Another biotech player has entered the burgeoning field of epitranscriptomics, or the complex modifications that control RNA. Gotham Therapeutics has launched with $54 million in funding from a syndicate led by Versant Ventures, Forbion, and SR One to develop drugs targeting RNA modifiers.
After years of focusing on the chemical modifiers of DNA, the biotech industry has in the past two years shifted its attention to using small molecules to harness RNA. One wave of companies is working on drugs that directly bind to RNA. Another wave, which includes Gotham and the recently-launched Accent Therapeutics, is focused on the enzymes and proteins that add, remove, or detect modifications to RNA. The end goal of all of these efforts is to treat diseases like cancer by controlling protein expression.
Gotham’s foundation traces back to 2014, when Versant partner Carlo Rizzuto met Weill Cornell Medicine’s Samie Jaffrey, a pioneer in the field of epitranscriptomics. Jaffrey’s lab had developed a technique for mapping out a modification called m6A (N6-methyladenosine), which tacks a methyl group onto an adenosine base in RNA, an addition that can lead to a host of disease-causing alterations. “At the time, it was really a white space,” Rizzuto says. Although the science wasn’t yet ripe for translating into actual drugs, the intervening years brought a flurry of activity—new labs joining the space and significant progress in identifying chemical modifiers and untangling their link to human disease.
In 2017, Versant put seed money into Gotham. “The critical deliverable for the seed phase that we ran was really to see if we could generate high-quality chemical matter against these targets,” Rizzuto says. Satisfied with the work Gotham’s small team managed to accomplish toward that goal, Versant led today’s current investment round.
Gotham, based in New York City, is using a semivirtual model to explore this complex new space, says CEO Lee Babiss. The company will pursue treatments for oncology, immunology, and neurology.
“It’s still a very early field,” Babiss says. “We feel very privileged to be working in a whole new area of biology and really creating new rules.”