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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 the discovery and development of novel drug modalities targeting RNA from an industry perspective as of November 2019.

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 in the field, i.e. technology companies, pharmaceutical partners, and investors;
• Technologies of RNA target discovery and discovery of drugs targeting RNA;
• RNA targets selected for drug discovery and the associated clinical indications or therapeutic areas;
• Preclinical and/or clinical experience with RNA-targeted novel drug modalities;
• Financing history and financial situation of RNA technology companies;
• Partnering deals (discovery and/or development collaborations, licensing, acquisitions);
• Comparative assessment of technology companies based on technology validation by data, partnering, and investors.

Furthermore, the report evaluates the partnering deals of pharmaceutical companies with RNA-targeted drug discovery companies regarding the scope of the agreement and economic terms, which allows to the identification of preferences of major pharmaceutical companies for RNA-targeted novel drug modalities.

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.

Methodology:

This report is based on information retrieved for 54 technology companies and 20 pharmaceutical companies. All information is fully referenced, either with scientific references (conference abstracts, Posters, presentations, full paper) or hyperlinks leading to the source of corporate information, such as press releases, corporate presentations, annual reports, SEC disclosures and homepage content.

Details about R&D strategy, collaboration and licensing agreements, acquisitions, financing rounds, and sources are described in the company profiles.

Based on the company, technology, and drug candidate profiles, each chapter analyzes the respective RNA targeting approach in a structured manner starting with an overview of corporate key characteristics, then discussing key features of technologies, describing the targets selected for RNA-targeted drug discovery. Furthermore, a complete history of financing rounds with a list of investors is provided. Partnering deals are described regarding the scope of the agreement and the economic terms. Eventually, a comparative assessment is performed for the technology players in each RNA targeting approach.

The report includes Tables that summarize specific information to allow comprehensive comparisons. Illustrations are used to explain the mechanism of action of the various RNA-targeted drug technologies, pharmacologic effects, and molecular structures.

Perspective

Over the past several years, the pharmaceutical industry has increased its efforts to identify novel drug targets outside of the usual G protein-coupled receptors, ion channels, and enzymes of multiple families, including kinases, to provide a greater variety of therapeutic options for patient populations across several disease states. In this context, recent attention has turned to look for regulators of RNA function, including small molecules and biologics.

Previously considered "difficult to the drug" on a structural and selectivity basis, considerable efforts in academia and industry are now focused on targeting RNA as a therapeutic modality. These efforts are driven based on both the biological significance of RNAs, as well as a growing number of studies implicating aberrant RNA biology in many human diseases. RNAs mediate cellular processes such as the regulation of gene transcription, splicing, and the enhancement or inhibition of protein activity. Not surprisingly, based on the myriad important functions of cellular RNAs, their dysregulation is often associated with disease phenotypes and RNA molecules are increasingly recognized as potential targets for drug development efforts.

Small-molecule- and oligonucleotide-based therapies are being pursued toward the goal of targeting RNA biology. This broad range of approaches, which also includes the targeting of RNAs and RNA-binding proteins and RNA-modifying enzymes, is in line with the known complexity of RNA biology and regulation. Although it is now recognized that many RNAs can adopt complex tertiary structures akin to proteins, classically, RNA has been targeted in a sequence-based manner rather than a structure-based manner. Antisense oligonucleotides (ASOs) remain a powerful class of biologics targeting RNA based on the sequence.

What will you find in the report?

• Profiles of pure-play and diversified RNA-targeted drug technology companies;
• Description of Major Pharma's role in the field (partnering, acquisition and investing);
• Comprehensive description and analysis of emerging RNA target discovery and drugging technologies;
• RNA drugging technology selection and preferences of major pharma;
• Pharmacologic profiles of RNA-targeted novel drugs and drug candidates;
• Target selection, pipeline analysis and competition of drug candidates;
• Description and analysis of financing rounds (capital raised, investors);
• Economic terms of collaboration and licensing deals;
• Sources of financing.

Who will benefit from the report?

• Venture capital, private equity and investment managers;
• Managers of Big Pharma venture capital firms;
• Financial analysts;
• Business development and licensing (BDL) specialists;
• CEO, COO and managing directors;
• Corporate strategy analysts and managers;
• Chief Technology Officer;
• R&D Portfolio, Technology and Strategy Management;
• Clinical and preclinical development specialists.
• RNA target and drug discovery scientists.