Global RNA Therapeutics Market Outlook, 2031

The global RNA therapeutics industry covers medicines that use or target ribonucleic acid to treat or prevent disease, including small interfering RNA drugs that silence disease-causing genes, antisense oligonucleotides that modify RNA processing or reduce protein production, messenger RNA vaccines and therapeutics that instruct cells to make proteins, RNA aptamers, microRNA-targeting drugs, circular RNA and self-amplifying RNA, and RNA editing approaches. Approved products include Alnylam's Onpattro, Givlaari, Oxlumo, Leqvio with Novartis and Amvuttra; Ionis's Spinraza with Biogen, Wainua with AstraZeneca, Qalsody with Biogen, Tryngolza and Dawnzera; Arrowhead's Redemplo; Sanofi's Qfitlia; Sarepta's exon-skipping drugs; Novo Nordisk's Rivfloza; and mRNA vaccines from Moderna and Pfizer and BioNTech. Other companies include Arrowhead, Avidity Biosciences, acquired by Novartis, Dyne, Wave Life Sciences, Silence Therapeutics, Arcturus, CureVac, which BioNTech agreed to acquire in 2025, Orna Therapeutics, acquired by Eli Lilly, and many Chinese developers. RNA medicines are designed from genetic sequence information, allowing rapid development, and delivery technologies, including GalNAc conjugates for the liver and lipid nanoparticles, determine where they act. RNA therapeutics expanded from rare diseases into common conditions and faced a shift in the mRNA vaccine market. siRNA and antisense drugs reached larger populations: Alnylam's Amvuttra was approved in March 2025 for transthyretin amyloid cardiomyopathy, a common cause of heart failure, and became a major growth driver; Novartis's twice-yearly Leqvio lowers LDL cholesterol; Ionis's olezarsen, Tryngolza, was approved in December 2024 and Arrowhead's plozasiran, Redemplo, in November 2025 for familial chylomicronaemia syndrome, with trials in severe hypertriglyceridaemia; Sanofi's fitusiran, Qfitlia, was approved for haemophilia in March 2025; and Ionis's donidalorsen, Dawnzera, for hereditary angioedema in August 2025. Late-stage programmes target hypertension with zilebesiran, lipoprotein(a) with pelacarsen, olpasiran and lepodisiran, and Alzheimer's disease. Alnylam launched its Alnylam 2030 strategy in January 2026. In contrast, demand for mRNA COVID-19 vaccines fell sharply after 2022, and US policy became less supportive: in August 2025 the Department of Health and Human Services cancelled about USD 500 million of BARDA mRNA vaccine projects, and federal vaccine advisers narrowed COVID-19 vaccine recommendations. mRNA companies have pivoted toward cancer vaccines, rare diseases and in vivo cell therapy, and large pharmaceutical companies have invested in RNA platforms, including Novartis's agreement to acquire Avidity for about USD 12 billion in October 2025 and Lilly's acquisition of Orna in February 2026 for circular RNA and in vivo CAR-T. Market Dynamics Market Drivers • Ability to Target Previously Undruggable GenesRNA therapeutics can silence or modify almost any gene by matching its sequence, allowing treatment of diseases caused by proteins that small molecules and antibodies cannot target. This has produced effective treatments for genetic diseases such as transthyretin amyloidosis, spinal muscular atrophy, primary hyperoxaluria and haemophilia, and it is expanding to common diseases such as high cholesterol, hypertension and lipoprotein(a)-driven cardiovascular disease. • Infrequent Dosing and Platform EfficiencysiRNA drugs with GalNAc delivery can be given every three to six months, and some candidates aim for once or twice yearly dosing, improving adherence in chronic diseases. Because RNA drugs share chemistry and delivery platforms, new candidates can be designed and developed quickly, reducing discovery time and cost. Validated platforms attract partnerships and acquisitions from large pharmaceutical companies. Market Challenges • Delivery Beyond the LiverMost approved siRNA and many antisense drugs act in the liver, where GalNAc conjugates deliver them efficiently. Delivering RNA drugs to muscle, heart, brain, kidney, lungs and tumours is more difficult, and companies are developing antibody-oligonucleotide conjugates, new ligands and lipid nanoparticles to reach these tissues. Intrathecal injection is required for some brain-targeted drugs, limiting convenience. • mRNA Vaccine Demand Decline and Policy UncertaintySales of mRNA COVID-19 vaccines fell sharply after 2022, leading Moderna, BioNTech and others to cut costs and refocus. US policy changes in 2025, including the cancellation of BARDA mRNA vaccine projects and narrower vaccine recommendations, increased uncertainty for mRNA vaccine development. Safety concerns, reimbursement for high-priced rare disease drugs and competition from gene editing also affect the market. Market Trends • RNA Medicines for Common Cardiovascular and Metabolic DiseasessiRNA and antisense drugs are moving into large markets, including LDL cholesterol lowering with inclisiran, lipoprotein(a) reduction with pelacarsen, olpasiran, lepodisiran