RNA Interference Pipeline Therapeutics Assessment Report 2024 (Updated)

DelveInsight's, "RNA Interference Pipeline Insight 2024" report provides comprehensive insights about 20+ companies and 90+ pipeline drugs in the RNA Interference pipeline landscape. It covers the RNA Interference pipeline drug profiles, including clinical and nonclinical stage products. It also covers the RNA Interference pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

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Key Takeaways from the RNA Interference Pipeline Report
• In December 2024: University of Oxford- ORION-4 is a research study coordinated by the University of Oxford and co-sponsored by The University of Oxford and Novartis (Protocol: CTSU_MDCO-PCS-17-01 (CKJX839B12301)). The study aims to find out if a new cholesterol-lowering injection (inclisiran) safely lowers the risk of heart attacks and strokes in people who have already had one of these conditions, or who have had an operation or procedure to treat blocked arteries.
• In October 2024:- Novartis Pharmaceuticals-Two Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Inclisiran in Children (2 to Less Than 12 Years) With Homozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol. This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 2 to
• DelveInsight's RNA Interference pipeline report depicts a robust space with 20+ active players working to develop 90+ pipeline therapies for RNA Interference treatment.
• The leading companies working in the RNA Interference Market include Alnylam Pharmaceuticals, Suzhou Ribo Life Science, Arrowhead Pharmaceuticals, Dicerna Pharmaceuticals, Sylentis, Vir Biotechnology, Arbutus Biopharma, Silenseed, OliX Pharmaceuticals, Bio-Path Holdings, Janssen Pharmaceuticals, Eli Lilly and Company, Amgen, Sirnaomics, Silence Therapeutics, Phio Pharmaceuticals, Medesis Pharma, Atalanta Therapeutics, Mirimus/ Biogen, Mallinckrodt/Silence Therapeutics, and others.
• Promising RNA Interference Pipeline Therapies in the various stages of development include siG12D LODER, Inclisiran, Bevasiranib, and others.

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RNA Interference Emerging Drugs Profile
• Vutrisiran: Alnylam Pharmaceuticals
Vutrisiran is an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of ATTR amyloidosis, which encompasses both hATTR and wild-type ATTR (wtATTR) amyloidosis. It is designed to target and silence specific messenger RNA, potentially blocking the production of wild-type and variant transthyretin (TTR) protein before it is made. Quarterly, and potentially biannual, administration of vutrisiran may help to reduce deposition and facilitate the clearance of TTR amyloid deposits in tissues and potentially restore function to these tissues. Vutrisiran utilizes Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate delivery platform, designed for increased potency and high metabolic stability that may allow for infrequent subcutaneous injections.

• Cemdisiran: Alnylam Pharmaceuticals
Cemdisiran is an investigational RNAi therapeutic targeting the C5 component of the complement pathway in development for the treatment of complement-mediated diseases. The complement system plays a central role in immunity as a protective mechanism for host defense, but its dysregulation results in life-threatening complications in a broad range of human diseases including paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic-uremic syndrome (aHUS), myasthenia gravis, neuromyelitis optica, and membranous nephropathy, amongst others. Complement component C5, which is predominantly expressed in liver cells, is a genetically and clinically validated target; loss of function human mutations are associated with an attenuated immune response against certain infections and Intraveneous anti-C5 monoclonal antibody (mAb) therapy has demonstrated clinical activity and tolerability in a number of complement-mediated diseases. A subcutaneously administered RNAi therapeutic that silences C5 represents a novel approach for the potential treatment of complement-mediated diseases. Cemdisiran utilizes Alnylam's ESC-GalNAc conjugate technology, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index. The safety and efficacy of cemdisiran have not been evaluated by the U.S. Food and Drug Administration or any other health authority. Alnylam's partner Regeneron initiated Phase III studies of cemdisiran and pozelimab combination in myasthenia gravis and paroxysmal nocturnal hemoglobinuria.

• SR061: Suzhou Ribo Life Science
Designed as a siRNA, SR061 inhibits the expression of the Caspases 2 gene via RNAi mechanism by stopping retinal ganglion cell (RGC) apoptosis and secondary axonal degeneration, thereby preventing further deterioration of vision and visual field in the diseases and achieving therapeutic effect of vision protection. SR061 holds the potential of becoming the First-In-Class neuroprotective agent being developed to treat non-arteritic anterior ischemic optic neuropathy (NAION), for which there is no standard therapy at present. One Phase I/IIa clinical study and one Phase II/III international multi-center clinical study (including 34 Chinese subjects) have been completed. A phase III confirmatory clinical study has been carried out in the group of patients, and the IND application is being prepared.

