New York, USA, June 12, 2023 (GLOBE NEWSWIRE) -- Propulsion of RNA Interference Therapy Clinical Trial Pipeline as Novel and Extensive 90+ Therapies Likely to Enter in the Domain | DelveInsight

The RNA interference therapy pipeline and clinical trial analysis report deliver important insights on ongoing research, clinical strategies, upcoming therapeutics, and commercial analysis.

DelveInsight’s 'RNA Interference Therapy Competitive Landscape – 2023' report provides comprehensive global coverage of available, marketed, and pipeline RNA interference therapy in various stages of clinical development, major pharmaceutical companies working to advance the pipeline space, company assessment, comparative assessment, and future growth potential of the RNA interference therapy competitive domain.

Key Takeaways from the RNA Interference Therapy Competitive Landscape Report

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RNA Interference Therapy Overview

RNA interference (RNAi) is one of several RNA silencing processes that use short RNAs as guides for sequence-specific silencing. In C. elegans, RNAi was found and characterized as sequence-specific mRNA degradation triggered by long double-stranded RNA (dsRNA). In nearly all human cells, the RNA interference (RNAi) pathway affects mRNA stability and translation. The development of new medications is a never-ending effort to conduct successful, safe clinical trials while maintaining clinical efficacy. Until the development of monoclonal antibody treatment, small-molecule medicines were thought to be the cure-all for all ailments. Although monoclonal antibody therapy has been successful in treating many diseases, it still faces challenges with tissue penetration, production, and purification. When current pharmacological technology fails, RNA interference (RNAi) treatments provide an alternative treatment option. Furthermore, therapies for viral disorders are soon rendered obsolete due to mutational escape. Because the development of RNAi methods fulfills both demands, it could be the next breakthrough to revolutionize the drug market.

RNA interference is a highly conserved biological process that mounts an inhibitory molecular response to pathogenic viral dsRNA. Many studies have indicated that RNAi can be utilized therapeutically in vitro and in vivo to target many respiratory viruses, including SARS-CoV and possibly SARS-CoV-2, in the treatment and prevention of infection. Before such medicines become commonplace, further study at both the basic and clinical levels is required.


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RNA Interference Therapy Pipeline Analysis: Drug Profile

Cemdisiran: Alnylam Pharmaceuticals, Inc

Cemdisiran is an investigational RNAi therapy in development for the treatment of complement-mediated disorders that target the C5 component of the complement pathway. The complement system is an important part of immunity as a protective mechanism for host defense, but its dysregulation causes life-threatening complications in a variety of human diseases such as PNH, atypical hemolytic-uremic syndrome (aHUS), myasthenia gravis, neuromyelitis optica, and membranous nephropathy, among others. Regeneron Pharmaceuticals and Alnylam Pharmaceuticals announced a collaboration in April 2018 to discover, develop, and commercialize new RNA interference (RNAi) therapeutics for a wide range of diseases by addressing disease targets expressed in the eye and central nervous system (CNS), as well as a limited number of targets expressed in the liver. Regeneron, an Alnylam partner, has begun Phase III investigations of cemdisiran and pozelimab in myasthenia gravis and paroxysmal nocturnal hemoglobinuria.

ARO-APOC3: Arrowhead Pharmaceuticals, Inc.

ARO-APOC3 is a subcutaneously delivered RNAi therapy that inhibits the production of Apolipoprotein C-III (Apoc-III), a component of triglyceride-rich lipoproteins (TRLs) such as VLDL and chylomicrons, as well as a critical regulator of triglyceride metabolism. The company believes that decreasing Apoc-III hepatic production will result in decreased VLDL synthesis and assembly, improved TRL breakdown, and improved clearance of VLDL and chylomicron remnants. It is now being researched as a possible treatment for severe hypertriglyceridemia (SHTG), familial chylomicronemia syndrome (FCS), and mixed dyslipidemia. Arrowhead’s RNA interference therapy is currently in Phase III trials for Familial Chylomicronemia and Phase II trials for mixed Dyslipidemia and SHTG. In addition, Arrowhead has completed the Phase I trial for Hypertriglyceridemia and FCS. Arrowhead Pharmaceuticals got orphan drug designation from the FDA in June 2019 for ARO-APOC3 in the treatment of FCS.

A snapshot of the RNA Interference Therapy Pipeline Drugs mentioned in the report:

RNA Interference Therapy CompanyPhase MoA
CemdisiranAlnylam Pharmaceuticals, IncPhase IIIMyasthenia gravis; Paroxysmal nocturnal hemoglobinuria
ARO-APOC3Arrowhead Pharmaceuticals, Inc.Phase IIIHyperlipoproteinaemia type I
VIR-2218Vir BiotechnologyPhase IIHepatitis B; Hepatitis D
AB-729Akamis BioPhase IIHepatitis B; Hepatitis D
LY3819469Eli Lilly and CompanyPhase IIHypolipoproteinaemia
SYL 1801SylentisPhase IIAge-related macular degeneration
OLX 101AHugelPhase IIHypertrophic scars
AB 729Arbutus BiopharmaPhase IIHepatitis B; Hepatitis D
SLN124Intradigm CorporationPhase I/IIPolycythaemia vera
JNJ-75220795Janssen PharmaceuticalsPhase IFatty liver; Non-alcoholic steatohepatitis
STP707SirnaomicsPhase IHepatic fibrosis; Liver cancer; Non-small cell lung cancer; Primary sclerosing cholangitis; Solid tumours; Squamous cell cancer

Learn more about the emerging RNA interference therapy @ RNA Interference Therapy Clinical Trials

Scope of the RNA Interference Therapy Competitive Landscape Report 

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Table of Contents

1.RNA Interference Therapy Pipeline Report Introduction
2.RNA Interference Therapy Pipeline Report Executive Summary
3.RNA Interference Therapy Pipeline: Overview
4.RNA Interference Therapy Marketed Drugs
4.1.DELYTACT: Daiichi Sankyo
5.RNA Interference Therapy Clinical Trial Therapeutics
6.RNA Interference Therapy Pipeline: Late-Stage Products (Pre-registration)
7.RNA Interference Therapy Pipeline: Late-Stage Products (Phase III)
7.1. Cemdisiran: Alnylam Pharmaceuticals
8.RNA Interference Therapy Pipeline: Mid-Stage Products (Phase II)
8.1.VIR-2218: Vir Biotechnology
9.RNA Interference Therapy Pipeline: Early-Stage Products (Phase I)
10.RNA Interference Therapy Pipeline Therapeutics Assessment
11.Inactive Products in the RNA Interference Therapy Pipeline
12.Company-University Collaborations (Licensing/Partnering) Analysis
13.Unmet Needs
14.Market Drivers and Barriers
15.Appendix

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