RNA therapy

GENEGUT: RNA therapy for ileal Crohn's Disease

Given the clinical success of mRNA-based vaccines for COVID-19, now is the right time to further push beyond the state of the art in RNA-based therapies and focus on the urgent and unmet need for advanced therapies targeting non-communicable diseases affecting tissues other than the liver/blood. CD, as a highly prevalent disease with an unmet need for targeted treatment, is an ideal candidate for which to develop a safe, effective, targeted treatment exploring non-invasive routes of administration, such as oral delivery.

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Ribonucleic acid (RNA) is a nucleic acid present in all living cells and has a vital biological role in regulation of gene expression and protein synthesis. Unlike DNA, however, RNA is most often single-stranded. An RNA molecule has a backbone made of alternating phosphate groups and the sugar ribose, rather than the deoxyribose found in DNA. Attached to each sugar is one of four bases: adenine (A), uracil (U), cytosine (C) or guanine (G). Different types of therapeutic RNAs exist including: messenger RNA (mRNA) and short interfering RNA (siRNA).

RNA-based drugs are a new class of biologics on the path to become a major platform in drug development alongside small molecules and other biologics which grant unprecedented specificity and control over protein expression (ref). Based on the accumulated results of long-term research in the field of RNA therapy spanning several decades, therapeutic agents for various diseases are being rapidly developed. Since 2018, four short interfering RNAs (siRNA) (Onpattro, Givlaari, Inclisiran, and Oxlumo) targeting liver were approved by American and European regulatory agencies, joined in 2020 by the first two mRNA-based vaccines against SARS-CoV-2. These therapeutics tend to target diseases that cannot be treated with other conventional drug groups, and several clinical studies are underway for a variety of RNA-based therapeutics against various incurable diseases.(Young-Kook Kim, p.455).

The historical timeline of important discoveries in RNA biology and key developments in RNA therapy.
Source: Young-Kook Kim, RNA therapy: rich history, various applications and unlimited future prospects (Experimental & Molecular Medicine (2022) 54:455–465).