and zerlasiran, hypertension with zilebesiran, severe hypertriglyceridaemia with plozasiran and olezarsen, and transthyretin amyloid cardiomyopathy with vutrisiran and nucresiran. Outcome trials under way could establish RNA medicines as mainstream cardiovascular treatments. Obesity and metabolic targets are also being explored. • Extrahepatic Delivery, mRNA Therapeutics and In Vivo Cell EngineeringCompanies are extending RNA medicines to muscle, heart, central nervous system and adipose tissue through antibody-oligonucleotide conjugates, as developed by Avidity and Dyne, and new targeting ligands. mRNA technology is being applied to personalised cancer vaccines, protein replacement for rare diseases and in vivo CAR-T cell therapy using lipid nanoparticles, as pursued by Orna, Capstan and others, while RNA editing offers a way to correct mutations without altering DNA. Segment Analysis siRNA and antisense oligonucleotide therapeutics account for the largest share of RNA therapeutics revenue excluding COVID-19 vaccines, while mRNA products remain large but have declined from their pandemic peak. Small interfering RNA drugs, including patisiran, vutrisiran, givosiran, lumasiran, inclisiran, nedosiran, fitusiran and plozasiran, silence target genes through the RNA interference pathway and have become the fastest-growing segment, led by Amvuttra's expansion into cardiomyopathy. Antisense oligonucleotides, including nusinersen, inotersen, eplontersen, tofersen, olezarsen, donidalorsen and exon-skipping drugs for Duchenne muscular dystrophy, bind RNA to reduce protein production or change splicing, and they are important in neurology, rare diseases and cardiometabolic disease. Messenger RNA products, including COVID-19 vaccines from Moderna and Pfizer and BioNTech, RSV vaccines and investigational cancer vaccines and protein replacement therapies, generated very large revenues in 2021 and 2022 but have since declined. RNA aptamers, such as avacincaptad pegol for geographic atrophy, bind proteins like antibodies. Antibody-oligonucleotide conjugates, such as those developed by Avidity and Dyne, target muscle diseases. Self-amplifying and circular RNA are newer mRNA technologies offering longer or stronger expression. MicroRNA-targeting drugs and RNA editing therapies are in earlier development. Rare genetic diseases account for most approved RNA therapeutics, while cardiovascular and metabolic diseases, infectious diseases and oncology represent major and growing application areas. Rare genetic diseases, including hereditary transthyretin amyloidosis with polyneuropathy, spinal muscular atrophy, Duchenne muscular dystrophy, acute hepatic porphyria, primary hyperoxaluria, SOD1 amyotrophic lateral sclerosis, familial chylomicronaemia syndrome, hereditary angioedema and haemophilia, account for most approved RNA therapeutics. Cardiovascular and metabolic diseases, including transthyretin amyloid cardiomyopathy, high LDL cholesterol, lipoprotein(a), hypertension and severe hypertriglyceridaemia, have become the largest growth opportunity, with Amvuttra and Leqvio already large and several outcome trials under way. Infectious diseases, including COVID-19, RSV and influenza vaccines and investigational siRNA treatments for hepatitis B and D, form a major segment. Oncology includes personalised mRNA cancer vaccines, such as Moderna and Merck's intismeran autogene in melanoma, and RNA drugs targeting cancer genes. Neurological diseases, including Huntington's disease, Alzheimer's disease, Parkinson's disease and ALS, are targeted by antisense and siRNA drugs delivered to the central nervous system. Ophthalmic diseases, kidney diseases and muscle diseases are further areas. Subcutaneous injection is the most common route for siRNA and many antisense drugs, while intramuscular, intravenous and intrathecal routes are used for vaccines and specific therapies. Subcutaneous injection is used for most GalNAc-conjugated siRNA drugs, including vutrisiran, inclisiran, givosiran, lumasiran, fitusiran and plozasiran, and for antisense drugs such as eplontersen, olezarsen and donidalorsen, often at intervals of one to six months, and some can be self-administered at home. Intramuscular injection is used for mRNA vaccines against COVID-19, RSV and influenza, and for investigational cancer vaccines. Intravenous infusion is used for lipid nanoparticle-delivered drugs such as patisiran, for exon-skipping drugs in Duchenne muscular dystrophy, for antibody-oligonucleotide conjugates and for some mRNA therapeutics and in vivo CAR-T products. Intrathecal injection into the spinal fluid is used for nusinersen and tofersen to reach the central nervous system. Intravitreal injection is used for ophthalmic RNA drugs such as aptamers. Inhaled and oral delivery of RNA drugs are research goals. Delivery technologies, including GalNAc conjugates, lipid nanoparticles, antibody conjugates and new peptide and lipid ligands, determine which tissues RNA drugs reach. Hospitals and specialty clinics are the main