Prime editing set to enter human trials

Credit: Microsoft/Bing

Recently David Liu announced that Prime Medicine will likely submit the first human trial application in 2024. The standard version of CRISPR uses an RNA guide to find the editing site in the genome. Prime editing, on the other hand, also uses the same RNA molecule to direct the correction, in short, to specify what to do as well as where to go.

This insight blossomed in Andrew Anzalone’s mind a few years ago during his PhD at Columbia University. The first practical demonstration came with a paper published in Nature in 2019 after joining the Liu’s Lab at the Broad Institute. Since then, this platform has been used in hundreds of experiments to fix all kinds of mutations in vitro and in animal models.

Meanwhile, the company co-founded by Anzalone and Liu has begun work on 18 treatments, the most advanced for chronic granulomatous disease. To learn more, from the eureka moment to the latest developments, we suggest listening to the Close to the Edge podcast and reading Alex Philippidis’ article in GEN.

CRISPR rising stars

Andrew Anzalone (Broad Institute), Jennifer Hamilton (Berkeley) and Cameron Myhrvold (Princeton)

December is time for rankings and forecasts. Let’s start with STAT News celebrating young talents who could become the next generation of scientific superstars. Three CRISPR researchers appear among STAT wunderkinds. As a postdoc at the Broad Institute, Andrew Anzalone helped make a key advance by developing prime editing, where the same RNA molecule specifies the target and the desired edit. Jennifer Hamilton, from Berkeley, works on solving one of the major hurdles of CRISPR-based therapies: delivering the genome editor to the desired cells. Cameron Myhrvold, has since worked at the Broad Institute on developing CRISPR-based diagnostics such as CARMEN and is about to start his own lab at Princeton.