Programmable ASO Therapeutics
RNA Actuators create a sequence-specific approach to precisely target regRNA transcripts.
The ability to modify genes and gene expression using various therapeutic techniques has unlocked the potential to treat numerous diseases that were once considered out of reach. Yet many approaches to gene upregulation have significant limitations. By amplifying mRNA to increase healthy gene expression, we believe that we can do better.
RAP Platform™
Map regRNA
Identify RNA Hotspots
Program for Druggability
The Right Tools: RNA Amplifiers
Antisense oligonucleotides are an accurate, efficient and proven way to engage desired RNA targets. Building upon the power of this technology, our RNA Amplifiers can be programmed to engage regRNA targets, producing tunable increases in protein expression. We design RNA Amplifiers to leverage current and future delivery systems to enable drug delivery to multiple different types of tissue throughout the body.
RNA Amplifiers are a new category of synthetic gene regulators
Therapeutic Potential
Adjustable Dosing
Cell- and Tissue-Specific
Hundreds of Diseases
We are building a deep pipeline of therapeutic candidates that have the potential to transform the treatment of a wide variety of diseases.
Partnering to Capitalize on our Potential
Our platform enables expansive opportunities across multiple therapeutic areas. Given the enormity of the potential, we can’t do it all alone. We intend for our approach and unparalleled understanding of regRNAs, oligonucleotide technology and gene expression to open up vast horizons for drug discovery and development.
Partnering with biopharma companies with expertise in a given therapeutic area is integral to maximizing the potential of our platform for patients. Our aim is to empower partners with a rapid, efficient approach to targeting the diseases that matter most to them.