RNA Competitive Landscape

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 therapies are a rapidly advancing class of treatments that use ribonucleic acid (RNA) molecules to prevent, treat, or potentially cure diseases by targeting the genetic instructions inside cells. Unlike traditional drugs that mainly target proteins, RNA therapeutics can directly influence gene expression and protein production. Major categories include messenger RNA (mRNA), small interfering RNA (siRNA), antisense oligonucleotides (ASOs), aptamers, and CRISPR-associated RNA technologies. These therapies have gained global attention after the successful development of mRNA vaccines during the COVID-19 pandemic.

Different RNA therapies work through distinct biological mechanisms. mRNA therapies deliver synthetic messenger RNA into cells so they can produce therapeutic proteins. siRNA therapies silence harmful genes by degrading target messenger RNA before proteins are produced. Antisense oligonucleotides bind to RNA transcripts and modify or block protein production, including correcting abnormal RNA splicing in genetic disorders. Researchers are also developing RNA-based genome-editing approaches using CRISPR systems. These technologies provide opportunities to target diseases previously considered “undruggable.”

RNA therapeutics are being investigated and approved for a broad range of diseases, including rare genetic disorders, cancers, cardiovascular diseases, and infectious diseases. mRNA vaccine technology demonstrated how rapidly RNA platforms can be adapted for emerging infections. Several FDA-approved RNA medicines are already used clinically, and many more are in clinical development worldwide.

Despite major progress, RNA therapies still face important challenges related to delivery, stability, immune reactions, and manufacturing. RNA molecules are naturally fragile and can degrade quickly in the body, so advanced delivery systems such as lipid nanoparticles and engineered carriers are often required. Scientists are continuously improving chemical modifications and targeting strategies to enhance safety and effectiveness. Experts believe RNA therapeutics may transform precision medicine because they can be designed relatively quickly and tailored to individual genetic diseases. Future developments may expand their use in personalized cancer treatment, regenerative medicine, and chronic disease management.

RNA therapies Pipeline Report Highlights

• In May 2026, China’s NMPA approved a new clinical trial application (CTA) for SGB-9768 for the treatment of complement-mediated hematologic disorders, including Paroxysmal Nocturnal Hemoglobinuria (PNH) and Atypical Hemolytic Uremic Syndrome (aHUS).
• In April 2026, The U.S. FDA granted Fast Track designation for imdusiran (AB-729) for the treatment of chronic hepatitis B (cHBv), a designation intended to facilitate development and expedite review.
• In April 2026, Regeneron Pharmaceuticals, Inc. announced detailed positive results from the Phase III NIMBLE trial evaluating investigational cemdisiran in adults with generalized myasthenia gravis (gMG) were published in The Lancet and presented for the first time in an oral plenary session at the American Academy of Neurology (AAN) Annual Meeting. Cemdisiran is a novel siRNA therapeutic that durably reduces circulating levels of complement factor 5 (C5), allowing for every three months dosing.
• In February 2026, The U.S. FDA granted Orphan Drug Designation (ODD) for SGB-9768 for the treatment of Immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN).
• In January 2026, Health Canada approved REDEMPLO™ (plozasiran) to reduce triglycerides in adults with Familial Chylomicronemia Syndrome (FCS).
• In January 2026, AVM and Ai Technology Group, who signed a definitive merger agreement in October 2025, provided an update in January 2026 that they are extending funding for the planned merger and that AVM has re-commenced its FDA Phase 2 trial (OPAL Trial)
• In November 2025, The U.S. FDA approved REDEMPLO® (plozasiran) as the first and only medicine for genetically confirmed or clinically diagnosed FCS, based on positive results from the Phase III PALISADE study.
• In November 2025, SanegeneBio announced the presentation of Phase I clinical data for SGB-9768 at ASN Kidney Week 2025, showing a favorable safety profile and significant, dose-dependent reduction in serum C3 levels.
• In July 2025, The FDA approved a new label update for LEQVIO, enabling its use as a first-line monotherapy (along with diet and exercise) to lower LDL-C in adults with hypercholesterolemia, removing the previous requirement to use it on top of statin therapy.
• In April 2025, The European Medicines Agency (EMA) granted Orphan Drug Designation to CBP-4888 for the treatment of sFlt1-mediated preterm preeclampsia.
• In March 2025, RiboX Therapeutics announced that the first patient had been dosed in a Phase I/IIa clinical trial (SPRINX-1) for RXRG001, a circular RNA therapy for radiation-induced xerostomia and hyposalivation.
• In March 2025, Arrowhead announced topline results from Part 2 of a Phase I/II study of ARO-C3 in patients with IgA nephropathy (IgAN). The study showed the drug achieved deep and sustained reductions in alternative pathway complement activity and proteinuria.
• In January 2025, Comanche Biopharma was awarded an Innovation Passport by the U.K.'s Innovative Licensing and Access Pathway (ILAP) Steering Group for CBP-4888.
• In November 2024, The U.S. FDA cleared the IND application for HG202, making it the world’s first-ever CRISPR/Cas13 RNA-editing therapy for clinical use in treating neovascular age-related macular degeneration (nAMD).
• In October 2024, The FDA approved an Investigational New Drug (IND) application for RXRG001, making it the first circular RNA therapy to receive FDA clearance to enter clinical trials.
• In August 2024, Arrowhead announced a $500 million strategic financing facility with Sixth Street to advance plozasiran towards a 2025 launch and fund pipeline growth.
• In July 2024, Sirnaomics announced the successful completion of the second cohort in its Phase I clinical study of STP122G, a GalNAc-based RNAi therapeutic targeting Factor XI for anticoagulant treatment.
• In May 2024, Arbutus announced that data from Phase IIa trials of imdusiran (AB-729) would be presented at the EASL Congress 2024. The data covers its use in combination therapies aimed at achieving functional cure for cHBv.

