RNA Interference- Pipeline Insight, 2024

RNA Interference- Pipeline Insight, 2024

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

Geography Covered

• Global coverage

RNA Interference Understanding

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. RNA interference (RNAi) or Post-Transcriptional Gene Silencing (PTGS) is a conserved biological response to double-stranded RNA that mediates resistance to both endogenous parasitic and exogenous pathogenic nucleic acids, and regulates the expression of protein-coding genes. This natural mechanism for sequence-specific gene silencing promises to revolutionize experimental biology and may have important practical applications in functional genomics, therapeutic intervention, agriculture and other areas. Therapeutically, RNAi works via delivery of small RNA duplexes, including microRNA (miRNA) mimics, short interfering RNAs (siRNAs), short hairpin RNAs (shRNAs), and Dicer substrate RNAs (dsiRNAs). As a therapeutic, all four types of RNAi inputs are currently in clinical trials. Major considerations include toxicity, efficacy, and delivery. RNAi therapeutic toxicity occurs via improper target recognition or immunogenic effects from exogenously introduced RNA, in addition to any toxic effects due to delivery methodology. RNA interference is a highly conserved biological mechanism that responds to pathogenic viral dsRNA to mount an inhibitory molecular response. Many studies have shown that RNAi can be used therapeutically to target many respiratory viruses in vitro and in vivo, including SARS-CoV and potentially SARS-CoV-2, in the treatment and prevention of infection.

""RNA Interference- Pipeline Insight, 2024"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the RNA Interference pipeline landscape is provided which includes the disease overview and RNA Interference treatment guidelines. The assessment part of the report embraces, in depth RNA Interference commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, RNA Interference collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

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

RNA Interference Emerging Drugs Chapters

This segment of the RNA Interference report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

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.

Further product details are provided in the report……..

RNA Interference: Therapeutic Assessment

This segment of the report provides insights about the different RNA Interference drugs segregated based on following parameters that define the scope of the report, such as:

• Major Players in RNA Interference

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

• Phases

DelveInsight’s 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) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates
• 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
• Molecule Type

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule
• Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

RNA Interference: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses RNA Interference therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging RNA Interference drugs.

RNA Interference Report Insights

• RNA Interference Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

RNA Interference Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing RNA Interference drugs?
• How many RNA Interference drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of RNA Interference?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the RNA Interference therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for RNA Interference and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• 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

Key Products

• Vutrisiran
• Cemdisiran
• 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
• ARO-AAT
• ARO-ANG3

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