Vertex gets more in vivo gene editing Verve

Vertex’s latest tie-up in in vivo gene editing, with Verve Therapeutics, raises plenty of questions. First, the companies are not saying what liver disease they are targeting – although this is not unusual for these types of deals, as shown by the table below. Next, the agreement could spur doubts about Vertex’s other forays into in vivo gene editing including its most recent, a collaboration with Mammoth Biosciences. Mammoth is developing “ultra-small” Crispr systems, while Verve traditionally uses more precise base editing, which avoids the potentially harmful double-stranded DNA breaks seen with Crispr/Cas9. However, yesterday’s press release does not mention base editing – hopefully it will soon become clear whether Vertex and Verve are indeed using this technology, or something else. To complicate matters further, Verve licenses the base editing tech from Beam Therapeutics; that group’s in vivo efforts are largely focused on liver diseases, while Verve, until now, has concentrated on cardiovascular projects, although its agreement with Beam also covers two undisclosed targets. Verve, which took its lead asset, VERVE-101, into the clinic last week, wasted no time in carrying out a $225m fundraising; its stock slumped 13% this morning.

The table in this story has been updated to include the Moderna/Metagenomi deal.