Emerging Novel RNA-Targeted Drug Modalities Open the Door for Novel Treatments

BARCELONA, Spain I November 13, 2022 I La Merie Publishing released today its newest report entitled “RNA-Targeted Novel Drug Modalities Based on RNA Editing, Epitranscriptomics, Direct RNA Targeting, Splicing Modulation, Translation Regulation, lncRNA & regRNA Targeting & More: a landscape analysis of technologies, targets, business and financing from an industry perspective”. This report provides a landscape description and analysis of discovery and development of novel drug modalities targeting RNA from an industry perspective as of November 2019.

Since the characterization of mRNA in 1961, the understanding of the roles of RNA molecules has significantly grown. Beyond serving as a link between DNA and proteins, RNA molecules play direct effector roles by binding to various ligands, including proteins, DNA, other RNAs, and metabolites. Through these interactions, RNAs mediate cellular processes such as the regulation of gene transcription and the enhancement or inhibition of protein activity. As a result, the misregulation of RNA molecules is often associated with disease phenotypes, and RNA molecules have been increasingly recognized as potential targets for drug development efforts, which in the past had focused primarily on proteins. Both small molecule-based and oligonucleotide-based therapies are being pursued in efforts to target RNA.

With the discovery of functional RNA molecules, it became clear that drug discovery, which had previously focused solely on proteins, should also be applied to RNA. This approach carries great potential for at least three reasons. First, targeting a misregulated disease-related RNA might be more amenable to drug development especially if targeting proteins involved in the same pathway may lead to undesirable side effects. Second, for some proteins currently considered difficult to drug, it might only be possible to modulate their effect by targeting the corresponding mRNA. Finally, several functional RNAs have been found to play essential roles in the proliferation of viral, fungal, and bacterial pathogenic organisms. Targeting RNA thus opens the door to novel treatments for both infectious and noninfectious diseases.

La Merie Publishing’s new report describes and analyzes the discovery and development of novel drug modalities targeting RNA from an industry perspective. RNA-Targeted Drug Modalities covered by this report are:

  • mRNA Translation Regulation
  • RNA Splicing Modification
  • RNA Trans-Splicing
  • RNA Editing
  • Epitranscriptomics
  • lncRNA & regRNA
  • Direct RNA Targeting

The report addresses for each approach to target RNA with novel treatment modalities:

  • Stakeholders;
  • Technologies;
  • RNA targets & clinical indications or therapeutic areas;
  • Preclinical and/or clinical experience;
  • Financing;
  • Partnering;
  • Comparative assessment.

Each approach is presented in a separate chapter based on individual company profiles, technology profiles and drug or drug candidate profiles.Technology company profiles address the following aspects:

  • General overview (founders, foundation year, technology source, location, number of employees), main business activities;
  • Funding history and financial situation
  • Technology overview;
  • Partnering;
  • Targets and pipeline.

This report is based on information retrieved for 54 technology companies and 20 pharmaceutical companies. All information is fully referenced

The report RNA-Targeted Novel Drug Modalities Based on RNA Editing, Epitranscriptomics, Direct RNA Targeting, Splicing Modulation, Translation Regulation, lncRNA & regRNA Targeting & More: a landscape analysis of technologies, targets, business and financing from an industry perspective can be acquired at La Merie Publishing’s online store: https://lamerie.com/report/rna-targeted-novel-drug-modalities-based-on-rna-editing-epitranscriptomics-direct-rna-targeting-splicing-modulation-translation-regulation-lncrna-regrna-targeting-more-a-landscape-analysis/

About La Merie Publishing

La Merie Publishing is an independent business information provider for the biotechnology and pharmaceutical industry. La Merie Publishing prepares brief and full reports. La Merie Publishing products can be purchased in the online store www.lamerie.com and at selected Resellers.

To leverage its expertise and knowledge accumulated in the in-house database, La Merie Publishing offers custom report preparation services for corporate clients, including, but not limited to, pipeline analysis reports, drug profiles and any other competitive intelligence elaboration of interest.

SOURCE: La Merie Publishing

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