ADARx Technology
At ADARx, our innovative RNA technology is designed to produce targeted therapeutics capable of tackling a broad range of diseases. By combining our proprietary technology, deep scientific expertise, and an efficient drug discovery process, we are pioneering the next generation of RNA therapeutics with superior efficacy, tolerability, precision, and durability.
- Compact: Our RNA candidates are precisely engineered to be compact, enabling efficient cellular delivery and optimal stability.
- Modular: Our RNA molecules have a modular architecture that can be easily adapted to a variety of tissues, cell-types and therapeutic targets.
- Versatile: The delivery components are designed to meet the unique requirements of each disease, ensuring that our RNA-based treatments achieve consistent and effective therapeutic outcomes across a broad range of indications.
Our next-generation technology has the potential to enable precise mRNA silencing, reduce off-target effects and provide maximal therapeutic benefits. By directly addressing the disease at its molecular origins, ADARx product candidates are designed to be transformative, long-lasting, and life-changing treatments that redefine what is possible in-patient care.
RNAi
RNA Editing
Delivery
RNA interference (RNAi) is a naturally occurring cellular process that regulates protein expression. It involves small-interfering RNA (siRNA) molecules that target and degrade specific messenger RNA (mRNA) transcripts. After integration into the RNA-induced silencing complex (RISC), the siRNA guides the complex to selectively bind to and catalytically degrade the target mRNA, halting the production of the encoded protein.
This intrinsic mechanism forms the basis for siRNA therapeutics—medicines designed to selectively silence disease-causing proteins at the molecular level.
ADARx Proprietary MST™ (mRNA Silencing Technology)
At ADARx, we apply our proprietary MST™ technology across all RNAi programs, optimizing each drug candidate for precise targeting, deep target reduction, and sustained durability. Our advanced RNA technologies enhance the key elements of delivery, RISC engagement, and RNA sequence optimization. This integrated approach ensures that each MST™-based therapeutic is engineered for potent, precise and durable therapeutic impact.
Genetic mutations can sometimes generate faulty mRNAs that lead to the production of dysfunctional, disease-causing proteins. Within cells, naturally occurring RNA editing mechanisms adjust the genetic message after it has been transcribed from DNA but before it is translated into protein. Enzymes known as ADARs (adenosine deaminases acting on RNA) facilitate this dynamic editing process by selectively modifying RNA sequences. Leveraging RNA editing offers an innovative therapeutic approach to correct pathogenic mutations at the RNA level, thereby restoring proper protein function without altering the underlying DNA.
ADARx Proprietary EditR™ (RNA Editing Technology)
EditR™ represents a foundational breakthrough in RNA editing technology, enabling the precise correction of disease-causing mutations at the RNA level. We have engineered our RNA editing compounds to achieve enhanced editing efficiency with high precision. Building on these advancements, our ongoing research has demonstrated promising in vivo RNA editing of disease-causing mutations, and the successful restoration of normal protein production. These results highlight the transformative potential of EditR™ technology in the development of novel RNA editing therapeutics.
Effective RNA therapeutics require precise delivery to the cells that need them most. Without targeted delivery strategies, RNA molecules often fail to efficiently penetrate target cells, leading to lack of effect.
ADARx CTDTM (Cell-Targeted Delivery)
With our CTD™ technology, we skillfully engineer targeting ligands that deliver our RNA therapeutics to the target cell types and tissues through receptor-mediated internalization. Our approach has the potential for selective cellular uptake and maximal therapeutic effects.
Targeting the Liver
We have developed proprietary clusters of N-acetylgalactosamine (GalNAc) ligands, that enable efficient binding to the asialoglycoprotein receptor (ASGPR) highly expressed on liver cells. By incorporating our proprietary GalNAc clusters into our RNA drug candidates, we design our product candidates to have efficient delivery to the liver, potentially leading to precise and robust therapeutic effects.
Reaching the Central Nervous System (CNS)
To effectively treat the most serious neurologic diseases such as Alzheimer’s disease, it is critical to deliver RNAi therapeutics directly into the CNS. Ensuring that siRNA molecules efficiently reach neuronal and glial cell targets is no small task. At ADARx, we address this challenge using ligands engineered to engage cell-specific receptors. By conjugating these ligands to our siRNA candidates, we achieve precise targeting, enhanced cellular uptake, and ultimately a greater therapeutic impact for patients suffering from neurological disorders.
