Antisense And RNAi Therapeutics Market- Global Industry Size, Share, Trends, Opportunity, and Forecast, Segmented By Technology (RNA Interference, Antisense RNA), By Application (Ocular, Cardiometabolic & Renal Disorders, Cancer, Respiratory Disorders, Skin Diseases, Neurodegenerative Disorders, Genetic Disorders, Infectious Diseases), By Route of Administration (Intravenous Injections, Intrathecal Injections, Subcutaneous Injections, Other), By Region and Competition, 2020-2030F

Market Overview

The Global Antisense And RNAi Therapeutics Market, valued at USD 5.20 Billion in 2024, is projected to experience a CAGR of 19.19% to reach USD 14.91 Billion by 2030. Antisense and RNAi therapeutics represent a molecular medicine class that precisely modulates gene expression by either inhibiting protein production via antisense oligonucleotides or degrading messenger RNA through RNA interference, offering targeted interventions for various diseases. The market's expansion is fundamentally driven by the rising global incidence of genetic and chronic disorders, alongside a burgeoning understanding of intricate molecular disease mechanisms enabling highly specific therapeutic design. Growing investments in research and development further propel this growth by expanding the addressable therapeutic landscape for conditions with limited treatment options.

A notable challenge potentially impeding market expansion remains the persistent complexity in achieving efficient and precisely targeted *in vivo* delivery of these nucleic acid-based molecules to specific cells and tissues, which is critical for optimal therapeutic outcomes. According to the American Society of Gene & Cell Therapy, in the first quarter of 2023, companies in the US signed 110 deals to promote advancements in gene and RNA therapies, indicating significant investment and activity within this therapeutic space.

Key Market Drivers

The global Antisense and RNAi Therapeutics Market is significantly influenced by the increasing burden of genetic and chronic diseases, which establishes a profound unmet medical need for innovative treatments. These therapies offer precise modulation of gene expression, addressing the root causes of many disorders where conventional treatments fall short. The substantial patient population suffering from these conditions drives consistent demand for novel therapeutic avenues. According to the World Health Organization, in February 2025, "Rare diseases: a global health priority for equity and inclusion," over 7,000 known rare diseases collectively impact more than 300 million people globally, with 70% of these conditions starting in childhood. This widespread impact underscores the critical need for advanced therapeutic solutions like antisense and RNAi.

Concurrent advancements in drug delivery and nucleic acid technologies represent another pivotal market driver, enabling the effective and safe deployment of these complex molecular medicines. Historically, challenges in delivering nucleic acids to specific cells and tissues limited their therapeutic potential. Ongoing innovations in carrier systems, such as lipid nanoparticles and targeted conjugates, are enhancing stability, improving cellular uptake, and expanding the anatomical reach of these therapies beyond traditionally accessible organs. According to BioPharma Dive, in November 2024, "An RNAi renaissance is creating a new generation of startups," new techniques for delivering RNAi drugs to tissues beyond the liver were creating opportunities, with Alnylam Pharmaceuticals having revealed early evidence in 2023 of getting an RNAi drug into the brain. Such technological progress is crucial for unlocking the full therapeutic spectrum of antisense and RNAi drugs, facilitating their application across a broader array of diseases. The commercial success of these modalities is evident; according to the article "RNA Therapeutics 2024: From Pandemic Pivots to Precision Powerhouses," published in April 2025, Novartis' LEQVIO® (inclisiran) generated $754 million in 2024, demonstrating the commercial viability of RNAi therapies.

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Key Market Challenges

The persistent complexity in achieving efficient and precisely targeted *in vivo* delivery of nucleic acid-based molecules to specific cells and tissues directly impedes the growth of the Global Antisense and RNAi Therapeutics Market. This challenge arises from the inherent properties of these molecules, such as their size, negative charge, and susceptibility to enzymatic degradation, which hinder their ability to cross biological barriers and reach their intracellular targets in sufficient concentrations. Consequently, the development of novel therapies is prolonged due to the extensive research required for suitable delivery vehicles, increasing overall development costs and time to market.

