Table of Content
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
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
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