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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).
AIM ImmunoTech Inc., formerly known as Hemispherx Biopharma Inc., is a biopharmaceutical company based in Ocala, Florida that is focused on the research and development of therapeutics to treat multiple types of cancers, various viruses and immune-deficiency disorders. [2] Founded in 1990, the company has twenty-three employees. [3]
The company develops RNA therapeutics for the treatment of rare diseases such as ornithine transcarbamylase deficiency, and respiratory diseases such as cystic fibrosis. As of 2021 [update] , vaccine medicines include a vaccine candidate for COVID-19 and an influenza vaccine in the preclinical development phase.
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.
Cancer and Genetics Research Complex [1] is an interdisciplinary research center located at the University of Florida (UF). The goal of this facility is "to harness the diverse academic talents and resources of the genetic research community at UF to improve the health and well-being of our citizenry."
Regulus Therapeutics Inc. is a clinical stage biopharmaceutical company focused on the development of first-in-class drugs that target microRNAs to treat a broad range of diseases. Regulus was established in September 2007 by Alnylam Pharmaceuticals and Isis Pharmaceuticals.