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RNA Interference Pipeline Assessment, FDA Approvals, Clinical Trials, Emerging Drugs, and Therapeutics Assessment 2023 | Major Companies – Alnylam Pharmaceuticals, Suzhou Ribo Life Science, and Others

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RNA Interference Pipeline Assessment, FDA Approvals, Clinical Trials, Emerging Drugs, and Therapeutics Assessment 2023 | Major Companies – Alnylam Pharmaceuticals, Suzhou Ribo Life Science, and Others

May 15
16:12 2023
RNA Interference Pipeline Assessment, FDA Approvals, Clinical Trials, Emerging Drugs, and Therapeutics Assessment 2023 | Major Companies - Alnylam Pharmaceuticals, Suzhou Ribo Life Science, and Others

DelveInsight’s, “RNA Interference Pipeline Insight 2023” 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 RNA Interference clinical trials and nonclinical stage products. It also covers the RNA Interference therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

 

Recent Developmental Activities in the RNA Interference Treatment Landscape

 

  • In April 2022, Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced that the U.S. Food and Drug Administration (FDA) has accepted the Company’s New Drug Application (NDA) for vutrisiran, an investigational RNAi therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. The FDA has set an action date of April 14, 2022, under the Prescription Drug User Fee Act (PDUFA), and the Agency has indicated that they are not currently planning an advisory committee meeting as part of the NDA review

 

  • In November 2021, Arrowhead Pharmaceuticals Inc. announced that its collaborator, Janssen Pharmaceuticals, Inc., (Janssen) one of the Janssen Pharmaceutical Companies of Johnson & Johnson, has disclosed its collaboration with Arrowhead on investigational compound JNJ-75220795, which is in a Phase I clinical study

 

  • In November 2021, Arrowhead Pharmaceuticals announced that it entered into an exclusive license agreement with GSK under which GSK will develop and commercialize ARO-HSD.

 

Request a sample and discover the recent breakthroughs happening in the RNA Interference Pipeline landscape @ RNA Interference Pipeline Outlook

 

RNA Interference Overview

RNA interference (RNAi) is one of the pathways, collectively named RNA silencing pathways that employ small RNAs as guides for sequence-specific silencing. RNAi was discovered in C. elegans and defined as sequence-specific mRNA degradation induced by long double-stranded RNA (dsRNA). The RNA interference (RNAi) pathway regulates mRNA stability and translation in nearly all human cells.

 

Key Takeaways from the RNA Interference Pipeline Report

 

  • Over 20+ RNA interference companies are evaluating 90+ RNA interference pipeline therapies in various stages of development, and their anticipated acceptance in the RNA interference market would significantly increase market revenue.

 

  • The leading RNA interference companies include Alnylam Pharmaceuticals, Suzhou Ribo Life Science, Arrowhead Pharmaceuticals, Sylentis, Vir Biotechnology, Arbutus Biopharma, Silenseed, OliX Pharmaceuticals, Bio-Path Holdings, Janssen Pharmaceuticals, Eli Lilly and Company, Dicerna Pharmaceuticals, Amgen, Sirnaomics, Silence Therapeutics, Sylentis, Silence Therapeutics, Suzhou Ribo Life Science, Phio Pharmaceuticals, Amylon Therapeutics, Aro Biotherapeutics, DTx Pharma, BONAC Corporation, and others

 

  • Promising RNA interference pipeline therapies include SR061, ARO-APOC3, DCR-PHXC, SYL-1001, Fitusiran, Zilebesiran, VIR-2218, AB 729, Antisense K-ras RNA gene therapy, OLX 101A, Lumasiran, Prexigebersen-A, JNJ-3989, LY 3561774, DCR-HBVS, Olpasiran, STP705, ARO-HSD, ARO-MUC5AC, RBD1016, ANGPTL3 siRNA, SLN124, JNJ-75220795, LY3819469, ARO-HIF2, SYL 1801, STP707, DRC-AUD, SLN360, SR043, SR044, PH 894, PH 762PF-06650833, Polyoxidonium, Remimazolam, OP-101, Rayaldee, OT-101, ADM03820, OLT 1177, NVXCoV 2373, DFV890, Crizanlizumab, MAS-825, NBT-NM108, Ambrisentan, Sargramostim, Gam-COVID-Vac, mRNA-1283, MP1032, Molnupiravir, M5049, Covifenz, INOmax, MW33, RNA convalescent plasma, LB1148, Mavrilimumab, and others.

 

  • The RNA Interference companies and academics are working to assess challenges and seek opportunities that could influence RNA Interference R&D. The RNA Interference therapies under development are focused on novel approaches to treat/improve RNA Interference.

 

For further information, refer to the detailed RNA Interference Drugs Launch, RNA Interference Developmental Activities, and RNA Interference News, click here for RNA Interference Ongoing Clinical Trial Analysis

 

RNA Interference Emerging Drugs

 

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.

 

RNA Interference Pipeline Therapeutics Assessment

There are approx. 20+ key companies which are developing RNA Interference therapies. The RNA Interference companies which have their RNA Interference drug candidates in the most advanced stage, i.e. Pre-Registration include Alnylam Pharmaceuticals.

 

Find out more about the RNA Interference Pipeline Segmentation, Therapeutics Assessment, and RNA Interference Emerging Drugs @ RNA Interference Treatment Landscape

 

Scope of the RNA Interference Pipeline Report

 

  • Coverage- Global

 

  • RNA interference Companies- Alnylam Pharmaceuticals, Suzhou Ribo Life Science, Arrowhead Pharmaceuticals, Sylentis, Vir Biotechnology, Arbutus Biopharma, Silenseed, OliX Pharmaceuticals, Bio-Path Holdings, Janssen Pharmaceuticals, Eli Lilly and Company, Dicerna Pharmaceuticals, Amgen, Sirnaomics, Silence Therapeutics, Sylentis, Silence Therapeutics, Suzhou Ribo Life Science, Phio Pharmaceuticals, Amylon Therapeutics, Aro Biotherapeutics, DTx Pharma, BONAC Corporation, and others

 

  • RNA interference pipeline therapies- SR061, ARO-APOC3, DCR-PHXC, SYL-1001, Fitusiran, Zilebesiran, VIR-2218, AB 729, Antisense K-ras RNA gene therapy, OLX 101A, Lumasiran, Prexigebersen-A, JNJ-3989, LY 3561774, DCR-HBVS, Olpasiran, STP705, ARO-HSD, ARO-MUC5AC, RBD1016, ANGPTL3 siRNA, SLN124, JNJ-75220795, LY3819469, ARO-HIF2, SYL 1801, STP707, DRC-AUD, SLN360, SR043, SR044, PH 894, PH 762PF-06650833, Polyoxidonium, Remimazolam, OP-101, Rayaldee, OT-101, ADM03820, OLT 1177, NVXCoV 2373, DFV890, Crizanlizumab, MAS-825, NBT-NM108, Ambrisentan, Sargramostim, Gam-COVID-Vac, mRNA-1283, MP1032, Molnupiravir, M5049, Covifenz, INOmax, MW33, RNA convalescent plasma, LB1148, Mavrilimumab, and others.

 

  • RNA interference Pipeline Segmentation: Product Type, Molecule Type, Route of Administration

 

Dive deep into rich insights for drugs for RNA Interference Pipeline Companies and Therapies, click here @ RNA Interference Unmet Needs and Analyst Views

 

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

 

Got Queries? Find out the related information on RNA Interference Mergers and acquisitions, RNA Interference Licensing Activities @ RNA Interference Recent Trends, and Future Perspectives

 

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