A Cambridge biotech drugging the molecular edits cancer uses to survive. The first to put an RNA-modifying enzyme inhibitor into a human being.
CAMBRIDGE, UNITED KINGDOM | FOUNDED 2015 | CLINICAL-STAGE
In a building off the Cambridge Biomedical Campus, a small team checks a number that almost nobody else in the world can check: how a single patient's RNA changed after swallowing a pill. The pill is STC-15. The patient is in a Phase 2 sarcoma trial. And the entire premise - that you can drug the enzymes that edit RNA - was, until recently, a question for academic papers, not pharmacies.
STORM Therapeutics is what happens when a footnote in biology grows up. RNA, the molecule we were taught simply carries instructions from DNA to protein, turns out to be heavily annotated - chemically tagged, methylated, edited in flight. Cancer reads those annotations. STORM writes drugs that erase them.
For decades, drug hunters chased DNA and proteins. RNA sat in the middle, treated as a courier - dutiful, temporary, beneath suspicion. The inconvenient truth was that RNA gets modified more than 150 different ways, by roughly 300 enzymes, and that those modifications quietly decide which genes get translated and when. Cancer, it turns out, is an excellent forger.
The opportunity was enormous and, conveniently for a biology textbook, almost entirely ignored. A target space of hundreds of enzymes, untouched by any approved drug. The catch: to drug an RNA-modifying enzyme, you first have to prove the modification matters, then prove you can measure it, then prove you can change it without breaking everything else. Three impossible-sounding steps. STORM took them as a to-do list.
Fig. 1 - The math of a frontier: a vast target space, and exactly one company that had gotten a drug from it into a patient.
STORM was spun out of the University of Cambridge in 2015 by Professor Tony Kouzarides and Professor Eric Miska, two scientists who had spent careers on the regulatory machinery sitting on top of the genome. Kouzarides had helped build the modern understanding of how chromatin is chemically marked; he would later be knighted for services to healthcare innovation. The bet was simple to state and very hard to fund: RNA modifications are a drug target, and we are early.
Being early is romantic in retrospect and terrifying in real time. There was no playbook, no competitor to copy, no validated target to fast-follow. So STORM did the unglamorous thing - it built the tools first. An RNA mass-spectrometry and analytics platform to actually read the modifications, then a discovery engine to design molecules against the enzymes that write them.
SCIENTIFIC FOUNDER
Cambridge professor, chromatin and epigenetics pioneer, knighted for services to healthcare innovation.
SCIENTIFIC CO-FOUNDER
Cambridge professor whose RNA biology research helped seed the company's platform.
CHIEF EXECUTIVE
Leads STORM through the clinical-stage chapter: Phase 2, partnerships and the Series C.
STC-15 is an oral, first-in-class small-molecule inhibitor of METTL3 - the enzyme responsible for adding the m6A methyl mark to messenger RNA. That mark is one of the levers cancer stem cells pull to keep dividing. Switch METTL3 off, and the forged instructions stop getting written. It sounds tidy. Getting it from a Nature paper to a patient's bloodstream took the better part of a decade.
The science arrived with receipts. STORM published data in Nature showing its METTL3 inhibitor worked against acute myeloid leukemia, then data in Cancer Discovery showing METTL3 inhibition could rouse the immune system against tumors. In 2022 the program cleared regulators to begin first-in-human dosing - the first time any RNA-modifying enzyme inhibitor entered a clinical trial. Beyond METTL3, STORM has advanced first-in-class inhibitors of the tRNA enzyme METTL1.
Oral METTL3 inhibitor. First-in-human, first-in-class. Now in Phase 2 for selected sarcomas after durable Phase 1 tumor regression.
First-in-class inhibitors of a tRNA methyltransferase, presented at the ESMO Targeted Anticancer Therapies Congress.
RNA mass spectrometry plus target biology - the instrument that turns "RNA is modified" into "here is a molecule that changes it."
Fig. 2 - A decade compressed. Note the gap between "interesting paper" and "patient dosed." That gap is the whole job.
In April 2026 STORM raised a $56 million Series C - funded entirely by existing backers including M Ventures, Pfizer Ventures, Taiho Ventures, IP Group, UTokyo IPC and Fast Track Initiative. There is a particular kind of validation in returning investors writing the whole check: people who have already seen the data deciding to see more of it. The money is pointed squarely at the Phase 2 sarcoma study, designed to support a potential accelerated approval path.
The proof is not only financial. In Phase 1, STC-15 showed durable tumor regression across multiple sarcoma subtypes. And in January 2026 STORM signed a collaboration with AlidaBio to use next-generation sequencing platforms to measure exactly how m6A marks shift in treated patients - turning "we think it works" into "here is the molecular fingerprint."
Fig. 3 - Capital follows conviction. The Series C came from the same investors who funded the last one.
STORM's stated aim is to harness RNA epigenetics into a new class of medicines - first in oncology, with an eye toward inflammation, viral infection and CNS disease. The ambition is bigger than any single molecule. If STC-15 works, it does not just treat sarcoma; it proves a category. It tells every other drug hunter that the 150-plus modifications and 300-odd enzymes are fair game.
That is the quiet leverage of being first. Wilde would have appreciated the irony: the most radical thing a company can do is make a wild idea look ordinary. STORM wants RNA-modifying enzymes to become as unremarkable a drug target as a kinase. Unremarkable, here, is the goal.
Return to where we started: a 14-person team reading how one patient's RNA changed overnight. A decade ago that sentence would have been science fiction - there was no drug to give, no enzyme cleared as a target, no platform sensitive enough to catch the shift. STORM built each of those, in order, and then handed the result to a person with cancer.
The Phase 2 readout will decide a great deal. But the harder thing - proving that RNA epigenetics is somewhere you can actually find medicine - has already happened. The room is no longer asking whether the idea is real. It is asking how far it goes. That is a very different room than the one Kouzarides and Miska walked into in 2015.
STORM Therapeutics didn't just enter a field. It made the field credible enough to crowd into.