Watch out, CRISPR. The RNA-editing race is on

Abstract

Joshua Rosenthal isn’t your typical biotech entrepreneur. The cephalopod scientist at the Marine Biological Laboratory in Woods Hole, Massachusetts, has spent most of his life studying the nervous systems of squid—along with the occasional octopus. But in April 2018, Rosenthal found himself in Boston pitching to investors at Atlas Venture an idea for a new kind of therapy, inspired by a mechanism that squid use to edit their RNA. RNA, a short-lived cousin to its better-known partner DNA, is the blueprint for protein production in cells. Rosenthal told the Atlas investors about how squid and octopuses make prolific use of an enzyme called ADAR to catalyze thousands of single-letter changes to their RNA code. Those minor edits alter the structure and activity of proteins that control electrical impulses in the animals’ nerves. Humans have ADAR enzymes in our bodies, too, where they do the same thing, just less prolifically. Rosenthal’s