Table of Contents

 

1          Executive Summary

 

2          Introduction & Overview

 

3          mRNA Translation Regulation by Small Molecules

 

3.1       Overview

3.1.1    Companies

3.1.2    Technologies and Targets

3.1.3    Preclinical and Clinical Experience

3.1.4    Partnering and Financing

3.1.5    Comparative Assessment

3.2       Company Profiles

3.2.1    Anima Biotech

3.2.2    eFFECTOR Therapeutics

3.2.3    Eloxx Pharmaceuticals

3.3.      Selected Technology Profile

3.3.1    Translation Control Therapeutics Platform

3.4       Drug and Drug Candidate Profiles

3.4.1    BAY 1143269

3.4.2    eFT226

3.4.3    eIF4E Inhibitors

3.4.4    ELX-02

3.4.5    Tomivosertib

3.4.6    Translarna

 

4          RNA Splicing Modification by Small Molecules

 

4.1       Overview

4.1.1    Companies

4.1.2    Technologies and Targets

4.1.3    Preclinical and Clinical Experience

4.1.4    Partnering and Financing

4.1.5    Comparative Assessment

4.2       Company Profiles

4.2.1    H3 Biomedicine

4.2.2    Panorama Medicine

4.2.3    PTC Therapeutics

4.2.4    Skyhawk Therapeutics

4.3       Selected Technology Profile

4.3.1    RNA Splicing Platform

4.4       Drug Candidate Profiles

4.4.1    Branaplam

4.4.2    E7107

4.4.3    H3B-8800

4.4.4    PTC258

4.4.5    Risdiplam

 

5          Direct RNA Targeting by Small Molecules

 

5.1       Overview

5.1.1    Companies

5.1.2    Technologies

5.1.3    Targets and Indications

5.1.4    Partnering and Financing

5.1.5    Comparative Assessment

5.2       Companies

5.2.1    Arrakis Therapeutics

5.2.2    Expansion Therapeutics

5.2.3    Novation Pharmaceuticals

5.2.4    Nymirum

5.2.5    Ribometrix

5.2.6    Saverna Therapeutics

5.2.7    Target RNA

 

6          Indirect RNA-Targeted (Epitranscriptomic) Small Molecules

 

6.1       Overview

6.1.1    Companies

6.1.2    Technologies

6.1.3    Targets and Indications

6.1.4    Partnering and Financing

6.1.5    Comparative Assessment

6.2       Company Profiles

6.2.1    AC Immune

6.2.2    Accent Therapeutics

6.2.3    EPICS Therapeutics

6.2.4    Gotham Therapeutics

6.2.5    ImStar Therapeutics

6.2.6    STORM Therapeutics

6.2.7    Twentyeight-Seven Therapeutics

 

7          Major Pharmaceutical Companies as Stakeholders in RNA-Targeted Small Molecule R&D

 

7.1       Overview

7.2       Companies

7.2.1    Biogen

7.2.2    Boehringer Ingelheim

7.2.3    Bristol-Myers Squibb

7.2.4    Celgene

7.2.5    Eli Lilly

7.2.6    Merck

7.2.7    Novartis

7.2.8    Pfizer

7.2.9    Roche

7.2.10  Takeda Pharmaceutical Co

7.2.11  UCB

 

8          Outlook and Perspectives

 

9          References

 

10        Addendda

 

Addendum 1: Small Molecule mRNA Translation Regulators

Addendum 2: Small Molecule RNA Splicing Modifiers

Addendum 3: Direct RNA-Targeted Small Molecules

Addendum 4: Indirect RNA-Targeted Small Molecules (Epitranscriptomics)