This fundamental hurdle restricts the widespread application of antisense and RNAi therapeutics, confining many investigational and approved agents to conditions where local administration or established, albeit limited, delivery strategies are viable. According to the American Society of Gene and Cell Therapy, in 2024, non-viral delivery methods, vital for many RNAi and antisense oligonucleotides, constituted 8% of all abstracts presented at their annual meeting, exhibiting an 80% increase in submissions over the previous year. This significant and growing research emphasis highlights the ongoing challenge in optimizing targeted delivery, preventing these advanced therapies from fully addressing numerous diseases that require systemic distribution or targeting of difficult-to-access organs.

Key Market Trends

The global Antisense and RNAi Therapeutics Market is experiencing significant shifts driven by strategic moves into more prevalent disease areas and a notable increase in clinical development activities. These trends underscore the maturation of the technology and a robust future growth trajectory.

Antisense and RNAi therapeutics are increasingly expanding into broader therapeutic areas beyond rare diseases. This strategic diversification involves targeting more prevalent chronic conditions such as cardiovascular, metabolic, and neurological disorders, indicating a growing confidence in the technology's applicability to larger patient populations. According to Novotech CRO's "RNA Therapeutics Clinical Trials Whitepaper" published in August 2024, the Asia-Pacific region demonstrated a compound annual growth rate exceeding 26% in RNA therapy trials from 2019 to 2023, encompassing diverse therapeutic areas like oncology, metabolic disorders, and cardiovascular conditions. This expansion is driven by enhanced understanding of disease mechanisms and improvements in targeted delivery systems. For example, Alnylam Pharmaceuticals, during its February 2025 R&D Day, revealed advancements in ALN-4324, an investigational RNAi therapeutic for type 2 diabetes, a widespread metabolic disorder, alongside updates on programs targeting obesity, further illustrating this strategic pivot.

A second significant trend is the increasing clinical trial activity and pipeline expansion across the Antisense and RNAi Therapeutics Market. This surge in development reflects substantial investment and growing confidence in the therapeutic potential of these modalities. A burgeoning pipeline signifies a healthy innovation ecosystem and anticipates future market growth across numerous indications. As of January 31, 2024, an Avalere Health Advisory reported that at least 131 RNA-based therapies, encompassing antisense oligonucleotides and RNA interference therapies, were actively being studied in clinical trials. This robust pipeline indicates a sustained focus on bringing novel treatments to market. In a notable development, Ionis Pharmaceuticals announced in January 2024 that its late-stage pipeline had expanded to include nine medicines in Phase 3 trials for 11 potential indications, highlighting significant advancement in its therapeutic portfolio and demonstrating the industry's commitment to clinical progress.

Segmental Insights

The RNA Interference (RNAi) segment is experiencing rapid growth within the global antisense and RNAi therapeutics market. This accelerated expansion is largely propelled by significant advancements in siRNA delivery technologies, which bolster the potent gene silencing capabilities inherent to RNAi therapeutics. These innovations facilitate more precise targeting of RNA molecules to specific cells, enhancing therapeutic efficacy across a broader range of conditions including viral infections, cancer, and various genetic disorders. Moreover, increasing regulatory approvals from bodies such as the US Food and Drug Administration, alongside substantial investments in research and development, are strengthening market confidence and expanding access to these innovative treatments.

Regional Insights

North America leads the global Antisense and RNAi Therapeutics Market due to a confluence of supportive factors. The region benefits from a robust ecosystem comprising a significant presence of prominent biopharmaceutical companies and advanced research institutions. These entities consistently drive substantial investments in research and development, fostering innovation in RNAi-based therapeutic solutions. Furthermore, a favorable regulatory framework, notably spearheaded by the U. S. Food and Drug Administration, actively supports and streamlines the approval processes for novel antisense and RNAi drugs, accelerating market access for these advanced treatments. This strong regulatory backing, combined with sophisticated healthcare infrastructure and a focus on advanced therapies for various diseases, solidifies North America's dominant position.

