RNA 치료법 : 경쟁 구도(2026년)

DelveInsight's, "RNA Therapies - Competitive landscape, 2026," report provides comprehensive insights about 80+ companies and 100+ drugs in RNA Therapies Competitive landscape. It 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 Therapies: Understanding

RNA Therapies: Overview

RNA therapy refers to the treatment or prevention of diseases using RNA-based molecules. RNA therapeutics involve the use of coding RNA such as mRNA as well as non-coding RNAs such as small interfering RNAs (siRNA), antisense oligonucleotides (ASO) to target mRNA, aptamers, ribozymes, and clustered regularly interspaced short palindromic repeats-CRISPR-associated (CRISPR/Cas) endonuclease to target proteins and DNA.

Types of RNA therapy

RNA-based drugs can be classified into four unique categories based on their structural characteristics and mode of action.

Antisense oligonucleotides: Antisense oligonucleotides modulate the expression of target RNAs via sequence-specific binding, and although the structure of these antisense oligonucleotides is determined primarily by their specific sequence, their chemistry can be modulated to produce novel effects.

Small interfering RNAs: siRNAs use the endogenous RNAi pathway to modulate the expression of their target RNAs. In native RNAi, endogenous small RNAs form a complex with Argonaute (Ago) protein to produce an RNA-induced silencing complex (RISC), which then suppresses the expression of their target mRNAs via sequence-specific binding.

Aptamers: Aptamers are nucleic acid constructs designed to bind specific proteins to modulate their function. Several RNA-based aptamers are under development, and additional aptamer-based therapeutics are expected to be available in the future.

Messenger RNAs: mRNA-based therapies exert their therapeutic effect by exploiting the fact that even exogenous mRNAs can be translated into functional proteins. mRNA-based therapies can be divided into two broad subcategories based on their purpose. In the first category, exogenous mRNAs are introduced into cells to replace or supplement endogenous proteins. In the second category, the mRNA transcript is designed to act as a vaccine against infectious diseases or cancer antigens.

Delivery Systems for RNA Therapeutics

The delivery of RNA-based drugs remains one of the single largest challenges for these kinds of therapies. Most delivery methods for these RNA-based therapeutics can be categorized as the addition of targeting moieties, the encapsulation of RNAs into lipid-based nanoparticles, and direct delivery into the target organ without extensive modification

Advantages of RNA therapy

RNA-based drugs are known to exhibit a wide variety of advantageous traits, making them ideal candidates for the development of various novel therapeutic strategies. Here, I will discuss some of the major advantages of these technologies over more conventional therapeutic compounds.

Report Highlights:

• In July 2024, Rgenta Therapeutics, announced the clearance of its Investigational New Drug application (IND) by the U.S. Food and Drug Administration (FDA) for RGT-61159, which is being developed for the potential treatment of adenoid cystic carcinoma (ACC), colorectal cancer (CRC) and other solid tumors as well as acute myeloid leukemia (AML).
• In June 2024, Ascidian Therapeutics, announced a research collaboration and licensing agreement with Roche for the discovery and development of RNA exon editing therapeutics targeting neurological diseases.
• In May 2024, Achilles Therapeutics plc announced a research collaboration with Arcturus Therapeutics Holdings Inc. to evaluate best-in-class, self-amplifying mRNA (sa-mRNA) personalized cancer vaccines (PCVs) targeting clonal neoantigens. The research collaboration will combine Achilles' best-in-class AI-driven, tumor-targeting technology with Arcturus' world-leading sa-mRNA platform.
• In April 2024, Ipsen and Skyhawk Therapeutics had signed an exclusive worldwide collaboration to discover and develop novel small molecules that modulate RNA for rare neurological diseases. The agreement includes an option pursuant to which Ipsen would acquire exclusive license for the worldwide rights to develop successful development candidates (DC).
• In April 2024, MiNA Therapeutics, announced a research collaboration and licensing agreement option with Nippon Shinyaku Co., Ltd. The collaboration will allow for the discovery, and potential development and commercialization of RNAa therapeutic candidates targeting rare neurodegenerative diseases for which there are currently no treatment options.
• In January 2024, Boehringer Ingelheim had agreed to collaborate with Suzhou Ribo Life Science Co and Ribocure Pharmaceuticals AB (Ribo), to develop new RNAi therapeutics for nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH). The deal exceeds a value of $2 billion and will utilize Ribo's expertise in small interfering RNA (siRNA) treatments.
• In January 2024, Ascidian Therapeutics announced that the FDA has cleared its application to begin the first RNA-editing clinical trial in the United States.

