Accent Therapeutics launched Friday with $40 million in the bank to develop cancer treatments based on epitranscriptomics, or the science around RNA structure, stability, function and translation in cell biology.
The Cambridge, Mass.-based biotech is taking aim at RNA-modifying proteins (RMPs), which have been linked to certain cancers in recent studies, the company said. RNA is commonly referred to as the "blueprints" for DNA. And if DNA is the house in this analogy, RMPs are homeowner's choices that modify the structure of the house, says Chief Scientific Officer Robert Copeland, Ph.D.
"All those modifications are what create a personalized unique home. In an analogous way, modifications of RNA molecules define the spectrum of biological functions a cell is going to have," he said. "These modifications control which RNAs are translated into proteins and the modifications themselves affect the structure, function and stability of individual RNA constructs."
Accent is working to understand which RMPs are involved in which cancer indications, and, within those indications, which patients would most likely respond to this type of treatment. It closed its series A round in September, with the support coming from The Column Group, Atlas Venture and EcoR1 Capital. Since then, the startup has finished a systematic target validation study.
"We've detected about 600 genes that encode for proteins involved in RNA modification. We looked systematically at all 600 of those genes across more than 300 different cancer cell lines," he said. These included both solid tumors and blood cancers.
The company has picked out 20 RMPs it believes have "strong implications" in specific cancer indications and prioritized them according to unmet medical need and what it knows about the biology of those targets, including whether they are likely to be a druggable. Accent has narrowed its sights to four targets and is "well on our way" to identifying chemical matter against each one, Copeland said.
Accent is currently a team of 10 and expects to grow to 15 by the end of the year. Over the next few years, it plans to grow to about 25 or 30 staffers, where it will cap its workforce.
How will it execute its mission with such a small workforce?
"We're hiring seasoned folks with experience in drug discovery and drug development, and the ability to manage large groups of scientists at a distance, so we can leverage externalization in an effective and efficient way," Copeland said.