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RNA-Targeted Novel Drug Modalities Market Research Report 2022: RNA Editing, Epitranscriptomics, Direct RNA Targeting, Splicing Modulation, Translation Regulation, lncRNA & regRNA Targeting -

PRESS RELEASE: Paid content from Business Wire
Press release content from Business Wire. The AP news staff was not involved in its creation.
January 5, 2023 GMT

DUBLIN--(BUSINESS WIRE)--Jan 5, 2023--

The “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” report has been added to’s offering.

This report provides you with a landscape description and analysis of discovery and development of novel drug modalities targeting RNA from an industry perspective.

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

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, in order to provide a greater variety of therapeutic options for patient populations across a number of disease states. In this context, recent attention has turned to looking for regulators of RNA function, including small molecules and biologics.


Previously considered “difficult to drug” on a structural and selectivity basis, considerable efforts in academia and industry are now being 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 structure-based manner. Antisense oligonucleotides (ASOs) remain a powerful class of biologics for targeting RNA based on sequence.

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


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

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


Key Topics Covered:

1 Executive Summary

2 Overview & Methodology

3 mRNA Translation Regulation

3.1 Overview

3.1.1 Companies

3.1.2 Technologies

3.1.3 Targets

3.1.4 Preclinical and Clinical Experience

3.1.5 Financing

3.1.6 Partnering

3.1.7 Comparative Assessment

3.2 Company Profiles

3.2.1 Anima Biotech

3.2.2 Ceptur Therapeutics

3.2.3 eFFECTOR Therapeutics

3.2.4 Eloxx Pharmaceuticals

3.2.5 PIC Therapeutics

3.2.6 Transine Therapeutics

3.3. Selected Technology Profile

3.3.1 mRNA Lightning Technology

3.3.2 SINEUP Technology

3.3.3 STRI (Selective Translation Regulation Inhibitor) Platform

3.3.4 TURBO-ZM Technology

3.4 Drug and Drug Candidate Profiles

3.4.1 eIF4E STRI

3.4.2 ELX-02

3.4.3 Tomivosertib

3.4.4 Translarna

3.4.5 ZKN-013

3.4.6 Zotatifin

4 RNA Splicing Modification

4.1 Overview

4.1.1 Companies

4.1.2 Technologies

4.1.3 Targets

4.1.4 Preclinical and Clinical Experience

4.1.5 Financing

4.1.6 Partnering

4.1.7 Comparative Assessment

4.2 Company Profiles

4.2.1 Chordia Therapeutics

4.2.2 Envisagenics

4.2.3 Hemavant Sciences

4.2.4 Panorama Medicine

4.2.5 PTC Therapeutics

4.2.6 Reborna Biosciences

4.2.7 Rgenta Therapeutics

4.2.8 Skyhawk Therapeutics

4.2.9 Stoke Therapeutics

4.3 Selected Technology Profile

4.3.1 PAN-ACEA Platform

4.3.2 SkySTAR Technology

4.3.3 SpliceCore Technology

4.3.4 TANGO Technology

4.4 Drug and Drug Candidate Profiles

4.4.1 Branaplam

4.4.2 CTX-712

4.4.3 Evrysdi

4.4.4 PTC518

4.4.5 RVT-2001

4.4.6 STK-001

4.4.7 TEC-1

4.4.8 WVE-N531

5 RNA Trans-Splicing

5.1 Overview

5.1.1 Companies

5.1.2 Technologies

5.1.3 Targets

5.1.4 Preclinical and Clinical Experience

5.1.5 Financing

