Overview
RNA Therapeutics
In 2006, Dr. Andrew Fire and Dr. Craig Mello received the Nobel Prize in Physiology or Medicine for their groundbreaking discovery of RNA interference (RNAi) – a natural gene-silencing mechanism triggered by double-stranded RNA. Since then, RNA-based technologies have demonstrated success in treating diseases where traditional therapies have limitations, taking advantage of the body’s own cellular processes to control protein expression at its source.
Our Unique Approach at ADARx
At ADARx, we have developed proprietary, next-generation siRNA technologies that are collectively designed to optimize our siRNA sequences and delivery to tissues both in the liver and outside the liver. Using these technologies, we seek to develop novel therapeutics prioritizing chronic diseases where deep, durable target suppression can enable meaningful clinical benefit for patients with significant unmet need.
Our approach centers on three core pillars:
- Sequence engineering: Leverage our proprietary computational platform to generate unique insights from oligonucleotide-enzyme interactions, with the goal of consistently driving differentiated potency and durability for our therapeutic candidates.
- Broad delivery: Unlock the potential of siRNA therapeutic candidates beyond the liver via targeted delivery to extrahepatic tissues, such as adipose, neurons, microglia, skeletal muscle, cardiac muscle and ocular tissues, thereby broadening the landscape of diseases we can seek to address.
- Strategic indication selection: Prioritize diseases, with a focus on chronic conditions, where our technologies have the potential to drive deep, or even near complete, and durable target suppression to enable meaningful clinical benefit for patients with significant unmet need.
By combining deep siRNA expertise, an unwavering commitment to innovation, and a patient-centric focus, ADARx is advancing programs designed to selectively silence the target at its source and overcome the limitations of existing siRNA therapeutics to realize the full potential of siRNA therapeutics.