RNA Therapies: Company and Product Profiles (Marketed Therapies)

1. Company Overview: Novartis

Novartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, the company use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, the company consistently rank among the world's top companies investing in research and development. Novartis products reach nearly 1 billion people globally and they are finding innovative ways to expand access to our latest treatments. About 125 000 people of more than 140 nationalities work at Novartis around the world.

Product Description: Leqvio

Leqvio (inclisiran) is an injectable medication administered every six months to lower levels of 'bad' cholesterol called low-density lipoprotein (LDL) cholesterol. Leqvio is a siRNA designed to block a protein made in the liver called PCSK9 (proprotein convertase subtilisin kexin type 9). PCSK9 reduces the number of receptors available to clear 'bad' LDL cholesterol from your blood. Leqvio works by interfering with the translation of PCSK9 messenger RNA (mRNA). This limits the amount of PCSK9 produced, which helps to lower the amount of 'bad' cholesterol in the blood. Leqvio is used as an add-on to diet and statin therapy (another type of medication used to treat high cholesterol), to treat high LDL levels in certain adults with high cholesterol or atherosclerosis. Novartis has obtained global rights to develop, manufacture and commercialize Leqvio under a license and collaboration agreement with Alnylam Pharmaceuticals, a leader in RNAi therapeutics.

RNA Therapies: Company and Product Profiles (Pipeline Therapies)

1. Company Overview: Arrowhead Pharmaceuticals

Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead's RNAi-based therapeutics leverage this natural pathway of gene silencing.

Product Description: ARO-APOC3

ARO-APOC3 is designed to reduce 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. The drug is currently in Phase III stage of development for the treatment of patients with Hyperlipoproteinaemia type I.

2. Company Overview: Arbutus Biopharm

Arbutus Biopharma Corporation is a clinical-stage biopharmaceutical company leveraging its extensive virology expertise to develop novel therapeutics that target specific viral diseases. The current focus areas include Hepatitis B virus (HBV), SARS-CoV-2, and other coronaviruses. To address HBV, the company is developing a RNAi therapeutic, an oral PD-L1 inhibitor, and an oral RNA destabilizer to potentially identify a combination regimen with the aim of providing a functional cure for patients with chronic HBV by suppressing viral replication, reducing surface antigen and reawakening the immune system. The company believes the lead compound, AB-729, is the only RNAi therapeutic with evidence of immune re-awakening. AB-729 is currently being evaluated in multiple Phase II clinical trials.

Product Description: AB-729

AB-729 is an RNA interference (RNAi) therapeutic specifically designed to reduce all HBV viral proteins and antigens, including hepatitis B surface antigen, which is thought to be a key prerequisite to enable reawakening of a patient's immune system to respond to the virus. AB-729 targets hepatocytes using Arbutus' novel covalently conjugated N-Acetylgalactosamine (GalNAc) delivery technology that enables subcutaneous delivery. Clinical data generated thus far has shown single- and multi-doses of AB-729 to be generally safe and well-tolerated while providing meaningful reductions in hepatitis B surface antigen and hepatitis B DNA. The drug is currently in multiple Phase II stage of development for the treatment of chronic hepatitis B virus (cHBV).

3. Company Overview: Arrowhead Pharmaceuticals

Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead's RNAi-based therapeutics leverage this natural pathway of gene silencing.

