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.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER