RNA Interference Therapy-Competitive Landscape, 2026

This “RNA Interference Therapy - Competitive landscape, 2026,” report provides comprehensive insights about 40+ companies and 90+ drugs in RNA Interference Therapy 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.

Geographical Coverage: Global

RNA Interference Therapy: Understanding

RNA Interference Therapy: 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.

Development of new therapeutics is a constant battle for successful, safe, clinical trials that maintain clinical efficacy. Small-molecule drugs were traditionally the solution for all diseases until the development of monoclonal antibody therapy. Monoclonal antibody therapy has been successful for many diseases, but still incurs difficulties with tissue penetration, manufacturing, and purification. RNA interference (RNAi) therapeutics provide alternative treatment options when current drug technology fails. Additionally, therapeutics for infectious diseases are quickly obsolete owing to mutational escape. Development of RNAi strategies addresses both needs; thus, it may be the next technology to revolutionize the drug market.

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.

Fast progress in RNAi technology has shown promise for use in reverse genetics and therapy. However, mechanistic complexities of this technology still need to be determined. RNAi has now been established as a revolutionary tool for functional genomics in organisms. Multiple studies have defined the role of RNAi in mammalian and plant defense systems. A plethora of studies have utilized RNAi technology to modulate gene expression. RNAi-based full genomic screens have allowed identification of specific genes, controlling a given trait with high accuracy.

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. More research is needed both at the basic and clinical settings before such therapies are made routine.

Latest Developments

• In February 2026, SanegeneBio, a clinical-stage biotechnology company developing innovative RNAi therapeutics, announced a global licensing agreement with Genentech, a member of the Roche Group, to advance one of its RNAi programs based on its proprietary RNAi platform. Under the agreement, SanegeneBio grants Genentech exclusive worldwide rights to develop and commercialize the program. SanegeneBio will be responsible for early development activities and Genentech will subsequently lead all future clinical development and commercialization activities.
• In September 2025, Arrowhead Pharmaceuticals, Inc. announced a global licensing and collaboration agreement with Novartis for ARO-SNCA, Arrowhead’s preclinical stage siRNA therapy against alpha-synuclein for the treatment of synucleinopathies, such as Parkinson’s Disease, and for other additional collaboration targets that will utilize Arrowhead’s proprietary Targeted RNAi Molecule (TRiM™) platform. Upon closing, Arrowhead will receive USD 200 million as an upfront payment and is eligible to receive up to USD 2 billion in potential milestone payments plus royalties on commercial sales.
• In March 2023, Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, and Medison Pharma, a global pharma company focused on providing access to highly innovative therapies for patients in international markets, announced an expansion of their existing partnership to a multi-regional agreement that includes Poland, Czech Republic, Hungary, Slovakia, Lithuania, Estonia and Latvia, in addition to Israel.
• In July 2021, Arbutus Biopharma Corporation and Vaccitech entered into a clinical trial collaboration agreement to evaluate an innovative therapeutic combination for the treatment of subjects with chronic hepatitis B virus (HBV) infection (CHB) who are already receiving standard-of-care nucleos(t)ide reverse transcriptase inhibitor (NrtI) therapy.
• In December 2021, Arbutus Biopharma Corporation and Qilu Pharmaceutical, entered into an exclusive licensing agreement and strategic partnership for the development and commercialization of AB-729 for the treatment or prevention of hepatitis B in mainland China, Hong Kong, Macau and Taiwan.
• In November 2021, Arrowhead Pharmaceuticals Inc. announced that its collaborator, Janssen Pharmaceuticals, Inc., (Janssen) one of the Janssen Pharmaceutical Companies of Johnson & Johnson, has disclosed its collaboration with Arrowhead on investigational compound JNJ-75220795, which is in a Phase 1 clinical study.
• In August 2020, Assembly Biosciences and Arbutus Biopharma Corporation, entered into a clinical collaboration agreement to evaluate Assembly’s lead hepatitis B virus (HBV) core inhibitor candidate ABI-H0731 in combination with Arbutus’ proprietary GalNAc delivered RNAi therapeutic AB-729 and standard-of-care nucleos(t)ide reverse transcriptase inhibitor (NrtI) therapy for the treatment of patients with chronic HBV infection. As part of the collaboration, the companies may add cohorts in the future, to evaluate other patient populations and/or combinations.
• In April 2018, Regeneron Pharmaceuticals and Alnylam Pharmaceuticals announced a collaboration to discover, develop and commercialize new RNA interference (RNAi) therapeutics for a broad range of diseases by addressing disease targets expressed in the eye and central nervous system (CNS), in addition to a select number of targets expressed in the liver.

