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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]
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]
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 ...
Additionally, RNA therapies can be costly to develop and manufacture. Despite these challenges, the field is rapidly evolving, with numerous RNA-based drugs and vaccines in development, offering new hope for patients with previously untreatable conditions. RNA Therapies Pipeline Analysis: Drug Profile. LEQVIO: Novartis
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 ...
Based on region, North America held the major share of RNA therapeutics market in 2024 due to strong presence of key pharma companies and favorable regulatory environment for novel drug development. The key players operating in the global RNA therapeutics market include Alphavax, Arcturus Therapeutics, Atyr Pharma and Gritstone Bio among others.
Osteoporosis, including drug- and cancer-related osteoporosis, giant cell tumour of bone and hypercalcaemia of malignancies: Hypercholesterolaemia, cataract, urinary retention, hypocalcaemia, osteonecrosis of the jaw and anaphylaxis. Gemtuzumab ozogamicin: IV: CD33 antibody that induces apoptosis of the tagged cell. Acute myeloid leukaemia
Moderna, Inc. (/ m ə ˈ d ɜːr n ə / mə-DUR-nə) [4] is an American pharmaceutical and biotechnology company based in Cambridge, Massachusetts, that focuses on RNA therapeutics, primarily mRNA vaccines. These vaccines use a copy of a molecule called messenger RNA (mRNA) to carry instructions for proteins to produce an immune response.
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