Epigenos Biosciences brings epigenetic precision to gene therapy
By Nancy Lamontagne | This article originally appeared via NCBiotech's blog
Gene therapy continues to advance, with 26 gene therapies approved by the U.S. Food and Drug Administration (FDA) as of April 2025. Yet key challenges remain, especially in managing the level and duration of gene expression following treatment. Epigenos Biosciences Inc., a spinout from the University of North Carolina at Chapel Hill, is addressing this gap by harnessing epigenetics to develop therapies with greater precision and control.
“We hope to change the way people do gene therapy in the future,” said Joe Ruiz, co-founder of Epigenos. “By offering a way to control gene expression after treatment, we can make therapies safer and more adaptable — ultimately improving outcomes for patients who need them most.”
From lab to market
Epigenos was formed after Ruiz recognized the potential of technology developed by Nate Hathaway, Ph.D., co-founder and associate professor at UNC’s Eshelman School of Pharmacy. Working with Hathaway, Ruiz drew on his experience in launching startup companies to turn the technology into a commercial venture. In 2020, Epigenos received its first grant and officially launched.
Hathway’s technology uses natural epigenetic processes to dial gene activity up or down, rather than rewriting the genetic code. The company is using this to develop therapies for rare diseases and cancers driven by a single gene that is inappropriately turned on or off.
In conditions like Friedreich’s Ataxia — a rare genetic disorder that causes progressive damage to the nervous system — restoring just 50% of normal gene activity can be curative. However, traditional gene therapies often deliver too much, which can harm the heart. They've struggled to move forward because those therapies can’t be dialed back. The Epigenos approach allows fine-tuned, reversible control, delivering just enough to be effective, without risking toxicity.
“People are just starting to realize how important epigenetics is and how it affects disease, but no one is trying to use it as a therapeutic approach,” said Ruiz. “Precision delivery of gene therapy is crucial for Friedreich’s Ataxia and other rare diseases with similar therapeutic windows, which is where we're focusing our expertise.”
Finding the right business path
The company’s original technology relied on the gene targeting capabilities of the gene-editing technology CRISPR-Cas, which is heavily encumbered by existing licenses. During early business development, it became apparent that the sublicensing required for commercialization would pose a significant burden for a startup.
This led to the pursuit of an alternative technological path based on a technology called Zinc Fingers, a gene targeting technology developed before CRISPR. Since the Zinc Fingers patent has expired, using it would not require licensing to commercialize the Epigenos product.
With a $250,000 small business loan from the North Carolina Biotechnology Center in 2022, Epigenos conducted proof-of-concept studies that demonstrated that this older gene-targeting technology could be used with Hathway’s novel technology, offering a quicker path to commercialization. The loan also enabled the company to conduct a Freedom to Operate analysis, which confirmed that incorporating the Zinc Fingers technology wouldn’t infringe on any existing intellectual property.
"Epigenos uses an innovative approach that overcomes several hurdles with conventional gene therapies, providing a new avenue to cure patients and transform lives," said Jen Greenstein, senior director for the Focused Initiatives Team at NCBiotech. "They enable the ability to tune dosage and reverse the gene therapy, and they can expand disease targets by removing size restrictions on the genetic components delivered by AAV."
“NCBiotech is happy to support Epigenos in the development of its novel epigenetics platform," said Jason Doherty, senior director for investments in NCBiotech's Emerging Company Development group. "Epigenos’ ability to finely regulate gene expression offers tremendous promise to patients in a variety of disease indications for which traditional gene therapy approaches have yet to succeed."
Growing the network
Ruiz said that the NCBiotech portfolio managers he worked with — Jen Greenstein and Jason Doherty — were instrumental in connecting with other experts in the area.
“Through their efforts, I was able to secure two key collaborators with top-notch business credentials to help guide our business development efforts,” said Ruiz. “One of these is Joe McMahon from Bioventure Partners, who has worked with many startups in the area. The other collaborator has key expertise in epigenetics and is helping us identify sources of seed money and strategic partners.”
Epigenos also participates in NCBiotech’s Therapeutics Portfolio Company Lunch program, which brings CEOs from different portfolio companies together every quarter for ‘lunch and learns’ on various topics, including those suggested by the participants. “It's great to see other companies in the ecosystem that have portfolio loans and are at the same stage we are or are maybe a little more advanced,” said Ruiz. “We can learn a lot from each other.”
The guidance and transparency offered through the NCBiotech loan process have been critical in shaping the company’s path toward commercialization. "The staff offers honest, actionable feedback that helps us know exactly what’s needed to move forward,” Ruiz said. “Their input helps me understand what steps I need to take to push our technology toward commercialization and be competitive for the next round of funding."
"It has been a pleasure to work with Joe Ruiz, a strong leader and a technical expert who's a staple of our biotech community," Greenstein said.
Looking ahead
Last year, the company received a Kickstart Venture Award, which supported customer discovery efforts that helped it improve its pitch so that the technology’s capabilities more clearly aligned with market needs. As a result, they’ve received several positive responses from potential strategic partners.
“We’re looking to partner with gene therapy developers aiming to improve the precision and durability of their treatments,” said Ruiz. “We offer a path to making their first-generation products even better.”
In addition to securing partners, the company is working to raise additional funding to support animal studies that will thoroughly characterize their gene therapy system, including dosing levels, timing, stability and the ability to modulate expression after delivery. These foundational experiments are critical for developing a reliable and effective therapy before trials can begin in patients.
Previous coverage of Joe Ruiz and Epigenos:
UNC KickStart Venture Services celebrates awards to university startups
