Table of Contents
Overview
New York, NY – June 24, 2025 – Global Antisense and RNAi Therapeutics Market size is expected to be worth around US$ 28.6 billion by 2034 from US$ 5.2 billion in 2024, growing at a CAGR of 18.6% during the forecast period 2025 to 2034. In 2024, North America led the market, achieving over 43.2% share with a revenue of US$ 1.0 Billion.
The Antisense and RNA Interference (RNAi) Therapeutics market is witnessing significant growth, driven by rising demand for precision-targeted treatments and growing research into rare and genetic disorders. These therapies, which operate by silencing specific disease-causing genes at the mRNA level, are gaining traction as safer and more effective alternatives to traditional small-molecule drugs.
Antisense oligonucleotides (ASOs) and RNAi-based agents such as small interfering RNAs (siRNAs) are increasingly used to treat conditions like spinal muscular atrophy, hereditary transthyretin amyloidosis, and certain cancers. Key regulatory approvals, such as the FDA’s clearance of ONPATTRO (patisiran) and SPINRAZA (nusinersen), have validated the clinical utility of RNA-based therapies.
Ongoing advancements in delivery technologies, including lipid nanoparticles and GalNAc conjugates, are addressing past limitations related to stability and tissue targeting. This has expanded the pipeline, with over 100 RNA-based candidates currently in various stages of clinical development.
North America leads the market due to strong R&D infrastructure and early adoption, while Asia-Pacific is expected to witness robust growth through government-supported biotechnology initiatives. As healthcare systems increasingly focus on personalized medicine, antisense and RNAi therapeutics are poised to transform chronic and genetic disease management through highly specific and minimally invasive interventions.
Key Takeaways
- In 2024, the global market for antisense and RNAi therapeutics was valued at approximately USD 5.2 billion and is projected to grow at a compound annual growth rate (CAGR) of 18.6%, reaching an estimated USD 28.6 billion by 2033.
- Based on technology, the market is categorized into RNA interference (RNAi) and antisense RNA. Among these, antisense RNA emerged as the leading segment in 2023, accounting for 54.3% of the total market share, driven by its clinical maturity and regulatory approvals.
- In terms of application, the market is segmented into genetic diseases, cancer, skin disorders, respiratory disorders, ocular disorders, neurodegenerative disorders, infectious diseases, and cardiometabolic & renal disorders. Of these, neurodegenerative disorders represented the largest application segment, commanding a significant 43.5% market share, owing to the rising prevalence and the targeted therapeutic benefits of RNA-based modalities.
- Regarding the route of administration, the market is divided into intravenous, subcutaneous, intrathecal injections, and others. The intrathecal route dominated the segment in 2023, contributing to 53.8% of global revenue, largely due to its effectiveness in delivering drugs directly to the central nervous system.
- Geographically, North America led the global market, securing a 43.2% share in 2023, supported by advanced biotechnology infrastructure, early product approvals, and growing clinical investments in RNA-based therapies.
Segmentation Analysis
- Technology Analysis: In 2023, the antisense RNA segment dominated the market with a 54.3% share, attributed to its longer clinical history and the greater number of approved drugs compared to RNAi therapies. Antisense oligonucleotides (ASOs) have proven effective in treating diseases like spinal muscular atrophy and hereditary transthyretin amyloidosis. Established manufacturing platforms and reliable delivery mechanisms have further supported their market lead. However, RNAi technologies are gaining momentum, driven by advancements in siRNA delivery and superior gene-silencing capabilities.
- Application Analysis: Neurodegenerative disorders accounted for 43.5% of the total application share in 2023, driven by the critical need for effective therapies and the ability of antisense and RNAi drugs to target disease-causing genes. These therapies have shown encouraging results in conditions such as Huntington’s disease and ALS. Rising global aging populations and increasing investment in targeted neurological treatments are expected to sustain high demand for RNA-based approaches in managing chronic neurodegenerative diseases.
- Route of Administration Analysis: Intrathecal injections led the administration segment in 2023, holding a 53.8% revenue share. This dominance is due to their effectiveness in bypassing the blood-brain barrier, enabling direct delivery to the central nervous system. Such precision is especially critical for treating disorders like spinal muscular atrophy. Despite its invasive nature, intrathecal delivery remains the most efficient method for CNS-targeted RNA therapies. Research into alternative, less invasive routes is ongoing and may influence future market trends.
Market Segments
Technology
- RNA Interference
- Antisense RNA
Application
- Genetic Disease
- Cancer
- Skin Disorders
- Respiratory Disorders
- Ocular Disorders
- Neurodegenerative Disorders
- Infectious Disease
- Cardiometabolic & Renal Disorders
Route of Administration
- Intravenous Injections
- Subcutaneous Injections
- Intrathecal Injections
- Others
Regional Analysis
North America held the largest share in the antisense and RNAi therapeutics market in 2023, accounting for 43.2% of global revenue. This dominance is supported by high healthcare spending—reaching USD 4.5 trillion in 2022, as reported by the Centers for Medicare & Medicaid Services (CMS). The region benefits from advanced research infrastructure, strong regulatory support from the FDA, and active government funding. According to the National Institutes of Health (NIH), 2023 saw sustained investment in nucleic acid therapeutics research. Additionally, early adoption of advanced treatments for genetic disorders and cancer has further fueled market expansion.
