Small molecule drugs hold the potential to change the behavior of disease-causing RNA
Today, the majority of drugs and pharmaceuticals on the market that treat human disease are designed to target a specific protein. But University of North Carolina at Chapel Hill startup Ribometrix has a very different, revolutionary approach to new drug discovery that’s proving to be a breakthrough in the industry.
Ribometrix’s novel approach has the potential to improve millions of lives by offering new treatments for a range of diseases, ranging from cancer, neurological disorders, immunological diseases, and rare diseases such as Huntington’s Disease.
“What’s really exciting is that our specialized technologies give us the ability to go after a whole series of targets that were once considered un-targetable,” says Katie Warner, co-founder and director of research at Ribometrix. “All proteins – including those currently viewed as undruggable – are made from a messenger RNA. The Ribometrix platform allows us to potentially target any of these proteins by targeting those RNAs with small molecules. This is not a small idea, and has the potential to be a big game changer for what drug discovery can do.”
Until now, many didn’t think the science behind this exciting technology would be applicable to drug discovery. But Ribometrix figured out how to drug RNA in a way that’s systematic. One disease Ribometrix currently is pursuing is Huntington’s Disease, whose sufferers have a tremendous, unmet need for therapies and treatment. According to the Huntington’s Disease Society of America, the disease is a fatal genetic disorder that deteriorates a person’s physical and mental abilities and has no cure.
“With Huntington’s Disease, it’s a single gene that’s clearly the problem, but the protein has proven extremely challenging to target. We are able to avoid many of these challenges by instead specifically targeting the RNA associated with the pathological gene,” says Warner. “This presents a tremendous opportunity for us to make an impact.”
The company was founded in 2015 by Kevin Weeks, a Kenan Distinguished Professor of Chemistry at UNC-Chapel Hill, along with Warner, his former undergraduate student, who now serves as Senior Director of Research for the company. Over lunch one day – after reconnecting during Warner’s Ph.D. work – self-professed RNA nerds Weeks and Warner decided to start the company.
“We always knew it would be possible to drug RNA using these technologies, but we didn’t know if anyone outside the field would think it was plausible,” says Warner. Before they could launch a company, however, the two scientists realized they would need the right people and support.
To help turn their idea into reality, Weeks and Warner turned to UNC’s KickStart Venture Services. KickStart is part of the Innovate Carolina network, a comprehensive set of programs that ensures faculty, students and staff at UNC-Chapel Hill have the knowledge, tools and resources they need to turn their ideas into real-world innovations. KickStart Venture Services supports early-stage IP-based companies at Carolina in a variety of ways: business development, coaching, mentoring, funding, and making connections to key service providers, management, investors and space providers. In addition, Innovate Carolina provided additional support to Ribometrix with patent and market research services.
With the help of KickStart Venture Services and the Innovate Carolina network, Ribometrix was able to incorporate, recruit their first CEO, apply for NIH grants, find temporary lab space and create the business structure needed to continue to move forward with their work. And the company is growing at a steady pace. With eight full-time employees, it is looking to grow with additional employees in 2018.
“The university’s strong support network is critical to being successful – especially if you’re not business development experts,” says Warner. “The company was made possible with technology developed at UNC, with support from the UNC ecosystem. The initial ideas and innovation came from the academic side, but we were able to transfer this knowledge into something tangible and entrepreneurial for the greater good.”
The company continues to attract investors as well. In 2017, it raised $7.5 million in seed capital to support the translation of its research on RNA-targeted small molecules, which allows the company to continue developing the platform and continue discovering new drug candidates.
With headquarters in Durham, Ribometrix is proud of its efforts to keep the company local, hiring employees that are North Carolina based or have North Carolina ties. Warner adds, “As a UNC alumnus, I am so pleased we will make an impact in North Carolina. Not everyone has to move to Boston to have a rewarding career in science.”
In the coming year, Ribometrix will continue to prove that its technology can be applied systematically in drug discovery as well as raise additional funding to expand its staff. In addition, the company will also focus on expanding its target pipeline.
“For upcoming scientists, if you have an opportunity, take a leap and go after it,” says Warner. “Sometimes it won’t pay off, but when it does, it’s exciting and fulfilling to see the potential lives touched and the impact you’ve been able to have.”
Story courtesy of Innovate Carolina, photos by Lars Sahl