The term epitranscriptomics was first coined in 2012, when scientists discovered that chemical modifications in the RNA play an important role in gene expression.
RNA (ribonucleic acid) is a nucleic acid present in all living cells. RNA molecules act as messengers carrying instructions from DNA to control the production of proteins, although in some viruses, RNA rather than DNA is the carrier of genetic information.
Extensive research built around the chemical modifications of DNA, or ‘epigenetics,’ has been around for a long time, and epigenetic drugs are already available on the market.
Scientists are now hoping that the equivalent chemical modifications in RNA can also be used to treat disease.
Unlike gene therapy, which provides new DNA to cells, RNA therapy modifies or provides RNA to a patient’s cells. Despite the buzzing popularity of gene-editing technologies such as CRISPR, RNA therapy has the potential to address some of their limitations.
The first company in the world to devote themselves to this brand new field of epitranscriptomics is Storm Therapeutics. A University of Cambridge spin-out, the company is translating the ground-breaking work of the company’s scientific founders Tony Kouzarides and Eric Miska in RNA epigenetics into the discovery of drugs in oncology and other diseases.
“There are many, many more types of modifications and there are many, many more species of RNA in the cell, so there are a lot more intervention points with RNA modifications than there are with DNA,” Keith Blundy, CEO of Storm Therapeutics, told Labiotech.eu. “The difference is we didn’t know these RNA modifications were there until very recently.”
The company was set up with the help of M Ventures—the venture capital arm of the pharma giant, Merck—in 2015 with a seed investment.
“Storm Therapeutics was the first company created in the space, and we believe it is the most advanced company in the epitranscriptomics space,” said Hakan Goker, partner at the venture capital firm M Ventures.
“When we came across the papers from Tony’s lab in epitranscriptomics, we went into the field in more depth. It was quite obvious after looking into the space for a while that this kind of technology could open up a large basket of novel targets that one could aim at in disease.”
The company—which will mainly be focused on treating cancer—has since attracted much attention and funding.
In May of this year, Storm extended its Series A funding, raising an additional £14 million bringing the total Series A financing to £30 million—quite a feat for a company still in preclinical stages.
Storm is currently backed by blue-chip investors: Cambridge Innovation Capital, M Ventures, Pfizer Ventures, Taiho Ventures LLC and IP Group and Seroba Life Sciences.
There are currently only a handful of companies that have publicly stated that they are working in the field of epitranscriptomics. Despite the fact that there are no existing data proving the epitranscriptomics approach works in humans, they are attracting a lot of attention.
“The biology is very new. A lot of what we’re doing here is still to be proven,” explained Blundy. “We’re going to have to decide which are the important modifications, which are the enzymes that make the modification, and then make drugs against them.”
The ambitious company plans to start clinical trails in 2021—just five years after the company officially launched.