2022年7月23日 · In this review, the mechanisms of action, challenges, solutions, and clinical application of RNA-based therapeutics have been comprehensively summarized.

The main types of RNA therapeutics are those based on messenger RNA (mRNA), antisense RNA (asRNA), RNA interference (RNAi), and RNA aptamers. Of the four types, mRNA-based therapy is the only type which is based on triggering synthesis of proteins within cells, making it particularly useful in vaccine development. [ 3 ]

In this review we discuss general concepts of different classes of RNA-based therapeutics, including antisense oligonucleotides, aptamers, small interfering RNAs, microRNAs, and messenger RNA.

This review provides an overview of significant developments in RNA-based therapeutics. The classification of RNA-based therapies and their modes of action have been outlined. This review also covers critical challenges in applying these RNA therapies and possible solutions.

2022年7月23日 · The growing understanding of RNA functions and their crucial roles in diseases promotes the application of various RNAs to selectively function on hitherto "undruggable" proteins, transcripts and genes, thus potentially broadening the therapeutic targets.

2022年4月19日 · RNA therapy is a term used to describe the use of RNA-based molecules to modulate biological pathways to cure a specific condition. In general, the RNA sequence is...

RNA therapy refers to the treatment or prevention of diseases using RNA-based molecules. The recent advent of a series of effective messenger RNA-based vaccines in response to the COVID-19 pandemic has reignited research interest in RNA therapy.

In this review, the general concepts of five classes of RNA-based therapeutics, including RNA interference-based therapies, antisense oligonucleotides, small activating RNA therapies, circular RNA therapies, and messenger RNA-based therapeutics, will be discussed.

2023年9月20日 · In this review, we present an overview of different RNA‐based strategies to generate novel therapeutics, including antisense and RNAi‐based mechanisms, mRNA‐based approaches, and CRISPR‐Cas‐mediated genome editing.

“RNA therapeutics” refers to a disease treatment or drug that utilizes RNA as a component. In this context, RNA may be the direct target of a small-molecule drug or RNA itself may be the drug, designed to bind to a protein, or to mimic or target another RNA.

The SRT is a global network to support translational research and development of RNA therapeutics; establish standards for RNA manufacturing; create guidelines for clinical trials; facilitate public-private regulatory partnerships; to promote accessibility of RNA therapeutics for all patients; to offer public and professional education; to ...

What are RNA-based therapeutics? RNA molecules, deliver genetic instructions from DNA to protein-making machinery. As well as carrying these instructions for making proteins, RNAs help to turn genes on and off, assist chemical reactions, slice and dice other RNAs, and even build proteins — by transporting amino acids and linking them together.

2022年7月6日 · Here, we provide an overview of the current clinically relevant RNA therapeutics, mechanistic basis of their function, and strategies to improve their clinical use. We discuss the 17 approved RNA therapeutics and perform an in-depth analysis of the 222 ongoing clinical trials, with an emphasis on their respective mechanisms and disease areas.

2022年9月8日 · In this review, the general concepts of five classes of RNA-based therapeutics, including RNA interference-based therapies, antisense oligonucleotides, small activating RNA therapies, circular RNA therapies, and messenger RNA-based therapeutics, will be discussed.

2024年10月29日 · What Are RNA Therapeutics? RNA therapeutics leverage the biological processes of RNA to treat diseases by influencing gene expression or providing a blueprint for protein production. These therapies encompass several categories, including: mRNA (messenger RNA): Used to create proteins that the body may be lacking due to genetic mutations.

2023年5月22日 · RNA-based therapeutics hold immense potential for treating a wide range of diseases, including genetic disorders, cancer, and infectious diseases, bringing us closer to a new era of precision medicine and improved patient outcomes.

Current advances in RNA-based therapies have substantial promise in treating and preventing many human diseases and disorders through fixing the pathology instead of merely treating the symptomology similarly to traditional therapeutics.

2024年12月12日 · Alnylam is a pioneer in the science of RNAi. The company's scientists discovered five of the six RNAi therapeutics that have been approved by the Food and Drug Administration.

4 天之前 · BOSTON, Feb. 6, 2025 /PRNewswire/ -- TransCode Therapeutics, Inc. (NASDAQ: RNAZ), the RNA oncology company committed to more effectively treating cancer using RNA therapeutics, today announced that Cohort 3 of its Phase 1 clinical trial enrolled three patients and all have been dosed with TTX-MC138, its lead candidate. The Safety Review ...

6 天之前 · In her view, RNA-editing research is at an exciting juncture, with therapeutics development moving rapidly as companies and academic labs explore RNA-editing approaches both for rare genetic ...

4 天之前 · The company is committed to defeating cancer through the intelligent design and effective delivery of RNA therapeutics based on its proprietary TTX nanoparticle platform. The company's lead ...

2021年9月22日 · RNA therapeutics involve the use of coding RNA such as mRNA as well as non-coding RNAs such as small interfering RNAs (siRNA), antisense oligonucleotides (ASO) to target mRNA, aptamers, ribozymes, and clustered regularly interspaced short palindromic repeats-CRISPR-associated (CRISPR/Cas) endonuclease to target proteins and DNA.

RNA therapeutics involve the use of coding RNA such as mRNA as well as non-coding RNAs such as small interfering RNAs (siRNA), antisense oligonucleotides (ASO) to target mRNA, aptamers, ribozymes, and clustered regularly interspaced short ...

4 天之前 · The company is committed to defeating cancer through the intelligent design and effective delivery of RNA therapeutics based on its proprietary TTX nanoparticle platform. The company's lead therapeutic candidate, TTX-MC138, is focused on treating metastatic tumors which overexpress microRNA-10b, a unique, well-documented biomarker of metastasis.

2025年1月27日 · CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jan. 27, 2025-- Sarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, today announced positive topline results from Part 2 of EMBARK (Study SRP-9001-301), a global, randomized, double-blind, placebo-controlled, Phase 3 clinical study of ELEVIDYS …

5 天之前 · José Castillo, CEO of Quantoom Biosciences, added: “This collaboration showcases the transformative power of combining complementary expertise across borders. Together, we are not only advancing RNA technologies but also addressing global health inequities by making life-saving vaccines and therapeutics accessible to underserved populations.”

Nanomedicine is simply the application of nanotechnologies in a healthcare setting and the majority of benefits that have already been seen involve the use of nanoparticles to improve the behaviour of drug substances.