dec. 9, 2022, and has the title “We Can Cure Disease by Editing a Person’s DNA. Why Aren’t We?”)