Recently, a new class of drugs composed of short RNA and DNA has been approved. These drugs, called antisense and siRNA, bind to disease-associated aberrant RNA in the body and modulate the translation of disease-associated proteins. Based on the genetic sequence of a specific RNA, a complementary short RNA and DNA can be created to hybridize to the targeted RNA.
While this seems simple, lessons have been learned over four decades on providing drug-like properties to these short pieces of RNA and DNA. The important factors of success lie in the nucleotide sequence and the chemistry used to imbue the sequence with drug-like properties. While the antisense sequence is complementary to the RNA, challenges arise when the sequence also acts as a pathogen-associated molecular pattern and triggers unintended immune responses. Chemical Engineering of DNA and RNA has been shown to modulate immune responses.
Once an appropriate sequence is selected, the chemistry that provides the selected RNA drug-like properties must be applied. For example, gapmer chemistry, which I developed in the early 90s, is now widely used in antisense drug candidates that have been approved.
ARNAY Sciences is fine-tuning sequences and chemistry to create potent and safe drugs. With three decades of insights, ARNAY has designed novel chemical structures that significantly increase the potency and specificity of RNA therapeutic candidates. These chemical structures have broad applicability in developing RNA therapeutics and immunotherapy.
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