Market Size
The Global RNAi Therapeutics Market will grow from USD 2.98 billion in 2025 to USD 8.80 billion by 2033, at a CAGR of 14.6% (2026–2033)
RNA therapeutics are a novel class of medications based on ribonucleic acid (RNA). RNA interference is a biological process in which target mRNA molecules are neutralized, and RNA molecules are observed to inhibit gene expression or translation. Furthermore, RNA interference (RNAi) is a collection of small RNA-directed mechanisms that result in sequential-specific inhibition of genetic expression.
Market Summary
| Metrics | Details |
| Market CAGR | 14.6% |
| Segments Covered | By Type, By Application, By Route of Administration, By End User, and By Region |
| Report Insights Covered | Competitive Landscape Analysis, Company Profile Analysis, Market Size, Share, Growth, Demand, Recent Developments, Mergers and acquisitions, New Product Launches, Growth Strategies, Revenue Analysis, and Other key insights. |
| Fastest Growing Region | Asia Pacific |
| Largest Market Share | North America |
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Market Dynamics
The global RNAi therapeutics market growth is driven by the increasing incidence of coronary artery diseases, cardiac ailments, and neurodegenerative and infectious diseases, increasing collaborative activities between drug distribution companies and contract research organizations (CROs).
Increasing collaborative activities boosting the growth of the RNAi therapeutics market
The increasing collaborative activities between drug distribution companies and contract research organizations (CROs) contribute to RNAi therapeutics market growth. On June 21, 2021, Arrowhead Pharmaceuticals Inc and Horizon Therapeutics plc announced a global collaboration and license agreement for ARO-XDH, a previously undisclosed discovery-stage investigational RNA interference (RNAi) therapeutic being developed by Arrowhead as a potential treatment for people with uncontrolled gout. This collaboration with Horizon also represents a further expansion of Arrowhead Pharmaceuticals' rapidly growing pipeline of investigational RNAi therapeutics that utilize Arrowhead’s proprietary Targeted RNAi Molecule (TRiMTM) platform.” In January 2016, Ionis Pharmaceuticals (previously Isis Pharmaceuticals) initiated a Phase-I study of ISIS-HBV-L Rx in collaboration with GSK Pharmaceuticals. This acquisition aims to the treatment of Hepatitis B viral infection. And also, one more collaboration by Ionis pharmaceuticals with Akcea Therapeutics has led to the approval of the world’s first antisense-based molecule “Tegsdi” by the FDA and the European Commission in 2018.
Moreover, despite these factors, it has the potential to grow due to increasing interest exhibited by the pharmaceutical industries for the commercialization of these therapies. Quark, Alnylam, and silence therapeutics are other major players in this market with a strong research pipeline in RNAi therapeutics. For instance, Silence therapeutics mRNAi GOLD platform is designed to deliver gene-silencing medicine to targeted liver cells in the body by combining siRNA molecules with chemical address tags called GaINAc, a naturally occurring sugar that attaches specifically to liver cells. These RNAi molecules can then enter liver cells and silence targeted; disease-associated genes expressed in the liver.
In May 2021, Silence therapeutics completed its phase 1 study of SLN124, which is a gene silencing therapy for treating rare iron-loading anemia conditions, thalassemia, and myelodysplastic syndrome (MDS) patients.
High investment and strict regulations are likely to hamper the market growth
In recent years, there has been a decline in FDA drug approval rates. Receiving FDA approval for a new drug has become extremely challenging. It approved less than half the number of new drugs in 2016 (19 so far) when compared to 2015 (45 total) and 2014 (41 total). Hence, despite the large investments, there has been a decline in innovative drugs manufactured. FDA explains manufacturing standards and other complying issues as the major reasons for this declining trend. This can impede the growth of the RNAi therapeutics, especially since the miRNAs and siRNAs fall into the relatively new field of genetic medicine, wherein they may require more intensified clinical trials. The highly extensive clinical trials effectively result in low approval rates of drugs. This would mean that the stringent guidelines will be a major restraint on the growth of the market.
Market Segmentation Analysis
The oncology segment is expected to hold the largest share in this market segment
According to the World Health Organization, cancer is the second leading cause of death globally and responsible for 9.6 million deaths in 2018. Globally, about 1 in 6 deaths is due to cancer. The number of new cases is expected to increase by about 70% over the next two decades. Also, recent advancements, such as the development of small interfering RNA (siRNA) tolerant to nucleases and non-viral vectors, such as cationic liposomes and nanoparticles, can overcome this obstacle and facilitate the clinical use of RNAi-based therapeutics in the treatment of cancer.