Product Description: ARO-C3

ARO-C3, the company's investigational RNA interference (RNAi) therapeutic designed to reduce production of complement component 3 (C3) as a potential therapy for various complement mediated diseases. The company plans to present additional results at an upcoming complement-focused medical meeting. The therapy has so far been generally well tolerated, with no serious side effects reported and no participants discontinuing the study due to adverse events. The drug is currently in Phase I/II stage of development for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH).

4. Company Overview: Dicerna Pharmaceuticals, Inc.

Dicerna Pharmaceuticals, Inc. is a biopharmaceutical company focused on discovering, developing and commercializing medicines that are designed to leverage ribonucleic acid interference (RNAi) to silence selectively genes that cause or contribute to disease. Using our proprietary GalXC(TM) and GalXC-Plus(TM) RNAi technologies, Dicerna is committed to developing RNAi-based therapies with the potential to treat both rare and more prevalent diseases. By silencing disease-causing genes, Dicerna's GalXC platform has the potential to address conditions that are difficult to treat with other modalities. Initially focused on disease-causing genes in the liver, Dicerna has continued to innovate and is exploring new applications of its RNAi technology with GalXC-Plus, which expands the functionality and application of our flagship liver-targeted GalXC technology to tissues and cell types outside the liver, and has the potential to treat diseases across multiple therapeutic areas.

Product Description: DCR-AUD

DCR-AUD is Dicerna's GalXC(TM) RNAi investigational candidate designed to silence ALDH2 (aldehyde dehydrogenase 2) messenger RNA (mRNA) expression in the liver. DCR-AUD has been shown to induce long-lasting liver-specific ALDH2 mRNA knockdown in nonclinical studies. Some individuals are born with naturally occurring mutations in one or both gene copies that encode the ALDH2 enzyme. In these people with ALDH2 mutations, alcohol consumption can result in uncomfortable physiological effects that occur soon after drinking. These effects are thought to be the reason people with ALDH2 mutations are much less likely to be affected by AUD. Dicerna designed DCR-AUD based on human genetic data that suggest knocking down ALDH2 mRNA in individuals with AUD may provide similar physiological feedback that is protective against harmful levels of alcohol consumption. Preclinical research for DCR-AUD was supported by a grant from the National Institute on Alcohol Abuse and Alcoholism of the National Institutes of Health (NIH), under Award Number U44AA027404. The drug is currently in Phase I stage of development for the treatment of patients with alcoholism.

5. Company Overview: SanegeneBio

VYNE's mission is to improve the lives of patients by developing proprietary, innovative, and differentiated therapies for the treatment of immuno-inflammatory conditions. The Company's unique and proprietary pipeline includes access to a library of bromodomain & extra-terminal (BET) domain inhibitors licensed from In4Derm Limited. The BET inhibitor platform includes lead programs and access to a library of (BET) domain inhibitors for the potential treatment of immuno-inflammatory conditions.

Product Description: SGB-9768

SGB-9768, a siRNA-N-acetylgalactosamine (GalNAc) conjugate targeting C3, is delivered to liver using SanegeneBio's novel LEAD(TM) GalNAc platform to reduce production of complement component 3 through RNA interference. The advantages of GalNAc platform have been also fully verified, including safety, effectiveness, and stability. SGB-9768 can be administered every 3 or 6 months, with the advantages of low dosing frequency, good patient compliance, and long-term efficacy. SGB-9768 can continuously reduce C3 synthesis and has been shown in preclinical studies with superior efficacy than benchmark compounds, showing its potential to be the best-in-class siRNA drug targeting C3. The drug is currently in Preclinical stage of development for the treatment of patients with Immunological disorders.

6. Company Overview: Alnylam Pharmaceuticals

Alnylam has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam's commercial RNAi therapeutic products are ONPATTRO(R) (patisiran), GIVLAARI(R) (givosiran), OXLUMO(R) (lumasiran) and Leqvio(R) (inclisiran) being developed and commercialized by Alnylam's partner, Novartis. Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam is executing on its "Alnylam P5x25" strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile.

