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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 ...
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]
New York, USA, Oct. 16, 2024 (GLOBE NEWSWIRE) -- 80+ Pharma Companies Unite to Shape the Future of RNA-Based Therapeutics | DelveInsight. With over 80 key companies active in the RNA therapies space, the market is projected to grow due to advancements in mRNA, siRNA, and antisense oligonucleotide-based therapies.
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).
Burlingame, Sept. 09, 2024 (GLOBE NEWSWIRE) -- CoherentMI published a report, titled, RNA Therapeutics Market is estimated to value at US$ 2.5 Million in the year 2024 and is anticipated to reach US$ 948 Million by 2031, at a CAGR of 68% during forecast period 2024-2031.
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 ...
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 .