Editor’s note: WRAL TechWire and the Council for Entrepreneurial development are partnering to present a series of profiles and Q&As featuring companies startups and emerging companies participating in the CED’s annual Life Science conference coming up Feb. 28-March 1 in Raleigh. The latest profile is Ribometrix, which is based in Chapel Hill.

Ribometrix

  • Web site: http://www.ribometrix.com
  • CEO: Anil Goyal
  • Contact information: agoyal@ribometrix.com
  • Sub-sector: Biotech and Pharmaceutical
  • Headquarters: Chapel Hill, NC
  • Year Founded: 2015

COMPANY PROFILE

Ribometrix is discovering small molecule therapeutics for rare/orphan and unmet human diseases targeting RNAs using its proprietary disruptive RNA platform technologies.

  • Unmet need: RNAs have been of huge interest and represent a broad avenue to impact a large class of targets. Thus far, RNAs have only partially been targeted with oligonucleotides (antisense, siRNA, microRNA) due to a lack of systematic small molecule screening capabilities and design/SAR tools against RNA.
  • Disruptive Solution: Using its validated platforms, Ribometrix can first systematically discover druggable 3D structures in RNAs (no other drug discovery approach can achieve this robustly and rapidly at a reasonable cost). Second, Ribometrix can perform compound screening to find hit molecules, defining their mode of interaction and providing a specificity assessment in a single assay.

With these platforms, Ribometrix is enabling the rapid discovery of high confidence hits to progress into traditional lead optimization to design drug-like molecules (assets) for Huntington’s Disease, cancer, fibrosis, MS and other high-unmet, rare/orphan as well as broader population diseases.

FOUNDERS/MANAGEMENT TEAM

  • Kevin Weeks, PhD, Founder, Interim CEO​
  • Katie Warner, PhD, Co-founder and Director of Research
  • Anil Goyal, PhD, CLP, Chief Operating Officer
  • Brian Marron, PhD, Drug Discovery
  • Marilyn Thompson, Finance

KEY MILESTONES TO DATE

  • Funding: Total of $1.2M in non-dilutive funding (NIH, SBIR) in 2016
  • Signed exclusive WW license for technology platform from UNC-CH, April 2016
  • Selected for the UNC-Chapel Hill Innovation Showcase 2016
  • Awarded 4 grants from different funding sources showing the validation and strong rigorous review of the innovative ideas of Ribometrix

POWERED BY
Carolina KickStart, CED, NC Biotechnology Center, SBTDC, UNC Chapel Hill Office of Technology Development, NIH

Q&A

  • What is the primary pain point you are seeking to address?

Novel small molecule therapies that target underlying disease mechanisms are desperately needed for high-unmet, rare/orphan as well as broader population diseases such as Huntington’s Disease, cancer, fibrosis, MS etc.

Targeting RNAs to treat these diseases has been of huge interest, however thus far RNAs have only partially been targeted with oligonucleotides (antisense, siRNA, microRNA) due to lack of systematic small molecule screening and design/SAR tools against RNA.

  • What sets your company apart? What’s the “secret sauce”?

Secret sauce is Ribometrix’ disruptive MaP Technologies plus Ribometrix’ scientific team. Ribometrix’s disruptive MaP Technologies systematically discover druggable 3D structures in RNAs.

No other drug discovery approach can achieve this robustly and rapidly in reasonable costs. Ribometrix scientific team has significant know-how and experience in implementing these disruptive technologies in high-through RNA-based drug discovery.

This enables rapid discovery and design of drug-like molecules (assets) for Huntington’s, cancer, fibrosis, MS and other high-unmet, rare/orphan as well as broader population diseases.

  • Why should investors be interested in your company? What is the potential market size?

Investors should be in interested in participating in Ribometrix for its disruptive technology and its potential to develop high value therapies for many unmet diseases. Some of the small molecule therapeutic assets will be progressed by Ribometrix into clinical and commercial development and some of the assets will be licensed to pharma/biotech for development and commercialization.

Having a rich pipeline of therapeutic products (some internal, some partnered) will position the company for acquisition by large pharma or an exit via IPO in the future.

Ribometrix potential products are expected to address a broad range of disease markets, including cancer, fibrosis, multiple sclerosis, liver diseases, Huntington’s disease.

Considering the patient population numbers and unmet needs in these diseases, we believe the total market size could be in the wide range of $5-50 billion. In addition to the value of the potential products, Ribometrix will generate revenue (upfront and milestone payments) from its platform partnerships with large pharmaceutical and biotech companies.