Product Description: Cemdisiran

Cemdisiran (ALN-CC5) is a subcutaneously administered, investigational RNAi therapeutic targeting the C5 component of the complement pathway in development for the treatment of complement-mediated diseases. Cemdisiran utilizes our Enhanced Stabilization Chemistry (ESC)-GalNAc delivery platform.

RNA Therapies Analytical Perspective by DelveInsight

• In-depth Commercial Assessment: RNA Therapies Collaboration Analysis by Companies

The Report provides in-depth commercial assessment of drugs that have been included, which comprises collaboration, agreement, licensing and acquisition - deals values trends. The sub-segmentation is described in the report which provide company-company collaboration (licensing/partnering), company academic collaboration and acquisition analysis in tabulated form.

• RNA Therapies Competitive Landscape

The report comprises of comparative assessment of Companies (by therapy, development stage, and technology).

RNA Therapies Report Assessment

• Company Analysis
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions:

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing RNA Therapies drugs?
• How many RNA Therapies drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of RNA Therapies?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the RNA Therapies 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 Therapies and their status?
• What are the key designations that have been granted to the emerging and approved drugs?

Key Players

• Novartis
• Arrowhead Pharmaceuticals
• Arbutus Biopharm
• Dicerna Pharmaceuticals, Inc.
• SanegeneBio
• Alnylam Pharmaceuticals
• Eli Lilly and Company
• Janssen Pharmaceuticals
• Moderna Therapeutics
• OliX Pharmaceuticals
• ProQR Therapeutics
• Fujian Shengdi Pharmaceutical
• Silence Therapeutics
• WaVe life Sciences
• Sirnaomics
• ExoRNA Bioscience
• Comanche Biopharma
• Ractigen Therapeutics
• GeneCare Research Institute
• Sirana Pharma
• DTx Pharma
• Aptadel Therapeutics

Key Products

• Leqvio
• ARO-APOC3
• AB-729
• ARO-C3
• DCR-AUD
• SGB-9768
• Cemdisiran
• LY3561774
• JNJ-75220795
• mRNA 1230
• OLX 702A
• Sepofarsen
• HRS 5635
• Divesiran
• WVE 003
• STP 122G
• STP 707
• ER 2001
• CBP 4888
• RAG 17
• RECQL1 siRNA
• Research programme: musculoskeletal disorders
• Research programme: RNA therapeutics
• ADEL 001

Table of Contents

Introduction

Executive Summary

RNA Therapies: Overview

• Introduction
• Classification
• Risk factors
• Diagnosis
• Treatment

RNA Therapies -Analytical Perspective: In-depth Commercial Assessment

• RNA Therapies Collaboration Analysis by Companies

Competitive Landscape

• Comparative Assessment of Companies (by therapy, development stage, and technology)

Therapeutic Assessment

• Assessment by Product Type
• Assessment by Stage and Product Type
• Assessment by Route of Administration
• Assessment by Stage and Route of Administration
• Assessment by Molecule Type
• Assessment by Stage and Molecule Type

RNA Therapies: Company and Product Profiles (Marketed Therapies)

Novartis

• Company Overview

Leqvio

• Product Description
• Research and Development Activities
• Product Developmental Activities

RNA Therapies: Company and Product Profiles (Pipeline Therapies)

Late Stage Products (Phase III)

• Comparative Analysis

Arrowhead Pharmaceuticals

• Company Overview

ARO-APOC3

• Product Description
• Research and Development Activities
• Product Developmental Activities

Mid Stage Products (Phase II)

• Comparative Analysis

Arbutus Biopharm

• Company Overview

AB-729

• Product Description
• Research and Development Activities
• Product Developmental Activities

Early Stage Products (Phase I/II)

• Comparative Analysis

Arrowhead Pharmaceuticals

• Company Overview

ARO-C3

• Product Description
• Research and Development Activities
• Product Developmental Activities

Preclinical and Discovery Stage Products

• Comparative Analysis

SanegeneBio

• Company Overview

SGB-9768

• Product Description
• Research and Development Activities
• Product Developmental Activities

Inactive Products

• Comparative Analysis

RNA Therapies- Unmet needs

RNA Therapies - Market drivers and barriers