Programmable Therapeutics
The ability to modify genes and gene expression using various therapeutic techniques has unlocked the potential to treat numerous diseases that used to be considered out of reach — yet many approaches to protein replacement have significant limiations. By targeting regulatory RNAs using custom-built oligonucleotide drug candidates, we believe we can do it better.
Our goal: precise, potent and durable therapeutics that can be programmed to treat thousands of diseases.
Antisense oligonucleotides are an accurate, efficient and proven way to engage desired RNA targets. Building upon the power of this technology, our RNA Actuators can be programmed to engage regRNA targets, producing specific changes in protein expression. We design RNA Actuators to treat diseases throughout the body.
RNA Actuators are a
new category of synthetic gene regulators
Our RAP Platform™
Map regRNA
Identify RNA Hotspots
Program for Druggability
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 the 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.