settings for administering many RNA therapeutics, while specialty pharmacies, retail pharmacies and vaccination sites are important distribution channels. Hospitals and specialty clinics, including neurology, cardiology, haematology and rare disease centres, administer intrathecal, intravenous and physician-administered RNA therapies and identify eligible patients through genetic testing and specialist diagnosis. Cardiology practices are becoming important as RNA drugs for amyloid cardiomyopathy and lipid disorders expand, and inclisiran is given by healthcare professionals twice a year. Specialty pharmacies distribute high-cost self-administered or home-administered RNA drugs for rare diseases, handling reimbursement and patient support. Retail pharmacies and vaccination clinics administer mRNA vaccines, alongside public health programmes and physician offices. Home infusion and home nursing services deliver some therapies. Government immunisation programmes and national procurement buy vaccines. Contract development and manufacturing organisations produce oligonucleotides and mRNA, and oligonucleotide manufacturing capacity has expanded to meet demand for cardiovascular RNA drugs. Genetic testing laboratories and diagnostic companies play a key role in identifying patients. Regional Analysis North America leads the RNA therapeutics market because of high use of rare disease RNA drugs, early approvals and the concentration of RNA therapeutics companies, while Europe is a major market and Asia-Pacific is the fastest-growing region. North America, led by the United States, accounts for the largest share of RNA therapeutics revenue. Most RNA therapeutics companies, including Alnylam, Ionis, Arrowhead, Moderna, Dyne, Wave and Avidity, are based in the United States, and the FDA approved several RNA drugs between 2024 and 2025, including Amvuttra for cardiomyopathy, Qfitlia, Tryngolza, Dawnzera and Redemplo. US policy changes in 2025 reduced federal support for mRNA vaccine research, while cardiovascular and rare disease RNA drugs grew strongly. Canada has a smaller market. Europe is a major market, with strong uptake of RNA drugs for rare diseases and Leqvio, companies such as BioNTech, CureVac, Silence Therapeutics and Novartis, and health technology assessment affecting access to high-cost drugs. Asia-Pacific is the fastest-growing region, with rising use of RNA drugs in Japan, where several have been approved, and in China, where domestic companies are developing siRNA drugs for hepatitis B, lipids and hypertension and licensing them to global companies, and mRNA manufacturing has expanded. South Korea, Australia and Singapore have growing RNA research and manufacturing capabilities. India has expanded mRNA vaccine development. Latin America, the Middle East and Africa have limited access to high-cost RNA drugs but participated in mRNA vaccine distribution, and the WHO mRNA technology transfer hub in South Africa aims to build regional capacity. Key Developments February 2026: Lilly agreed to acquire Orna for up to USD 2.4 billion, gaining circular RNA and lipid nanoparticle technology for in vivo CAR-T and other RNA medicines. January 2026: Alnylam set out its strategy to expand RNAi therapeutics into larger cardiovascular, neurological and metabolic diseases, including phase 3 programmes for nucresiran and zilebesiran. November 2025: Arrowhead's siRNA therapy was approved to lower triglycerides in adults with familial chylomicronaemia syndrome, with trials ongoing in severe hypertriglyceridaemia. October 2025: Novartis agreed to acquire Avidity for about USD 12 billion to gain antibody-oligonucleotide conjugates for rare neuromuscular diseases. August 2025: The US Department of Health and Human Services cancelled about USD 500 million of BARDA-funded mRNA vaccine development projects. March 2025: Alnylam's vutrisiran was approved to treat the cardiomyopathy of wild-type or hereditary transthyretin amyloidosis, expanding RNAi therapy into a large heart failure population. Considered in this report • Historic Year: 2020 • Base Year: 2025 • Estimated Year: 2026 • Forecast Year: 2031 Aspects covered in this report • Global RNA Therapeutics Market with its value and forecast along with its segments • Various drivers and challenges • Ongoing trends and developments • Top profiled companies • Strategic recommendation By Molecule Type • siRNA • Antisense Oligonucleotides • mRNA • RNA Aptamers • Antibody-Oligonucleotide Conjugates • Other RNA Modalities By Application • Rare Genetic Diseases • Cardiovascular and Metabolic Diseases • Infectious Diseases • Oncology • Neurological Diseases • Other Diseases By Route of Administration • Subcutaneous • Intramuscular • Intravenous • Intrathecal and Other By End User • Hospitals and Specialty Clinics • Specialty Pharmacies • Retail Pharmacies and Vaccination Sites • Government Immunisation Programmes

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Sikandar Kesari

Research Analyst

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