Meanwhile, the Asia Pacific region is projected to register the fastest compound annual growth rate (CAGR) during the forecast period. Factors such as rising healthcare expenditure, increasing biotechnology investments, and growing awareness of rare disease management are key growth drivers. The World Bank noted steady growth in healthcare spending across East Asia and the Pacific. Regulatory reforms in countries like China, led by the National Medical Products Administration (NMPA), are also accelerating drug development and approval processes, thereby strengthening the region’s position in the global RNA therapeutics landscape.
Emerging Trends
- Rising FDA Approvals of Nucleic Acid Drugs: The pace of regulatory approvals has accelerated, with antisense and RNAi therapies now validated for clinical use. In April 2023, the FDA granted accelerated approval to tofersen (QALSODY) for SOD1-mutant amyotrophic lateral sclerosis, marking the first antisense treatment for this indication. Shortly thereafter, in 2023, vutrisiran received approval for hereditary transthyretin-mediated amyloidosis, delivered quarterly by subcutaneous injection. These milestones underscore growing confidence in oligonucleotide modalities.
- Advanced Chemical Modifications and Targeted Delivery: Significant improvements in oligonucleotide chemistry have been implemented to boost drug stability and tissue targeting. Conjugation of small interfering RNAs to N-acetylgalactosamine (GalNAc) ligands enables precise delivery to liver cells, enhancing potency at lower doses. Locked nucleic acid (LNA) and constrained ethyl (cEt) gapmer chemistries are now widely used to resist nuclease degradation and improve binding affinity. Such advances are propelling second-generation ASOs and siRNAs toward broader therapeutic applications.
- Personalized and N-of-1 Antisense Therapies: The antisense platform has demonstrated the flexibility to create individualized treatments. A landmark case is milasen, a custom-designed ASO for a single child with Batten disease, which was developed and granted emergency use within months. This approach highlights the potential to tailor oligonucleotide drugs to rare genetic mutations on a per-patient basis. Such agility is expected to spur bespoke therapies for ultra-rare disorders, leveraging rapid design–testing cycles.
- Expansion of Late-Stage Clinical Pipeline: The clinical development pipeline has broadened to encompass diverse indications, with multiple candidates in pivotal trials. At least four phase III studies are underway, including tofersen in ALS and inclisiran for familial hypercholesterolemia, the latter enrolling 482 patients (242 treated vs. 240 placebo) and demonstrating a 39.7% LDL-cholesterol reduction at 16 months. This late-stage momentum reflects growing validation of oligonucleotide therapies across neurology, cardiometabolic, and rare disease areas.
Use Cases
- Givosiran in Acute Hepatic Porphyria: Givosiran, an siRNA conjugated to GalNAc, targets ALAS1 mRNA in the liver to reduce toxic metabolite build-up. In a study of 94 patients, 74% of those treated experienced a reduction in porphyria attacks, with monthly dosing proving effective and well tolerated. Liver and kidney function monitoring is recommended during therapy.
- Inotersen for Hereditary Transthyretin Amyloidosis: Inotersen, a 2′-O-MOE-modified ASO, inhibits transthyretin (TTR) protein synthesis. In a randomized, double-blind phase III trial, 112 treated patients were compared with 60 placebo controls over 66 weeks. Significant improvements were observed in neuropathy impairment scores, confirming clinical benefit for patients with polyneuropathy due to TTR mutations.
- Fitusiran in Hemophilia A and B: Fitusiran is an siRNA that silences antithrombin mRNA in the liver to rebalance clotting. In a phase II trial of 25 hemophilia patients, monthly administration achieved an 81% reduction in antithrombin mRNA levels. Annualized bleed rate (ABR) decreased from 12 events per year at baseline to 1.7 events after 13 months, demonstrating robust bleeding control.
- Inclisiran for Heterozygous Familial Hypercholesterolemia: Inclisiran, an siRNA targeting PCSK9 mRNA, is administered only twice yearly. In a pivotal phase III trial with 482 participants (242 inclisiran vs. 240 placebo), LDL-cholesterol levels fell by 39.7% in the treatment arm at 16 months, whereas placebo saw an 8.2% increase. Approximately 65% of treated patients achieved LDL levels below 100 mg/dL, confirming durability and convenience of dosing.
Conclusion
The global antisense and RNAi therapeutics market is poised for transformative growth, driven by increasing demand for precision medicine, regulatory advancements, and strong clinical validation. With a projected CAGR of 18.6%, the market is expected to reach USD 28.6 billion by 2033.
Key innovations in delivery technologies and personalized therapeutics are expanding treatment potential across rare and chronic diseases. North America remains the market leader, while Asia Pacific is emerging rapidly. As RNA-based drugs continue to gain regulatory support and clinical success, these therapies are expected to play a central role in the future of targeted and individualized medical care.
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