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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).
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]
Ionis Pharmaceuticals, Inc. is a biotechnology company based in Carlsbad, California, that specializes in discovering and developing RNA-targeted therapeutics.The company has three commercially approved medicines: Spinraza (), Tegsedi (), and Waylivra (Volanesorsen), and has four drugs in pivotal studies: tominersen for Huntington's disease (together with Roche), tofersen for SOD1-ALS, AKCEA ...
ProQR Therapeutics NV (NASDAQ: PRQR) is a Dutch biotechnology company based in Leiden, the Netherlands, with a presence in Cambridge, Massachusetts, US. [1] The company was funded in 2012 by chief executive officer (CEO) Daniel A. de Boer. It specializes in the development of RNA therapeutics using its RNA editing platform technology called ...
Pharmacological Reviews is a quarterly peer-reviewed scientific journal publishing review articles on all aspects of pharmacology and related topics. It is published by the American Society for Pharmacology and Experimental Therapeutics. The editor-in-chief is Lynette C. Daws (The University of Texas Health Science Center at San Antonio).
Sirna Therapeutics, Inc. was a San Francisco, California based biotechnology company that explored the use of RNA interference in human disease therapy. Sirna's development pipeline included several small interfering RNA (siRNA) drugs , thought to stably silence the expression of specific disease-related genes.