RNA therapies Company and Product Profiles (Marketed Therapies)

1. Company Overview: Novartis

Novartis is a global innovative medicine company, focused on reimagining medicine to improve and extend people’s lives. The company develops and commercializes therapies across multiple disease areas, including cardiovascular, immunology, neuroscience, oncology, and rare diseases. Novartis combines advanced science and technology platforms such as gene and cell therapy, radioligand therapy, and RNA-based therapeutics to address serious diseases with high unmet medical need. In cardiovascular medicine, Novartis is committed to transforming care through innovative approaches that improve long-term patient outcomes and reduce disease burden worldwide.

Product Description: LEQVIO®

LEQVIO® (inclisiran) is a first-in-class small interfering RNA (siRNA) therapy developed by Novartis for lowering low-density lipoprotein cholesterol (LDL-C), also known as “bad” cholesterol. As an RNA therapy, inclisiran works through an RNA interference (RNAi) mechanism by preventing the liver from producing PCSK9, a protein that reduces the liver’s ability to remove LDL-C from the bloodstream. By silencing the PCSK9 gene message, LEQVIO helps increase LDL-C clearance and provides sustained cholesterol reduction. The therapy is administered as a subcutaneous injection initially, again at three months, and then every six months thereafter. Novartis describes LEQVIO as the first and only FDA-approved siRNA therapy for LDL-C reduction, designed to support adults with hypercholesterolemia or atherosclerotic cardiovascular disease who require additional cholesterol lowering despite standard therapy.

2. Company Overview: Arrowhead Pharmaceuticals

Arrowhead Pharmaceuticals is a clinical-stage biotechnology company focused on developing innovative RNA interference (RNAi) therapies that silence genes responsible for causing disease. The company leverages its proprietary Targeted RNAi Molecule (TRiM™) platform to design therapies that selectively reduce the production of disease-causing proteins. Arrowhead’s research and development efforts span multiple therapeutic areas, including cardiovascular, metabolic, pulmonary, liver, and rare diseases. According to the company, Arrowhead aims to develop transformative medicines by harnessing the natural mechanism of RNA interference to achieve durable gene silencing with infrequent dosing. The company has built a broad pipeline of investigational RNAi therapeutics targeting significant unmet medical needs worldwide.

Product Description: Plozasiran

Plozasiran is designed to reduce the production of the protein Apolipoprotein-CIII (ApoC3) through the natural RNA interference (RNAi) mechanism.  ApoC3 is a protein that is produced in liver cells and inhibits the formation and clearance of various lipids and lipoproteins, including triglycerides. Plozasiran  used for the treatment of severe hypertriglyceridemia and related dyslipidemias. The therapy is designed to reduce the production of apolipoprotein C-III (APOC3), a protein produced in the liver that plays a key role in regulating triglyceride metabolism. Using Arrowhead’s TRiM™ platform, ARO-APOC3 delivers small interfering RNA (siRNA) molecules to liver cells, where they silence APOC3 messenger RNA and reduce APOC3 protein synthesis.

