RNA interference - Pipeline Insight, 2024

This “RNA interference - Pipeline Insight, 2024” report provides comprehensive insights about 20+ companies and 20+ 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) 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.

Function - RNA molecules play numerous roles in both normal cellular processes and disease states. Generally, those RNA molecules that do not take the form of m RNA are referred to as noncoding, because they do not encode proteins. The involvement of noncoding m RNAs in many regulatory processes, their abundance, and their diversity of functions has led to the hypothesis that an RNA world may have preceded the evolution of DNA and proteins e-specific RNA degradation process (posttranscriptional gene silencing [PTGS]/RNA interference [RNAi]). RNA silencing is a novel gene regulatory mechanism that limits the transcript level by either suppressing transcription (transcriptional gene silencing [TGS]) or by activatingasequenc

RNA interference - RNA interference (RNAi) is a biological process by which double-stranded RNA (ds RNA) induces sequence-specific gene silencing by targeting m RNA for degradation. As a tool for knocking down the expression of individual genes post transcriptionally, RNAi has been widely used to study the cellular functionofgenes.

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 III, 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

Fitusiran: Alnylam Pharmaceuticals Fitusiran (ALN-AT3) is a subcutaneously administered, investigational RNAi therapeutic targeting antithrombin (AT) in development for the treatment of hemophilia and rare bleeding disorders (RBDs) by our collaborators at Sanofi Genzyme. The drug is currently in phase 3 of development for the treatment of patients with Haemophilia.

WVE 120102: WaVe life Sciences WVE 120102 is being developed by WaVe life Sciences for the treatment of Huntington disease. The therapy is based on RNAi technology and targets a specific single nucleotide polymorphism (SNP3) within the huntingtin gene. It is currently in phase I/II of clinicaldevelopment.

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 working on RNA interference

There are approx. 20+ key companies which are developing the RNA interference. The companies which have their RNA interference drug candidates in the most advanced stage, i.e. phase 3 include, Alnylam Pharmaceuticals.

Phases

This report covers around 20+ products under different phases of clinical development like

• Late-stage products (Phase III and
• Mid-stage products (Phase II and
• Early-stage products (Phase I/II and 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

• Infusion
• Intradermal
• Intramuscular
• Intranasal
• Intravaginal
• Oral
• Parenteral
• Subcutaneous
• Topical.

Molecule Type

Products have been categorized under various Molecule types such as

• Vaccines
• Monoclonal Antibody
• Peptides
• Polymer
• 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.

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 for RNA interference.

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 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 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
• WaVe life Sciences
• Medesis Pharma
• Bio-Path Holdings
• Sirnaomics
• Arrowhead Pharmaceuticals
• Silence Therapeutics
• Dicerna Pharmaceuticals
• Novo Nordisk
• Eli Lilly and Company
• Ionis Pharmaceuticals
• Quark Pharmaceuticals
• Pro QRTherapeutics
• Arbutus Biopharma
• Atugen AG
• Sylentis
• Bristol-Myers Squibb
• OliX Pharmaceuticals
• Janssen Pharmaceuticals
• Amgen
• mi RagenTherapeutics
• Regulus Therapeutics

Key Products

• Fitusiran
• WVE 120102
• si RNAtherapeutic
• BP 1003
• STP 705

Research programme: short interfering RNA-based therapeutics- IONIS AGT LRx

• QPI 1007
• Teprasiran
• Eluforsen
• RG 6346
• AB 729
• QR-421a
• Sepofarsen
• PF 4523655
• ALN VSP
• Atu027
• Bamosiran
• BMS 986263
• ARO APOC3
• Tivanisiran
• OLX 101A
• TAK 999
• Nedosiran
• Olpasiran
• Remlarsen
• MRG 110
• Lademirsen

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