RNA Interference Therapy: Company and Product Profiles (Marketed Therapies)

1. Company Overview: Alnylam Pharmaceuticals

Alnylam Pharmaceuticals is a pioneering biotechnology company specializing in RNA interference (RNAi) therapeutics. Alnylam is a global leader in developing RNAi-based medicines to treat diseases caused by gene overexpression or mutation. The company’s innovative therapies, such as Patisiran (Onpattro) and Inclisiran (Leqvio), target conditions like hereditary amyloidosis and hypercholesterolemia. Alnylam’s robust pipeline focuses on rare genetic, cardiovascular, hepatic, and CNS diseases, leveraging RNAi technology to silence specific disease-causing genes. Through groundbreaking research and approvals, Alnylam has transformed RNAi into a clinically viable therapeutic platform.

Product Description: Lumasiran

Lumasiran (Oxlumo) is an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients. HAO1 encodes glycolate oxidase (GO), an enzyme upstream of the disease-causing defect in PH1. OXLUMO works by degrading HAO1 messenger RNA and reducing the synthesis of GO, which inhibits hepatic production of oxalate - the toxic metabolite responsible for the clinical manifestations of PH1. In November 2020, Alnylam Pharmaceuticals, Inc. announced the US FDA approved OXLUMO™ (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients. PH1 is an ultra-rare genetic disease characterized by oxalate overproduction.

In November 2020, Lumasiran was approved by the US FDA for the treatment of PH1 to lower urinary oxalate levels in pediatric and adult patients and by the EMA for the treatment of PH1 in all age groups. Lumasiran is marketed in the U.S. and EU as OXLUMO®. In October 2022, Alnylam Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) approved a label expansion for OXLUMO® (lumasiran), an RNAi therapeutic administered via subcutaneous injection, now indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate (UOx) and plasma oxalate (POx) levels in pediatric and adult patients. The approval is based on positive efficacy and safety results of the ILLUMINATE-C Phase III study of OXLUMO in patients with severe renal impairment, including those on hemodialysis.

Lumasiran has received both U.S. and EU Orphan Drug Designations, a Breakthrough Therapy Designation, Priority review and pediatric rare disease designation from the US Food and Drug Administration (FDA), and a Priority Medicines (PRIME) designation from the European Medicines Agency (EMA).

RNA Interference Therapy: Company and Product Profiles (Pipeline Therapies)

1. Company Overview: Alnylam Pharmaceuticals/Regeneron Pharmaceuticals

Alnylam has led the translation of RNAi (RNA interference) from Nobel Prize-winning discovery into an innovative, entirely new class of medicines. Founded in 2002 by a team of distinguished life sciences leaders, Alnylam’s vision is to harness the potential of RNAi therapeutics to transform the lives of people living with diseases for which there are limited or inadequate treatment options. The pioneering work of the company has delivered the world’s first and only approved RNAi therapeutics - ONPATTRO® (patisiran) in 2018, GIVLAARI® (givosiran) in 2019, OXLUMO® (lumasiran) in 2020, and AMVUTTRA® (vutrisiran) in 2022. It is advancing a deep pipeline of innovative RNAi-based medicines in four therapeutic areas: genetic medicines, cardio-metabolic diseases, infectious diseases, and central nervous system (CNS) and ocular diseases.

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 is composed of small-interfering RNAs (siRNAs) directed against terminal complement component 5 (C5) of the complement pathway conjugated to a N-acetylgalactosamine (GalNAc) ligand, which has potential use in the treatment of complement-mediated diseases, such as paroxysmal nocturnal hemoglobinuria (PNH). Upon subcutaneous administration of cemdisiran, the GalNAc ligand moiety specifically binds to and is taken up by the asialoglycoprotein receptor (ASGPR) expressed on hepatocytes.

