New targets.
Breakthrough medicines.
We discover and develop novel drugs that act directly on disease-causing RNA.
Drugging RNA
Previously thought to be just an information molecule, we now know that dysregulated RNA is at the root of many human diseases. RNA is thus at the forefront of drug discovery.
The biological function of RNA is dictated not only by its nucleotide sequence and secondary structure, but also by its 3-dimensional structure.
We can cure diseases by developing new drugs that bind directly to RNA, altering its structure and thereby its function.
A Platform Anchored by NMR
We employ a powerful, iterative biophysics and computational engine that addresses the challenges and limitations that have historically hampered efforts to drug RNA.
We utilize NMR (nuclear magnetic resonance spectroscopy)-derived ligand binding data to guide molecular dynamics simulations and build accurate structural models. This structural information accelerates drug optimization.
The TargetRNA platform increases the success rate of lead compounds entering preclinical testing and can be applied to any disease-causing RNA.
High throughput biophysical screening
NMR
Structural model
