Table of Content

Abbreviations


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)

Figures & Tables

Table 1           Overview of Small Molecule Translation Regulator Companies

Table 2           Key Features of Technologies to Discover Small Molecule mRNA Translation Regulators

Table 3           Targets of Small Molecule mRNA Translation Regulator R&D Programs

Table 4           Profiles for Selected Small Molecule mRNA Translation Regulators

Table 5           Financing of Small Molecule RNA Translation Regulator Companies by Investors and Collaboration Partners

Table 6           Comparative Assessment of Small Molecule mRNA Translation Regulator Companies

Table 7           Overview of Companies with Small Molecule RNA Splicing Modifiers

Table 8           Key Features of Technologies to Discover Small Molecule RNA Splicing Modifiers

Table 9           Targets/Indications of Small Molecule RNA Splicing Modifier R&D Programs

Table 10         Profiles for Selected Small Molecule mRNA Translation Regulators

Table 11         Financing of RNA Splicing Modifier Companies by Investors and Collaboration Partners

Table 12         Comparative Assessment of Small Molecule RNA Splicing Modifier Companies

Table 13         Skyhawk Therapeutics‘ Strategic Collaborations with Major Biopharmaceutical Companies for mRNA Splicing Modifiers Discovered by SkySTAR Technology

Table 14         Overview of Companies with Direct RNA-Targeted Small Molecules

Table 15         Key Features of Technologies to Discover Direct RNA-Targeted Small Molecules

Table 16         Targets/Indications of Direct RNA-Targeted Small Molecule R&D Programs

Table 17         Financing of Direct RNA-Targeted Small Molecule Companies by Investors and Collaboration Partners

Table 18         Comparative Assessment of Direct RNA-Targeted Small Molecule Companies

Table 19         Overview of Companies with Epitranscriptomic Small Molecule Modulators

Table 20         Key Features of Technologies to Discover Epitranscriptomic Small Molecule Modulators

Table 21         Targets/Indications of Epitranscriptomic Small Molecule Modulator R&D Programs

Table 22         Financing of Epitranscriptomic Small Molecule Modulators Companies by Investors and Collaboration Partners

Table 23         Comparative Assessment of Epitranscriptomic Small Molecule Modulator Companies

Table 24         Major Pharmaceutical Companies as Stakeholders in RNA-Targeted Small Molecule R&D

Table 25         Comparison of Four Approaches in RNA-Targeted Small Molecule R&D