Inside the cell, the siRNAs bind to C5 mRNAs, which results in the inhibition of both the translation and expression of the C5 protein. This lowers plasma C5 levels, prevents C5 cleavage into pro-inflammatory components and blocks complement-mediated hemolysis. C5, a complement pathway protein, is expressed at high levels by the liver. Currently, the drug is in Phase III stage of its clinical development for the treatment of Paroxysmal Nocturnal Hemoglobinuria, Age-related Macular Degeneration (AMD), Geographic Atrophy (GA) and Myasthenia Gravis.

In August 2023, The U.S. Food and Drug Administration had granted the combination therapy orphan drug status for the treatment of MG. Pozelimab is an antibody therapy that’s being tested in combination with the RNA-based therapy cemdisiran as a potential treatment for myasthenia gravis (MG). The therapies in combination aim to ease disease severity by completely blocking the activation of the complement system, a part of the immune system thought to be involved in MG-driving autoimmune attacks. The treatment combo is being explored by Regeneron Pharmaceuticals, to be given as once-monthly subcutaneous, or under-the-skin, injections.

2. Company Overview: Amgen

Amgen is one of the world’s leading innovators in biotechnology, known for using advanced science and genetics to develop medicines that address serious diseases. Founded in 1980, the company has built a strong reputation for pioneering biologics and leveraging cutting-edge research in areas such as oncology, inflammation, cardiovascular disease, and rare conditions. With a focus on translating complex scientific insights into effective therapies, Amgen continues to expand its global impact, improving patient outcomes through both breakthrough medicines and ongoing investment in research and development.

Product Description: Olpasiran

Olpasiran (formerly AMG 890) is a small interfering RNA (siRNA) that lowers lipoprotein(a), also known as Lp(a). It is being investigated for the treatment of atherosclerotic cardiovascular disease.

3. Company Overview: SanegeneBio

SanegeneBio is an innovative RNA-focused biotechnology company dedicated to developing next-generation therapeutics that target diseases with high unmet medical need. Leveraging advanced RNA interference and delivery technologies, the company focuses on creating precise, safe, and effective treatments capable of modulating gene expression at its source. With a strong scientific foundation and a commitment to translational research, it aims to accelerate the path from discovery to clinic, ultimately improving outcomes for patients worldwide.

Product Description: SGB-9768

SGB-9768 is an experimental RNAi therapeutic targeting complement C3 for the treatment of complement-mediated kidney diseases. In October 2025, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to SGB-9768 for the treatment of C3 Glomerulopathy (C3G). As a novel RNAi therapy, SGB-9768 may significantly improve the lives of C3G patients with a subcutaneously-administered safe and effective medicine that can be dosed as infrequently as twice yearly.

4. Company Overview: Alys Pharmaceuticals

Alys Pharmaceuticals is an emerging innovator in the life-sciences sector, focused on developing advanced therapies that address unmet medical needs in chronic and complex diseases. Known for its science-driven approach, the company leverages cutting-edge research platforms to create targeted, high-impact treatments with the potential to improve patient outcomes on a global scale. With a pipeline rooted in strong clinical evidence and strategic collaborations, Alys Pharmaceuticals aims to accelerate the transition from laboratory breakthroughs to accessible, real-world healthcare solutions.

Product Description: ALY 101

ALY-101 is the first clinical candidate from Alys’s pipeline of siRNA molecules developed for dermatological applications. ALY-101 is designed for local and selective modulation of inflammation and targets JAK1, a key driver of alopecia areata, with a JAK1-targeting siRNA-lipid conjugate. For the initial indication, it is formulated as an intradermal injectable. Alys is also developing a trans-epidermal formulation of ALY-101 for the treatment of indications such as atopic dermatitis and vitiligo, which should reach clinical stage in 2026.

RNA Interference Therapy Analytical Perspective

In-depth Commercial Assessment: RNA Interference Therapy 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 Interference Therapy Competitive Landscape

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

RNA Interference Therapy 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 Interference Therapy drugs?
• How many RNA Interference Therapy 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 Therapy?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the RNA Interference Therapy 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 Therapy and their status?:
• What are the key designations that have been granted to the emerging and approved drugs?

This product will be delivered within 4-6 business days.