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

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Publisher: La Merie Publishing
Pages: 418
Format: PDF
Product Line: Full Report
Product Code: LMFR0037
Release Date: November of 2022

This report provides you with a landscape description and analysis of discovery and development of novel drug modalities targeting RNA from an industry perspective as of November 2022.

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 scope of the agreement and economic terms which allows to identify 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.


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.

On the basis of 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, followed by 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 which 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.


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.

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.

Table of Contents

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

Figures & Tables

Tables in the Text


1          Key Corporate Characteristics of Translation Regulator Companies

2          Key Features of Technologies to Discover mRNA Translation Regulators

3          Targets of mRNA Translation Regulator R&D Programs

4          Profiles for Selected mRNA Translation Regulators

5          Financing of mRNA Translation Regulator Companies

6          Partnering of RNA Translation Regulator Companies

7          Comparative Assessment of mRNA Translation Regulation Companies

8          Key Corporate Characteristics of Companies with RNA Splicing Modifiers

9          Key Features of Technologies to Discover RNA Splicing Modifiers

10        Targets/Indications of RNA Splicing Modifier R&D Programs

11        Profiles for Selected RNA Splicing Modifiers

12        Financing of mRNA Splicing Modifier Companies

13        Partnering of mRNA Splicing Modifier Companies

14        Comparative Assessment of Companies with RNA Splicing Modifiers

15        Chordia Therapeutics’ Pipeline of Small Molecules Targeting RNA Maturation Processes

16        Skyhawk Therapeutics‘ Strategic Collaborations with Major Biopharmaceutical Companies for mRNA Splicing Modifiers Discovered by SkySTAR Technology

17        Stoke Therapeutics’ Pipeline of Antisense Oligonucleotides for Protein Upregulation

18        Overview of mRNA Trans-Splicing Companies

19        Key Features of Technologies in mRNA Trans-Splicing

20        Targets of mRNA Trans-Splicing R&D Programs

21        Profiles of Selected mRNA Trans-Splicing Drug Candidates

22        Financing of RNA Trans-Splicing Companies

23        Comparative Assessment of mRNA Trans-Splicing Companies

24        Key Corporate Characteristics of Companies with RNA Editing Technology

25        Key Features of Technologies to Generate RNA Editing Constructs

26        Targets/Indications of RNA Editing R&D Programs

27        Financing of Companies with RNA Editing Technology

28        Partnering of Companies with RNA Editing Technology

29        Comparative Assessment of Companies with RNA Editing Technology

30        Shape Therapeutics’ Preclinical Pipeline of ADAR-Mediated RNA Editing Using gRNA

31        Key Corporate Characteristics of Epitranscriptomics Companies

32        Targets/Indications of Epitransciptomics R&D Programs

33        Financing of Epitranscriptomics Companies

34        Partnering of Epitranscriptomics Companies

35        Comparative Assessment of Epitranscriptomics Companies

36        Overview of lncRNA & regRNA Companies

37        Key Features of lncRNA & regRNA Technologies

38        lncRNA & regRNA Targets for Drug Discovery

39        Financing of lncRNA & regRNA Companies

40        Comparative Assessment of lncRNA & regRNA Companies

41        Key Corporate Characteristics of Companies with Direct RNA-Targeted Small Molecules

42        Key Features of Technologies to Discover Direct RNA-Targeted Small Molecules

43        Targets/Indications of Direct RNA-Targeted Small Molecule R&D Programs

44        Financing of Direct RNA-Targeted Small Molecule Companies

45        Partnering of Direct RNA-Targeted Small Molecule Companies

46        Comparative Assessment of Companies with Direct RNA-Targeted Small Molecules

47        Expansion Therapeutics’ Pipeline of Small Molecules interacting with RNA (SMiRNAs)

48        Overview of Companies with RNA-Targeted Drug Candidate Opportunities outside the Main Focus

49        Overview of Major Pharma’s Preferences for Deals with RNA-Targeted Drug Modality Companies

858 Therapeutics


Acadia Pharmaceuticals

ADARx Pharmaceuticals


Anima Biotech

Arrakis Therapeutics

Ascidian Therapeutics


Aurigene Discovery Technologies

Azor Biotech

Beam Therapeutics


Biosplice Therapeutics

Bristol Myers Squibb

CAMP4 Therapeutics

Ceptur Therapeutics

Chordia Therapeutics

Creyon Bio



eFFECTOR Therapeutics

Eli Lilly

Eloxx Pharmaceuticals


EPICS Therapeutics


Expansion Therapeutics

Flamingo Therapeutics

Haisco Pharmaceutical Co

HAYA Therapeutics

Hemavant Sciences

Ionis Pharmaceuticals




Korro Bio

Ladder Therapeutics



Mitsubishi Tanabe Pharma




Novation Pharmaceuticals


Panorama Medicine


PIC Therapeutics

ProQR Therapeutics

PTC Therapeutics

Reborna Bioscience

Remix Therapeutics

ReviR Therapeutics

Rgenta Therapeutics



Roivant Sciences


Samil Pharmaceutical Co


Saverna Therapeutics


Shape Therapeutics

Skyhawk Therapeutics

Solasia Pharma

Stoke Therapeutics

Storm Therapeutics

Syros Pharmaceuticals

Takeda Pharmaceutical Co


Transine Therapeutics

Twentyeightseven Therapeutics

Vertex Pharmaceuticals

VICO Therapeutics


Wave Life Sciences

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