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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]
Additionally, HIV-1 RNA has been targeted extensively in vitro by RNA-binding small molecules. In 2007, Miller and coworkers used dynamic combinatorial chemistry to screen a compound library against HIV-1 frameshift regulatory stem-loop RNA. They identified a hit compound that was selective for the regulatory sequence with micromolar binding ...
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 ...
Another promising approach is RNA editing, where specific mutations in mRNA can be corrected to restore normal gene function. These technologies enable highly targeted interventions, making RNA therapies particularly suitable for diseases caused by single gene mutations. While RNA therapies hold great promise, they also face several challenges.
The increasing focus on RNA-targeted therapeutics and advancements in biotechnology are fueling market growth globally. Competitive Landscape: The RNA-targeted small molecules market is highly competitive, driven by the strong presence of leading pharmaceutical and biotech companies.
Thus, RNA therapeutics that offers highly targeted and personalized treatment is gaining more attention in the precision medicine domain. Furthermore, increasing funding from both private and public organizations has resulted in high investment in R&D by key market players, which is supporting the market growth.
The collaboration will combine Ribometrix’s proprietary discovery platform for identifying and optimizing small molecule therapeutics that modulate RNA function by targeting three-dimensional ...
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]
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