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 remarkable potential for treating diseases where traditional therapies fall short, 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 RNA technologies that offer the versatility to address both rare and common diseases. By leveraging our innovative, highly differentiated RNA platform, we have made significant advancements in discovering and developing life-changing therapeutics.

Key Design Attributes of Our RNA Therapeutics

  • Target knockdown: Provide deep and meaningful reduction of disease-causing proteins.
  • Precision: Specifically silence pathogenic mRNA, sparing healthy cellular components and thereby reducing off-target effects
  • Durability: Provide long-lasting effect with no genome alterations and associated long-term safety concerns

Through cutting-edge science, an unwavering commitment to innovation, and a patient-centric focus, ADARx is poised to potentially set new standards in RNA therapeutics—unlocking a future where previously untreatable conditions can be effectively managed or even overcome.

› RNAi › RNA Editing › Delivery