Vertex doesn’t need to do more testing for rogue gene edits from sickle cell therapy trial, FDA advisors say

Just over a month be­fore the FDA makes a land­mark de­ci­sion about the first-ever ther­a­py to use CRISPR/Cas9 gene edit­ing, the agency is pay­ing close at­ten­tion to a hard-to-mea­sure ques­tion: What hap­pens if the treat­ment ed­its parts of the ge­net­ic code that it’s not sup­posed to?

On Tues­day, out­side ad­vi­sors to the agency con­clud­ed that Ver­tex Phar­ma­ceu­ti­cals, which is spon­sor­ing the po­ten­tial­ly land­mark treat­ment, did not need to do more test­ing to find po­ten­tial off-tar­get ed­its in cur­rent clin­i­cal tri­als for its ex­per­i­men­tal CRISPR-based ther­a­py, though it did sug­gest long-term mon­i­tor­ing for such ed­its.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.