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A spotlight on biotech companies focused on translating RNA editing to medical therapies.

Thorsten Stafforst remembers being told to stop wasting his time.

It was early last decade and scientists across the world were buzzing over a new tool, called CRISPR, that could precisely alter human DNA. Working in the German college town of Tubingen, Stafforst and fellow researchers at the local university were instead engrossed by the prospect of rewriting RNA, DNA’s chemical cousin.

“Everybody told me, ‘Why do you want to edit RNA?’” Stafforst said. “You can edit DNA now; that doesn’t make sense.”

Yet in 2012  and, shortly afterwards, a group at the  [ led byJoshua Rosenthal, now at MBL] figured out how to use a naturally occurring enzyme to swap out single “letters” in RNA sequences. Their discovery drew from research into the biology of  and , which are adept at rewriting their own RNA. And as with CRISPR, the findings pointed to a novel way of treating disease. In a world newly enchanted by gene editing, however, their papers were met with far less acclaim.

More than a decade later, RNA editing is a fast growing corner of the biotechnology sector. About a dozen companies, from privately held startups to established biotech firms, are pursuing the technology. One already has early, but promising, clinical trial results. Others could follow soon. And large pharmaceutical companies, such as ,  and , have taken an interest.

Source: RNA Editing: Emerging From CRISPR's Shadow | International Biopharmaceutical Industry (IBI)