Bayer jumps on board Mammoth's ultra-small CRISPR tech with sights set first on the liver
German drug giant Bayer has looked to reinvent itself in recent years, moving on from its past as a primarily consumer health brand into one built around next-gen therapies. Now, Bayer is taking a flyer on one of the children of CRISPR maven Jennifer Doudna’s hallowed lab working on tiny versions of the gene editing tech.
Bayer will pay $40 million upfront and more than $1 billion in potential downstream milestones for up to five in vivo gene editing candidates from Mammoth Biosciences, a Doudna lab spinout developing ultra-small enzymes for easier packaging and delivery into cells, the partners said Monday.
Mammoth, which signed a similar discovery deal with Vertex late last year, is developing CRISPR tools using enzymes roughly one-third the size of Cas9, the DNA scissors that helped make CRISPR famous, to add or delete targeted genes through double-stranded breaks.
The company has developed gene editing systems using both Cas14 and Casɸ, among other variants, which are two smaller versions that allow for far greater flexibility in terms of delivery to target tissues. That means Mammoth’s team can push its editors into smaller adeno-associated viral vectors (AAV) but also into certain lipid nanoparticles (LNP), the technology used to shuttle the mRNA-based Covid-19 vaccines into cells, according to early data.
According to Mammoth CSO Lucas Harrington, the possibility of fitting more “payload” into delivery vehicles could help increase editing efficiency in cells, potentially helping overcome current hurdles for CRISPR tech.
For Bayer, this deal comes a bit more than a year after the German drug giant, best known for its consumer health brand, acquired AskBio, an AAV-delivered gene therapy company whose pickup trumpeted Bayer’s move into next-gen therapeutics and away from its staid pharmaceutical past. That biotech came aboard with expertise in building a better AAV vector, a puzzle box technology with some safety concerns that companies all across the gene therapy sector are looking to solve.
Soon after that pickup, Bayer announced it would prop up a gene therapy umbrella within its pharmaceuticals business with the goal of adding a diversified set of modalities to drive its next-gen push.
“Bayer was really looking for in vivo applications, and I think our proposition is that smaller CRISPR systems make this a perfect combination,” Peter Nell, Mammoth’s chief business officer and head of therapeutic strategy, told Endpoints News. “For us, it’s also how a company runs a business and what they know. You identify people who understand this and aren’t going naively into this.”
Although five target areas are part of the deal, the partners are only saying now that the first target on the list is the liver, a tissue most companies believe is the easiest to hit. From there, the collaboration could run in any number of directions, which will be revealed at a later date. Meanwhile, Bayer and Mammoth plan to work together on a nonexclusive basis leveraging the biotech’s capabilities in ex vivo gene editing, the same sort of technology used to craft off-the-shelf cell therapies.
Back in October, Mammoth signed a similar licensing pact with Vertex worth $41 million upfront and $650 million in downstream milestones for two therapeutic areas that weren’t disclosed at the time of the announcement. A month before, Mammoth announced $195 million in new funding (including a $150 million Series D round and previously unannounced $45 million Series C).