RNA interference Pipeline and Clinical Trials Assessment 2023: FDA Approvals, Therapies and Key Companies involved by DelveInsight | Moderna Therapeutics, GenSight Biologics, Pfizer, bluebird bio

RNA interference Pipeline and Clinical Trials Assessment 2023: FDA Approvals, Therapies and Key Companies involved by DelveInsight | Moderna Therapeutics, GenSight Biologics, Pfizer, bluebird bio
(Las Vegas, Nevada, United States) As per DelveInsight’s assessment, globally, RNA interference pipeline constitutes 20+ key companies continuously working towards developing 90+ RNA interference treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight.

The RNA interference Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.


RNA interference Pipeline Insight, 2023 report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the RNA interference Market.


Some of the key takeaways from the RNA interference Pipeline Report:

  • Companies across the globe are diligently working toward developing novel RNA interference treatment therapies with a considerable amount of success over the years. 
  • RNA interference companies working in the treatment market are Olix Pharmaceuticals, Phio Pharmaceutic als, Siranomics, Arbutus Biopharma, Bio-Path Holdings, Vir Biotechnology, Alnylam Pharmaceuticals, Sylentis, and others, are developing therapies for the RNA interference treatment 
  • Emerging RNA interference therapies in the different phases of clinical trials are- OLX 703A, PH 894, STP707, AB-729, BP1001, VIR-2218, Cemdisiran, SYL1001, Lumasiran, and others are expected to have a significant impact on the RNA interference market in the coming years.   
  • In October 2022, Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, has announced that the U.S. Food and Drug Administration (FDA) has approved an expansion to the label for OXLUMO® (lumasiran), an RNAi therapeutic administered via subcutaneous injection. This expansion now includes an indication for treating primary hyperoxaluria type 1 (PH1) to reduce urinary oxalate (UOx) and plasma oxalate (POx) levels in both pediatric and adult patients. The FDA’s decision is supported by positive efficacy and safety findings from the ILLUMINATE-C Phase 3 study, which involved patients with severe renal impairment, including those receiving hemodialysis.
  • In October 2022, Regeneron Pharmaceuticals has commenced a Phase III clinical trial that is randomized and open-label, comparing pozelimab and cemdisiran in combination. This study aims to evaluate the safety and effectiveness of this therapy combination in patients diagnosed with paroxysmal nocturnal hemoglobinuria. These patients are currently undergoing treatment with eculizumab or ravulizumab. The trial is currently in an active phase and is not recruiting new participants. It is anticipated to conclude by June 2025.


RNA interference Overview

One of the mechanisms known as RNA silencing pathways, which use short RNAs as guides for sequence-specific silencing, is RNA interference (RNAi). Long double-stranded RNA (dsRNA)-induced sequence-specific mRNA degradation is known as RNAi, and it was first identified in C. elegans. In almost all human cells, the RNA interference (RNAi) pathway controls mRNA stability and translation.


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Emerging RNA interference Drugs Under Different Phases of Clinical Development Include:

  • OLX 703A: Olix Pharmaceuticals
  • PH 894: Phio Pharmaceutic als
  • STP707: Siranomics
  • AB-729: Arbutus Biopharma
  • BP1001: Bio-Path Holdings
  • VIR-2218: Vir Biotechnology
  • Cemdisiran: Alnylam Pharmaceuticals
  • SYL1001: Sylentis
  • Lumasiran: Alnylam Pharmaceuticals


RNA interference Route of Administration

RNA interference pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs, such as 

  • Intra-articular
  • Intraocular
  • Intrathecal
  • Intravenous
  • Ophthalmic
  • Oral
  • Parenteral
  • Subcutaneous
  • Topical
  • Transdermal


RNA interference Molecule Type

RNA interference Products have been categorized under various Molecule types, such as

  • Oligonucleotide
  • Peptide
  • Small molecule


RNA interference Pipeline Therapeutics Assessment

  • RNA interference Assessment by Product Type
  • RNA interference By Stage and Product Type
  • RNA interference Assessment by Route of Administration
  • RNA interference By Stage and Route of Administration
  • RNA interference Assessment by Molecule Type
  • RNA interference by Stage and Molecule Type


DelveInsight’s RNA interference Report covers around 90+ products under different phases of clinical development like

  • Late-stage products (Phase III)
  • Mid-stage products (Phase II)
  • Early-stage product (Phase I)
  • Pre-clinical and Discovery stage candidates
  • Discontinued & Inactive candidates
  • Route of Administration


Further RNA interference product details are provided in the report. Download the RNA interference pipeline report to learn more about the emerging RNA interference therapies


Some of the key companies in the RNA interference Therapeutics Market include:

Key companies developing therapies for RNA interference are – Moderna Therapeutics, GenSight Biologics, Pfizer, bluebird bio, CureVac, ReGenX Biosciences, TransCode Therapeutics, Bicycle Therapeutics, and others.


RNA interference Pipeline Analysis:

The RNA interference pipeline report provides insights into 

  • The report provides detailed insights about companies that are developing therapies for the treatment of RNA interference with aggregate therapies developed by each company for the same.
  • It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for RNA interference Treatment.
  • RNA interference key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
  • RNA interference Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type. 
  • Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the RNA interference market.

The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.


Download Sample PDF Report to know more about RNA interference drugs and therapies


RNA interference Pipeline Market Drivers

Increasing prevalence of genetic disorders and other chronic medical conditions, diverse and extensive pipeline therapies, rise in the number of geriatric population are some of the important factors that are fueling the RNA interference Market.


RNA interference Pipeline Market Barriers

However, RNAi silencing method suffers from high off-target effects, high cost of research and development and other factors are creating obstacles in the RNA interference Market growth.


Scope of RNA interference Pipeline Drug Insight    

  • Coverage: Global
  • Key RNA interference Companies: Olix Pharmaceuticals, Phio Pharmaceutic als, Siranomics, Arbutus Biopharma, Bio-Path Holdings, Vir Biotechnology, Alnylam Pharmaceuticals, Sylentis, and others
  • Key RNA interference Therapies: OLX 703A, PH 894, STP707, AB-729, BP1001, VIR-2218, Cemdisiran, SYL1001, Lumasiran, and others
  • RNA interference Therapeutic Assessment: RNA interference current marketed and RNA interference emerging therapies
  • RNA interference Market Dynamics: RNA interference market drivers and RNA interference market barriers 


Request for Sample PDF Report for RNA interference Pipeline Assessment and clinical trials


Table of Contents

1. RNA interference Report Introduction

2. RNA interference Executive Summary

3. RNA interference Overview

4. RNA interference- Analytical Perspective In-depth Commercial Assessment

5. RNA interference Pipeline Therapeutics

6. RNA interference Late Stage Products (Phase II/III)

7. RNA interference Mid Stage Products (Phase II)

8. RNA interference Early Stage Products (Phase I)

9. RNA interference Preclinical Stage Products

10. RNA interference Therapeutics Assessment

11. RNA interference Inactive Products

12. Company-University Collaborations (Licensing/Partnering) Analysis

13. RNA interference Key Companies

14. RNA interference Key Products

15. RNA interference Unmet Needs

16 . RNA interference Market Drivers and Barriers

17. RNA interference Future Perspectives and Conclusion

18. RNA interference Analyst Views

19. Appendix

20. About DelveInsight



About DelveInsight


DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.

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