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RNA therapeutics are a new class of medications based on ribonucleic acid (RNA). Research has been working on clinical use since the 1990s, with significant success in cancer therapy in the early 2010s. [1] In 2020 and 2021, mRNA vaccines have been developed globally for use in combating the coronavirus disease (COVID-19 pandemic). [2]
Recent discoveries implicating RNA in the pathogenesis of several forms of cancer and neuromuscular diseases have created a paradigm shift in drug discovery.This work combined with advances in structural characterization techniques such as NMR spectroscopy, X-ray crystallography, Cryogenic electron microscopy [1] together with computational modeling, [2] has pushed forward the realization that ...
There are many additional therapeutics that have been developed and are either in phase I or II of the clinical trials. Current RNA therapeutics in clinical trials range from a variety of target organs and diseases ranging from skin (potential treatment for disease such as keloid) to tumors (squamous cell lung cancer).
DelveInsight’s 'RNA Therapies Competitive Landscape – 2024' report provides comprehensive global coverage of available, marketed, and pipeline RNA therapies in various stages of clinical development, major pharmaceutical companies working to advance the pipeline space and future growth potential of the RNA therapies competitive domain.
RNA therapeutics provides treatments for hereditary disorders, cancer, and infectious diseases. RNAi therapeutics is considered as one of the most promising areas of modern drug discovery. This is due to its ability to treat previously undruggable disease targets through selective silencing of gene expressions.
Antisense therapy is a form of treatment that uses antisense oligonucleotides (ASOs) to target messenger RNA (mRNA). ASOs are capable of altering mRNA expression through a variety of mechanisms, including ribonuclease H mediated decay of the pre-mRNA, direct steric blockage, and exon content modulation through splicing site binding on pre-mRNA. [1]
PTX-COVID19-B is a messenger RNA (mRNA)-based COVID-19 vaccine, a vaccine for the prevention of the COVID-19 disease caused by an infection of the SARS-CoV-2 coronavirus, created by Providence Therapeutics—a private Canadian drug company co-founded by Calgary, Alberta-based businessman Brad T. Sorenson and San Francisco–based Eric Marcusson ...
Arrowhead’s products in development act through RNA interference (RNAi) mechanisms of action. [ 2 ] [ 3 ] The company focuses on treatments for hepatitis B , liver disease associated with alpha 1-antitrypsin deficiency and cardiovascular disease .