5.1.6 Comparative Assessment

5.2 Company Profiles

5.2.1 Ascidian Therapeutics

5.2.2 Rznomics

5.2.3 ViGeneron

5.3 Technology Profiles

5.3.1 Pre-mRNA Exon Editing Technology

5.3.2 Trans-Splicing Ribozyme Technology

5.4 Drug Candidate Profiles

5.4.1 ABCA4 Retinopathy

5.4.2 RZ001

5.4.3 RZ004

6 RNA Editing

6.1 Overview

6.1.1 Companies

6.1.2 Technologies

6.1.3 Targets & Preclinical Experience

6.1.4 Financing

6.1.5 Partnering

6.1.6 Comparative Assessment

6.2 Company Profiles

6.2.1 ADARx Pharmaceuticals

6.2.2 Beam Therapeutics

6.2.3 Creyon Bio

6.2.4 EdiGene

6.2.5 EditForce

6.2.6 KorroBio

6.2.7 Locanabio

6.2.8 ProQR Therapeutics

6.2.9 Shape Therapeutics

6.2.10 VICO Therapeutics

6.2.11 Wave Life Sciences

6.3 Technology Profiles

6.3.1 AIMer Technology

6.3.2 Axiomer Technology

6.3.3 CORRECTx Technology

6.3.4 LEAPER Technology

6.3.5 OPERA Technology

6.3.6 PPR Editing Platform

6.3.7 RNAfix Technology

6.4 Drug Candidate Profile

6.4.1 WVE-006

7 Epitranscriptomics

7.1 Overview

7.1.1 Companies

7.1.2 Targets

7.1.3 Financing

7.1.4 Partnering and

7.1.5 Comparative Assessment

7.2 Company Profiles

7.2.1 858 Therapeutics

7.2.2 Accent Therapeutics

7.2.3 EPICS Therapeutics

7.2.4 STORM Therapeutics

7.2.5 Twentyeight-Seven Therapeutics

7.3 Drug Candidate Profile

7.3.1 STC-15

8 lncRNA & regRNA

8.1 Overview

8.1.1 Companies

8.1.2 Technologies

8.1.3 Targets

8.1.4 Financing

8.1.5 Comparative Assessment

8.2 Company Profiles

8.2.1 CAMP4 Therapeutics

8.2.2 Flamingo Therapeutics

8.2.3 HAYA Therapeutics

8.2.4 NextRNA Therapeutics

8.2.5 Saverna Therapeutics

8.2.6 TargetRNA

8.3 Technology Profile

8.3.1 RAP Platform

8.4 Drug Candidate Profile

8.4.1 CMP-SCN

9 Direct RNA Targeting

9.1 Overview

9.1.1 Companies

9.1.2 Technologies

9.1.3 Targets and Indications

9.1.4 Financing

9.1.5 Partnering

9.1.6 Comparative Assessment

9.2 Company Profiles

9.2.1 Arrakis Therapeutics

9.2.2 Expansion Therapeutics

9.2.3 Ladder Therapeutics

9.2.4 Molecure

9.2.5 Novation Pharmaceuticals

9.2.6 Nymirum

9.2.7 Remix Therapeutics

9.2.8 ReVir Therapeutics

9.2.9 Ribometrix

9.3 Technology Profiles

9.3.1 DART Platform

9.3.2 REMaster Technology

9.3.3 rSM Platform

9.3.4 SMiRNA Technology

10 Opportunities

10.1 Overview

10.2 Company Profiles

10.2.1 AbbiVax

10.2.2 Biosplice Therapeutics

10.2.3 Kinnate

10.2.4 Syros Pharmaceuticals

10.3 Drug Candidate Profiles

10.3.1 Cirtuvivint

10.3.2 CRD-1968099

10.3.3 CTX-439

10.3.4 Lorecivivint

10.3.5 Obefazimod

11 Major Pharmaceutical Companies as Stakeholders in RNA-Targeted Novel Drug Modality R&D

11.1 Overview

11.2 Companies

11.2.1 Acadia Pharmaceuticals

11.2.2 Amgen

11.2.3 AstraZeneca

11.2.4 Biogen

11.2.5 Bristol Myers Squibb

11.2.6 Eli Lilly

11.2.7 Exelixis

11.2.8 Ipsen

11.2.9 Janssen

11.2.10 Lundbeck

11.2.11 Merck

11.2.12 Mitsubishi Tanabe Pharma

11.2.13 Novartis

11.2.14 Pfizer

11.2.15 Roche

11.2.16 Sanofi

11.2.17 Servier

11.2.18 Solasia Pharma

11.2.19 Takeda Pharmaceutical Co

11.2.20 Vertex Pharmaceuticals

12 References

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SOURCE: Research and Markets

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PUB: 01/05/2023 08:26 AM/DISC: 01/05/2023 08:25 AM