Sanofi turns to a Jennifer Doudna gene editing upstart for next-gen NK cell alliance

Big Pharmas — as well as more than a few biotechs — are on the hunt for the best, cutting-edge gene editing tech available, and today we can tell you about Sanofi’s choice.

The Paris-based giant’s head of oncology research and its chief of genomic medicine have settled on Scribe Therapeutics’ CasX editing tech for their work on NK cells. And the deal with the Jennifer Doudna spinout comes with a $25 million get-to-know-you upfront and a cool $1 billion in biobuck milestones, provided they can neatly knock down all the pins in the deal.

The deal gives Sanofi a chance to jump on a platform that can be used to engineer their ex vivo NK cells, something they’ve focused on quite closely since the $358 million Kiadis buyout in late 2020. Sanofi bought the biotech’s CD38 “knockout” NKs to combine with its anti-CD38 drug Sarclisa, and now wants to take it all one big step down the R&D road with the kind of tech that’s used to improve durability and targeting with more potential for combos.

It’s a non-exclusive pact, leaving Scribe’s gene editing platform open for other partnerships of a similar nature. Royalties are also in play here, but the partners are remaining openly vague about the financials on the big back end — not uncommon at this early stage of the alliance game.

“We have the first candidate in the clinic which is the wild-type supercharged NK cells that we know — especially as we’re thinking about moving into solid tumors — we have to armor these cells, engineer these cells to overcome the suppressive tumor microenvironment. That’s where some of these engineering technologies come into play,” Sanofi global head of oncology research Valeria Fantin says.

The pharma giant has a proprietary manufacturing tech in place that “supercharges” the cells,  adds Christian Mueller, global head of genomic medicine at Sanofi. This new pact with Scribe gives them some more versatility to knock out receptors, better engineer them, and use their NK drugs in combination with other products. His group has been working with Fantin’s crew trying to go to the next level with these cells, “and the missing part of the puzzle was the nuclease.”

“As we move into new areas and new tumors we do want to engineer these, we want to be able to engineer CARs onto these NK cells and knock out certain cell receptors that might be beneficial for the NK cell itself or in combination with any of our other products,” Mueller says.

It’s a big deal for Scribe, one of several biotech startups to come out of Doudna’s prolific lab. In a recent conversation with Endpoints News’ Zach Brennan, Scribe CEO Ben Oakes noted how they fit in with the increasingly crowded scene. And he isn’t reluctant to boast about Scribe:

It’s a little bit disingenuous that you can find a misspelling and perfectly correct it. Prime editors get a little bit better but there are plenty of reasons why they want to say they’re perfect. CasX can find different sites in the genome and with greater specificity, greater delivery and greater activity compared to the original Cas9 molecule.

Ben Oakes

“When we founded Scribe about four years ago now, our goal was to really take a different approach than we were seeing in the CRISPR field, which was to be a little more disciplined and intentional and recognizing that the nucleases that are available are bacterial immune systems,” Oakes tells me. “And so what we do is take novel nucleases and then layered in on top of that some really intensive protein and RNA, we call it molecular engineering, to hone them to be much more exquisite genome editors.”

Of course, we’re seeing a variety of players coming along in the field, each with their own special story to tell. David Liu’s Prime Medicine just set out to see if it can raise a couple of hundred million dollars for its ambitious goals in an IPO. At the beginning of the year, Pfizer teamed with base editing standout Beam — with gene editing tech originated by Liu at the Broad. Berkeley spinout Metagenomi aligned with Moderna, and will likely do more deals. Mammoth, just named an Endpoints 11 company alongside Metagenomi, has pacts in place with Vertex and Bayer. The list goes on.

Scribe is aligning itself with a Big Pharma player that’s been struggling to get its act together on the pipeline. Several of Sanofi CEO Paul Hudson’s picks have ended in troubled waters. And despite the Sarclisa approval, there’s plenty of concerns over whether or not Sanofi can overcome its long track record of slow motion failure — separate from Dupixent and other drugs brought in from outside players.

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