Evaluate pharmaceutical companies’ partnership agreements with RNA-targeted drug discovery companies

DUBLIN, January 6, 2023 /PRNewswire/ — The “New RNA-Targeting Drug Modalities Based on RNA Editing, Epitranscriptomics, Direct RNA Targeting, Splicing Modulation, Translation Regulation, lncRNA and regRNA Targeting, and More: An Overview Review of technologies, goals, business and finance from an Industry Perspective report has been added.” ResearchAndMarkets.com offering.

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This report provides a landscape overview and analysis of the discovery and development of new RNA-targeted drug modalities from an industrial perspective.

The report evaluates pharmaceutical companies’ partnership agreements with RNA-targeted drug discovery companies in terms of agreement scope and economic terms that help identify the preferences of major pharmaceutical companies for ways of targeting new drugs. ‘RNA.

In recent years, the pharmaceutical industry has increased its efforts to identify novel drug targets outside the usual G protein-coupled receptors, ion channels, and enzymes of multiple families, including kinases, in order to provide a greater variety of treatment options for patient populations in a number of disease states. In this context, recent attention has turned to the search for regulators of RNA function, including small molecules and biological products.

Previously considered “difficult to drug” on a structural and selective basis, substantial efforts in academia and industry are now focusing on targeting RNA as a therapeutic modality. These efforts are driven both by the biological significance of RNAs and by a growing number of studies implicating the biology of aberrant RNA in many human diseases.

RNAs mediate cellular processes such as regulation of gene transcription, splicing, and enhancement or inhibition of protein activity. Not surprisingly, based on the myriad of 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 with the goal of targeting RNA biology. This broad range of approaches, which also includes targeting of RNA 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 protein-like tertiary structures, classically, RNA has been targeted in a sequence-based rather than a structure-based way. Antisense oligonucleotides (ASOs) remain a potent class of biological products for sequence-based RNA targeting.

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:

  • General overview (founders, year of foundation, technology source, location, number of employees), main company activities

  • Financing history and financial situation

  • Technology overview

  • Partnerships

  • Goals and pipeline

The RNA-targeting drug modalities covered by this report are:

The report addresses for each approach to targeted RNA with new treatment modalities:

  • Industry stakeholders, i.e. technology companies, pharmaceutical partners, and investors

  • RNA target discovery technologies and RNA targeted drug discovery

  • Selected RNA targets for drug discovery and associated clinical indications or therapeutic areas

  • Preclinical and/or clinical experience with novel RNA-targeting drug modalities

  • Funding history and financial situation of RNA technology companies

  • Partnership agreements (collaborations for discovery and/or development, licensing, acquisitions)

  • Benchmarking of technology companies based on technology validation by data, partnerships and investors

What will you find in the report?

  • Profiles of diverse and pure-play RNA targeting pharmaceutical technology companies

  • Description of Major Pharma’s role in the industry (partnerships, acquisitions and investments)

  • Comprehensive description and analysis of emerging technologies for RNA target discovery and drug delivery

  • Selection of RNA doping technology and preferences of major pharmaceutical companies

  • Pharmacological profiles of novel RNA-targeted drugs and drug candidates

  • Target selection, pipeline analysis and drug candidate competition

  • Description and analysis of funding rounds (capital raised, investors)

  • Economic conditions of collaboration and license agreements

  • Funding sources

Who will benefit from the report?

  • Venture capital, private equity and investment managers

  • Manager of Big Pharma venture capital firms

  • Financial analysts

  • Business Development and Licensing (BDL) specialists.

  • CEO, COO and managing directors

  • Corporate strategy analysts and managers

  • Head of technology

  • R&D portfolio, technology and strategy management

  • Clinical and preclinical development specialists

  • RNA targeting and drug discovery scientists

Main topics covered:

1 Executive summary

2 Overview and methodology

3 Regulation of mRNA translation
3.1 Overview
3.1.1 Company
3.1.2 Technologies
3.1.3 Objectives
3.1.4 Preclinical and clinical experience
3.1.5 Financing
3.1.6 Partnerships
3.1.7 Benchmarking
3.2 Company Profiles
3.2.1 Biotech soul
3.2.2 Ceptur Therapeutics
3.2.3 eFFECTOR Therapeutics
3.2.4 Eloxx pharmaceutical products
3.2.5 PIC Therapeutics
3.2.6 Transin therapy
3.3. Selected technology profile
3.3.1 mRNA flash technology
3.3.2 SINEUP technology
3.3.3 Selective Translation Regulation Inhibitor (STRI) platform.
3.3.4 TURBO-ZM technology
3.4 Drugs and Drug Candidate Profiles
3.4.1 eIF4E STRI
3.4.2 ELX-02
3.4.3 Tomivosertib
3.4.4 Translation
3.4.5 ZKN-013
3.4.6 Zotatifine

