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Roche looks to genetic modifiers for new drug targets, teaming up with Scenic Biotech in $375M deal

Roche is gambling on a new way of discovering drug targets and, ultimately, promising to infuse more than $375 million into a small biotech if all goes well. A spinout of the Netherlands Cancer Institute and Oxford University, Scenic Biotech set out to pioneer a field that’s gaining some traction among top VCs in the US: to harness the natural protecting powers of genetic modifiers — specific genes that suppress a disease phenotype.

After each getting a PhD from the prestigious Dutch research center and spending time in the Boston/Cambridge biotech hub, Thijn Brummelkamp and Sebastian Nijman built their functional genomics platform and named it Cell-Seq. The tech allows scientists to sift through all 20,000 genes in haploid cells (which have only one set of chromosomes), identify the genes that modify a given disease, and create a library of maps pinpointing them.

“There have been other modifiers discovered but these have mostly been discovered by serendipity,” Nijman, who’s also the CSO, told Endpoints News. “What we are now doing is doing this systematically.”

Oscar Izeboud, a former banker who was recruited to be CEO this July, put it this way: “It’s a very fast way to find a needle in a haystack.”

Once they find it, the strategy is to then find gene products — proteins — that can be targeted with small molecule drugs or antibodies. In most cases they would be enzymes, such as QPCTL, which Scenic believes is a druggable modifier of the CD47 checkpoint.

The other internal efforts have mainly focused on rare inherited metabolic disorders, where Nijman and Izeboud believe the 20-people-and-growing team can handle the clinical work.

Their new partners at Genentech are interested in an area that Scenic has not gone into. In addition to the undisclosed upfront, the deal covers target selection fees as well as milestones and royalties.

Scenic, though, isn’t the only startup claiming to have the necessary tools to unpack genetic modifiers — an idea that’s central to the mystifying notion that people born into the same family, with the same genes, don’t always have the same disease.

San Francisco-based Maze Therapeutics launched last year with $191 million from a syndicate led by ARCH and Third Rock, boasting a similar genetics platform designed to unearth new targets. Nessan Birmingham’s Triplet Therapeutics, meanwhile, is focused on the DNA level, leveraging antisense oligonucleotides to hit the DNA damage response pathway.

In comparison Scenic’s beginning was much more humble, raising €6.5 million from European firms BioGeneration Ventures and INKEF Capital, with some support from the university-affiliated Oxford Sciences Innovation and later the Dutch government.

But it’s not stopping Nijman from dreaming big.

Aside from the big Roche collaboration, he also has in his corner seasoned drug such as GSK vet Chas Bountra and cell therapy expert Ton Schumacher, who’s just embarked on his own new venture with Arie Belldegrun and David Chang. If anything, the buzz that rivals are generating is welcome.

“People are starting to realize and wake up to the fact that this is now possible,” Nijman said. “We can now go after this type of drug target.”

Source: EndpointsNews