First RNA editing therapy nears clinic, as Wave ask regulators to greenlight human tests

An RNA edit­ing ther­a­py may be head­ed in­to hu­man test­ing for the first time, af­ter Wave Life Sci­ences sub­mit­ted an ap­pli­ca­tion to bring the treat­ment in­to the clin­ic, the com­pa­ny told End­points News in an ex­clu­sive in­ter­view.

Un­like gene edit­ing, which us­es tools such as CRISPR to make one-time, per­ma­nent changes to DNA, RNA edit­ing ther­a­pies would cor­rect ge­net­ic dis­eases by al­ter­ing short-lived mes­sen­ger RNA mol­e­cules. It’s akin to cor­rect­ing a ty­po on pho­to­copies of a doc­u­ment rather than al­ter­ing the orig­i­nal.

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.