4 Modification of RNA splicing
4.1 Overview
4.1.1 Company
4.1.2 Technologies
4.1.3 Objectives
4.1.4 Preclinical and clinical experience
4.1.5 Financing
4.1.6 Partnerships
4.1.7 Benchmarking
4.2 Company Profiles
4.2.1 Chordia Therapeutics
4.2.2 Envisagenics
4.2.3 Hemavant sciences
4.2.4 Medicine landscape
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 Drugs and Drug Candidate Profiles
4.4.1 Branaplam
4.4.2 CTX-712
4.4.3 Eurysdis
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 Trans-junction of RNA
5.1 Overview
5.1.1 Company
5.1.2 Technologies
5.1.3 Objectives
5.1.4 Preclinical and clinical experience
5.1.5 Financing
5.1.6 Benchmarking
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 modification
6.1 Overview
6.1.1 Company
6.1.2 Technologies
6.1.3 Objectives and preclinical experience
6.1.4 Funding
6.1.5 Partnerships
6.1.6 Benchmarking
6.2 Company Profiles
6.2.1 ADARx Pharmaceuticals
6.2.2 Beam Therapy
6.2.3 Creyon Bio
6.2.4 EdiGene
6.2.5 ModifyForce
6.2.6 CorroBio
6.2.7 Localabius
6.2.8 ProQR Therapy
6.2.9 Therapeutic form
6.2.10 Vico Therapeutics
6.2.11 Wave life sciences
6.3 Technology profiles
6.3.1 AIMer technology
6.3.2 Technology of axiomers
6.3.3 CORRECTx technology
6.3.4 LEAPER technology
6.3.5 OPERA technology
6.3.6 PPR change platform
6.3.7 RNAfix technology
6.4 Drug candidate profile
6.4.1 WVE-006

7 Epitranscriptomics
7.1 Overview
7.1.1 Company
7.1.2 Objectives
7.1.3 Financing
7.1.4 Partnerships e
7.1.5 Benchmarking
7.2 Company Profiles
7.2.1 858 Therapeutic
7.2.2 Accent Therapeutics
7.2.3 EPICS Therapeutics
7.2.4 STORM therapeutics
7.2.5 Twenty-eight-seven therapies
7.3 Drug candidate profile
7.3.1 STC-15

8 lncRNAs and regRNAs
8.1 Overview
8.1.1 Company
8.1.2 Technologies
8.1.3 Objectives
8.1.4 Funding
8.1.5 Benchmarking
8.2 Company Profiles
8.2.1 CAMP4 Therapeutics
8.2.2 Flamingo Therapeutics
8.2.3 HAYA Therapeutics
8.2.4 NextRNA Therapy
8.2.5 Therapeutic Saverna
8.2.6 Target RNA
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 Company
9.1.2 Technologies
9.1.3 Objectives and indications
9.1.4 Funding
9.1.5 Partnerships
9.1.6 Benchmarking
9.2 Company Profiles
9.2.1 Arrakis Therapeutics
9.2.2 Therapeutic Expansion
9.2.3 Ladder Therapeutics
9.2.4 Molecule
9.2.5 Novation Pharmaceutical Products
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 Pharmaceutical products Syros
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 the research and development of new RNA-targeted drugs
11.1 Overview
11.2 Company
11.2.1 Acadia Pharmaceutical Products
11.2.2 Amgen
11.2.3 AstraZeneca
11.2.4 Biogenes
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 Merk
11.2.12 Mitsubishi Tanabe Pharmaceutical
11.2.13 Novartis
11.2.14 Pfizer
11.2.15 Roche
11.2.16 Sanofi
11.2.17 Servant
11.2.18 Solasi Pharma
11.2.19 Takeda Pharmaceutical Co
11.2.20 Vertex Pharmaceuticals

12 References

For more information about this report, please visit https://www.researchandmarkets.com/r/wde808

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