Substantial pipeline for cancer therapies by companies and institutes such as Enzon Pharmaceuticals (Santaris Pharma), University of Texas, OncoGenex, Isarna Therapeutics, AstraZeneca (Ionis Pharmaceuticals), and INSYS Therapeutics, Inc. are expected to drive the market. In addition, many companies have invested in R&D for nanocarriers to deliver oligonucleotides for cancer treatment, which is expected to contribute to the oncology segment.
Market Geographical Share
North America region holds the largest market share of the global RNAi therapeutics market
The U.S. has several RNAi therapies that are in the developmental pipeline. Many biotechnology companies have invested heavily in RNAi therapeutic development. Many pharmaceutical developers have entered into cooperation agreements or licensing agreements with many small companies to capitalize on the expected growth in revenue during the forecast period of this market. For example, AstraZeneca’s deal with Ionis Pharmaceuticals is majorly investing heavily in RNA-interference technology. Also, in September 2018, Delos Capital developed MRNA-based drugs for chemotherapy-resistant cancer. Curamir Therapeutics received initial financial support. With the help of these new companies, acquisitions, and new inventions, North America is dominating the market.
Companies and Competitive Landscape
The global RNAi therapeutics market is highly competitive with the presence of local as well as global companies. Some of the key players which are contributing to the growth of the market include Alnylam Pharmaceuticals, Inc, Dicerna Pharmaceuticals, Silence Therapeutics, Arrowhead Pharmaceuticals, Inc, Sanofi, GlaxoSmithKline Plc, Arbutus Biopharma Corporation, Benitec Biopharma, Thermo Fisher Scientific Inc and Sirnaomics, Inc. The major players are adopting several growth strategies such as product launches, acquisitions, and collaborations, which are contributing to the growth of the market globally. For instance, on April 8, 2019, Regeneron Pharmaceuticals, Inc and Alnylam Pharmaceuticals, Inc 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. The collaboration will leverage both companies' scientific and technological expertise and will build on Alnylam's recent preclinical data showing potent and highly durable delivery of RNAi therapeutics to achieve target gene silencing in the eye and CNS.
Alnylam Pharmaceuticals, Inc
Overview: Alnylam Pharmaceuticals Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing RNA interference (RNAi) therapeutics for genetically defined diseases. The company was founded in 2002 and is headquartered in Cambridge, Massachusetts. It operates in 19 countries with more than 1K employees. It conducts its operations in RNAi (RNA interference) from Nobel Prize-winning discovery into an entirely new class of medicines. In 2018, its first medicine, ONPATTRO (patisiran), became the world’s first approved RNAi therapeutic. They are advancing a robust 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 Portfolio: It includes products like ONPATTRO (patisiran), GIVLAARI (givosiran) and OXLUMO (lumasiran).
Recent Developments:
- May 2026: Alnylam Pharmaceuticals advanced next-generation RNAi pipeline programs targeting cardiometabolic and rare genetic diseases, strengthening its leadership in siRNA-based therapeutic innovation and expanding late-stage clinical assets.
- April 2026: Ionis Pharmaceuticals reported progress in expanded RNA-targeting collaborations focused on neurological and cardiovascular indications, accelerating commercialization pathways for its RNA-based medicine portfolio.
- March 2026: Dicerna Pharmaceuticals integration efforts supported broader RNA interference therapeutic expansion within metabolic disease programs, enhancing pipeline synergy and delivery-platform optimization.
- February 2026: Arrowhead Pharmaceuticals advanced its TRiM™ platform-based RNA interference candidates, strengthening targeted gene-silencing efficiency and enabling broader indications in liver and pulmonary diseases.
- January 2026: Silence Therapeutics expanded clinical development of siRNA therapies using mRNAi GOLD™ platform, focusing on hematologic and cardiovascular disorders with improved precision delivery.
- December 2025: Novartis increased strategic investments in RNAi-based drug discovery partnerships, reinforcing its rare-disease pipeline and strengthening next-generation gene-silencing capabilities.
- November 2025: Roche expanded RNA-targeted therapeutic collaborations with biotech firms to accelerate development of precision RNAi medicines for oncology and neurological diseases.
- October 2025: Regeneron Pharmaceuticals enhanced genetic medicine programs incorporating RNA interference technologies to support long-duration gene-silencing treatments for chronic diseases.
- September 2025: Pfizer strengthened RNA-based therapeutic research initiatives focusing on liver-targeted RNAi platforms and next-generation delivery systems for systemic diseases.
- August 2025: Moderna expanded beyond mRNA vaccines into RNA therapeutics research collaborations, exploring RNA interference synergies and multi-platform genetic medicine strategies.
- July 2025: Sanofi accelerated RNA-targeted drug development partnerships to diversify its rare-disease pipeline and strengthen long-term investment in RNA-based precision medicine.