RNA therapies: Company and Product Profiles (Pipeline Therapies)

1. Company Overview: Alnylam Pharmaceuticals

Alnylam Pharmaceuticals is a leading independent biopharmaceutical company and a pioneer in RNA interference (RNAi) therapeutics. The company focuses on developing medicines that silence disease-causing genes through RNAi technology. Alnylam has developed multiple approved RNAi therapeutics and continues to advance a broad pipeline targeting rare, cardiovascular, metabolic, neurologic, and other serious diseases. The company describes itself as “leading the RNAi revolution” by leveraging gene-silencing science to transform the treatment of genetically defined diseases.

Product Description: Cemdisiran

Cemdisiran is an investigational RNAi therapeutic developed by Alnylam Pharmaceuticals and licensed to Regeneron. The drug is designed to silence the complement component 5 (C5) protein, which plays a key role in complement-mediated diseases. Cemdisiran is being evaluated for conditions including myasthenia gravis, paroxysmal nocturnal hemoglobinuria (PNH), and geographic atrophy. Currently, the drug is in Phase III  development stage.

2. Company Overview: Hyundai Bioscience Co., Ltd

Hyundai Bioscience Co., Ltd is a global biotechnology company focused on developing innovative therapeutics based on advanced drug delivery system technologies. The company is pioneering new treatment paradigms through broad-spectrum antivirals and pseudo-resistance therapeutics by leveraging mechanism-based drug development and proprietary delivery platforms. Hyundai Bioscience states that its mission is to provide safer, more effective, and scalable treatment options through scientific innovation and advanced biotechnology.

Product Description: CP-COV03

CP-COV03 is an investigational broad-spectrum antiviral therapeutic developed by Hyundai Bioscience Co., Ltd using niclosamide as its active ingredient combined with the company’s proprietary drug delivery system technology. The drug is designed as a host-directed antiviral therapy that promotes autophagy, enabling infected cells to eliminate viruses directly rather than targeting specific viral proteins. CP-COV03 is being developed for multiple viral infections including COVID-19 and other viral diseases. Currently, the drug is in Phase II/III  development stage.

3. Company Overview: Arbutus Biopharma

Arbutus Biopharma is a clinical-stage biopharmaceutical company focused on developing novel therapeutics for chronic hepatitis B virus (cHBV) infection. The company is committed to identifying a functional cure for hepatitis B by targeting different stages of the viral life cycle and reactivating the patient’s immune response. Arbutus leverages expertise in RNA interference (RNAi), oral antivirals, and immune modulation technologies to advance therapies aimed at reducing viral replication and antigen production. Arbutus is developing combination approaches designed to achieve sustained control of HBV infection and improve long-term clinical outcomes for patients living with chronic hepatitis B worldwide.

Product Description: AB-729

AB-729 is an investigational RNA interference (RNAi) therapeutic developed by Arbutus Biopharma for the treatment of chronic hepatitis B (cHBV). The therapy uses a conjugated GalNAc-delivered RNAi mechanism designed to specifically target hepatocytes and reduce the production of hepatitis B surface antigen (HBsAg), which plays a key role in maintaining chronic HBV infection and suppressing immune responses. By silencing HBV RNA transcripts, AB-729 aims to lower viral antigens and potentially restore the body’s immune control over the infection. AB-729 has demonstrated meaningful reductions in HBsAg levels in clinical studies and is being evaluated as part of combination treatment strategies intended to achieve a functional cure for chronic hepatitis B. The drug is currently in Phase II stage of clinical development for chronic hepatitis B.

4. Company Overview: SanegeneBio

SanegeneBio is a clinical-stage biotechnology company focused on developing innovative RNA interference (RNAi) therapeutics for obesity, cardiometabolic, autoimmune, and complement-mediated diseases. The company utilizes its proprietary LEAD™ (Ligand and Enhancer Assisted Delivery) platform and GalNAc delivery technologies to enable tissue-specific delivery of siRNA therapeutics with infrequent subcutaneous dosing. SanegeneBio is advancing a broad RNAi pipeline targeting liver and extrahepatic tissues to address significant unmet medical needs globally.

