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
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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
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
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Table of Content
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