• ARO-APOC3: Arrowhead Pharmaceuticals
ARO-APOC3 is a subcutaneously administered RNAi therapeutic designed to reduce the production of Apolipoprotein C-III (Apoc-III), a component of triglyceride-rich lipoproteins (TRLs) including VLDL and chylomicrons, and is a key regulator of triglyceride metabolism. The company believes that knocking down the hepatic production of Apoc-III may result in reduced VLDL synthesis and assembly, enhanced breakdown of TRLs, and better clearance of VLDL and chylomicron remnants. It is currently being developed as a potential treatment for patients with severe hypertriglyceridemia (SHTG), familial chylomicronemia syndrome (FCS), and mixed dyslipidemia. Arrowhead's investigational RNA interference (RNAi) therapeutic is currently under phase III trial for FCS and phase II for mixed dyslipidemia and SHTG. Furthermore, Arrowhead has successfully completed the phase I trial for hypertriglyceridemia and FCS.

• DCR-PHXC: Dicerna Pharmaceuticals
DCR-PHXC is the only RNAi investigational drug in development for the treatment of all types of primary hyperoxaluria (PH), and the most advanced product candidate utilizing Dicerna's GalXC™ technology. GalXC is a proprietary platform invented by Dicerna scientists to discover and develop next-generation RNAi-based therapies designed to silence disease-driving genes in the liver. Dicerna is evaluating DCR-PHXC in the PHYOX™ clinical trial program. The trial is currently under different phases.

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RNA Interference Companies
Alnylam Pharmaceuticals, Suzhou Ribo Life Science, Arrowhead Pharmaceuticals, Dicerna Pharmaceuticals, Sylentis, Vir Biotechnology, Arbutus Biopharma, Silenseed, OliX Pharmaceuticals, Bio-Path Holdings, Janssen Pharmaceuticals, Eli Lilly and Company, Amgen, Sirnaomics, Silence Therapeutics, Phio Pharmaceuticals, Medesis Pharma, Atalanta Therapeutics, Mirimus/ Biogen, Mallinckrodt/Silence Therapeutics, and others.

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 Pipeline Products have been categorized under various Molecule types such as
• Oligonucleotide
• Peptide
• Small molecule

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Scope of the RNA Interference Pipeline Report
• Coverage- Global
• RNA Interference Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• RNA Interference Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
• RNA Interference Companies- Alnylam Pharmaceuticals, Suzhou Ribo Life Science, Arrowhead Pharmaceuticals, Dicerna Pharmaceuticals, Sylentis, Vir Biotechnology, Arbutus Biopharma, Silenseed, OliX Pharmaceuticals, Bio-Path Holdings, Janssen Pharmaceuticals, Eli Lilly and Company, Amgen, Sirnaomics, Silence Therapeutics, Phio Pharmaceuticals, Medesis Pharma, Atalanta Therapeutics, Mirimus/ Biogen, Mallinckrodt/Silence Therapeutics, and others.
• RNA Interference Pipeline Therapies- siG12D LODER, Inclisiran, Bevasiranib, and others.

For a detailed overview of our latest research findings and future plans, read the full details of RNA Interference Pipeline on our website @ RNA Interference Drugs and Companies- https://www.delveinsight.com/sample-request/rna-interference-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Table of Content
1. Introduction
2. Executive Summary
3. RNA Interference: Overview
4. Pipeline Therapeutics
5. RNA Interference Therapeutic Assessment
6. RNA Interference- DelveInsight's Analytical Perspective
7. Late Stage Products (Pre-Registration)
8. Vutrisiran: Alnylam Pharmaceuticals
9. Drug profiles in the detailed report…..
10. Last Stage Products (Phase III)
11. Cemdisiran: Alnylam Pharmaceuticals
12. Drug profiles in the detailed report…..
13. Early Stage Products (Phase I)
14. Drug Name : Company Name
15. Drug profiles in the detailed report…..
16. Preclinical and Discovery Stage Products
17. Drug name: Company Name
18. Drug profiles in the detailed report…..
19. Inactive Products
20. RNA Interference Key Companies
21. RNA Interference Key Products
22. RNA Interference- Unmet Needs
23. RNA Interference Market Drivers
24. RNA Interference Barriers
25. RNA Interference Future Perspectives and Conclusion
26. RNA Interference Analyst Views
27. RNA Interference Key Companies
28. Appendix

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