Product Description: SGB-9768

SGB-9768 is an investigational siRNA-based RNAi therapeutic developed by SanegeneBio that targets complement component 3 (C3) for the treatment of complement-mediated kidney diseases, including IgA nephropathy (IgAN), C3 glomerulopathy (C3G), and immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN). The drug uses the company’s proprietary GalNAc liver-targeting delivery platform to suppress C3 expression through RNA interference, thereby reducing complement pathway overactivation. SGB-9768 is currently in Phase II clinical development and has received FDA Orphan Drug Designation for C3G and IC-MPGN.

5. Company Overview: Arrowhead Pharmaceuticals

Arrowhead Pharmaceuticals is a clinical-stage biotechnology company focused on developing RNA interference (RNAi) therapeutics designed to silence genes that cause disease. The company utilizes a broad portfolio of RNA chemistries and targeted delivery technologies to develop medicines across multiple therapeutic areas, including cardiometabolic, pulmonary, liver, and renal diseases. Arrowhead’s therapies harness the natural RNAi pathway to induce rapid, deep, and durable suppression of disease-causing genes. The company is advancing a broad pipeline of investigational RNA-based therapeutics intended to address diseases that were previously considered difficult or impossible to treat with conventional approaches.

Product Description: ARO-C3

ARO-C3 is an investigational RNA interference (RNAi) therapeutic developed by Arrowhead Pharmaceuticals for the treatment of complement-mediated diseases, including IgA nephropathy and other renal disorders. The therapy is designed to reduce liver production of complement component 3 (C3), a central protein involved in activation of the complement cascade that contributes to inflammation and tissue injury in complement-mediated diseases. By silencing C3 messenger RNA, ARO-C3 aims to modulate abnormal complement activation and potentially reduce disease progression. The therapy has demonstrated deep and sustained reductions in complement activity markers and proteinuria in clinical studies involving patients with IgA nephropathy. The drug is currently in Phase I/II stage of clinical development for complement-mediated renal diseases.

6. Company Overview: Sirnaomics

Sirnaomics is a clinical-stage RNA therapeutics biopharmaceutical company focused on the discovery and development of innovative RNA-based medicines for diseases with significant unmet medical needs, including cancer, fibrosis-related diseases, antiviral disorders, cardiometabolic diseases, and medical aesthetics. The company operates across the United States and Greater China and utilizes proprietary delivery technologies, including its polypeptide nanoparticle (PNP) platform and GalAhead™ GalNAc delivery platform, to develop siRNA and mRNA therapeutics. Sirnaomics has established a broad pipeline of RNAi candidates in both preclinical and clinical development stages.

Product Description: STP122G

STP122G is an investigational siRNA-based RNA interference therapeutic developed by Sirnaomics that targets Factor XI for anticoagulation treatment. The drug is part of the company’s proprietary GalAhead™ Factor XI RNAi therapeutic program and is designed to inhibit the production of Factor XI in hepatocytes through GalNAc-mediated liver-targeted delivery. STP122G is being developed for a broad range of anticoagulation-related indications, including prevention and treatment of stroke after atrial fibrillation, prevention of deep vein thrombosis, pulmonary embolism, and improving recovery after total knee replacement. STP122G is currently in Phase I clinical development and represents the company’s first GalAhead™-based RNAi drug candidate entering clinical trials.

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

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

• RNA therapies Company Analysis
• RNA therapies Therapeutic Assessment
• RNA therapies Pipeline Assessment
• RNA therapies Inactive drugs assessment
• RNA therapies Market Unmet Needs

Key Questions Answered In The RNA therapies Pipeline Report:

• 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?

RNA therapies Key Players

• Novartis
• Arrowhead Pharmaceuticals
• Alnylam Pharmaceuticals
• Hyundai Bioscience Co., Ltd
• Arbutus Biopharma
• SanegeneBio
• Sirnaomics
• Comanche Biopharma
• Regeneron Pharmaceuticals
• Silence Therapeutics plc
• AVM Biotechnology Inc
• RiboX Therapeutics Ltd.
• HuidaGene Therapeutics Co., Ltd.
• Immatics US, Inc.

RNA therapies Key Products

• LEQVIO®
• Plozasiran
• Cemdisiran
• CP-COV03
• AB-729
• SGB-9768
• ARO-C3
• STP122G
• SLN124
• ALN-CFB
• AVM0703
• CBP-4888
• RXRG001
• HG202
• IMA203