Recent Developments

• In June 2025, Dyne Therapeutics announced that its investigational therapy, DYNE-101, received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for the treatment of myotonic dystrophy type 1 (DM1). DYNE-101 is an antisense oligonucleotide (ASO) conjugated to an antibody, designed to promote functional improvement in individuals with DM1 by reducing toxic RNA. This designation signifies a notable advancement in the development of targeted antisense therapeutics for genetically driven neuromuscular diseases.

• In March 2025, Alnylam Pharmaceuticals received U.S. Food and Drug Administration (FDA) approval for Amvuttra (vutrisiran) to treat transthyretin amyloid cardiomyopathy (ATTR-CM). This expanded approval allows Amvuttra, an RNA interference (RNAi) therapeutic, to address both cardiomyopathy and polyneuropathy manifestations of ATTR amyloidosis. The approval positions Alnylam as a key competitor in the ATTR-CM market, offering a differentiated treatment option that demonstrated improvements in cardiovascular outcomes and survival.

• In October 2024, City Therapeutics launched with a Series A financing of $135 million, aiming to drive the future of RNA interference (RNAi)-based medicine. The company intends to utilize next-generation engineering of small interfering RNAs (siRNAs) to build a robust product engine. This initiative is designed to expand the therapeutic reach of RNAi-based medicines across multiple disease areas. The lead program is anticipated to commence clinical development by the end of 2025, contributing new therapies to the global RNAi therapeutics market.

• In March 2024, Celanese and Secarna Pharmaceuticals announced a research collaboration focused on advancing long-acting implants for the delivery of antisense oligonucleotides (ASOs). This partnership aims to leverage Celanese's VitalDose drug delivery platform with Secarna's ASO discovery capabilities. The goal is to develop ASO-eluting implants that could reduce dosing frequency and minimize off-target immune responses for various cardiometabolic, central nervous system, oncological, and rare diseases. This development is directly relevant to enhancing the administration methods within the global antisense therapeutics market.

Key Market Players

• Olix Pharmaceuticals, Inc.
• GSK plc
• Sanofi Aventis S.A.
• Alnylam Pharmaceuticals, Inc.
• Ionis Pharmaceuticals, Inc.
• Benitec Biopharma Inc
• Silence Therapeutics Inc.
• Arbutus Biopharma Inc.
• Sarepta Therapeutics Inc.
• Percheron Therapeutics Limited.

Report Scope:

In this report, the Global Antisense And RNAi Therapeutics Market has been segmented into the following categories, in addition to the industry trends which have also been detailed below:

• Antisense And RNAi Therapeutics Market, By Technology:

o   RNA Interference

o   Antisense RNA

• Antisense And RNAi Therapeutics Market, By Route of Administration:

o   Intravenous Injections

o   Intrathecal Injections

o   Subcutaneous Injections

o   Other

• Antisense And RNAi Therapeutics Market, By Application:

o   Ocular

o   Cardiometabolic & Renal Disorders

o   Cancer

o   Respiratory Disorders

o   Skin Diseases

o   Neurodegenerative Disorders

o   Genetic Disorders

o   Infectious Diseases

• Antisense And RNAi Therapeutics Market, By Region:

o   North America

§  United States

§  Canada

§  Mexico

o   Europe

§  France

§  United Kingdom

§  Italy

§  Germany

§  Spain

o   Asia Pacific

§  China

§  India

§  Japan

§  Australia

§  South Korea

o   South America

§  Brazil

§  Argentina

§  Colombia

o   Middle East & Africa

§  South Africa

§  Saudi Arabia

§  UAE

Competitive Landscape

Company Profiles: Detailed analysis of the major companies presents in the Global Antisense And RNAi Therapeutics Market.

Available Customizations:

Global Antisense And RNAi Therapeutics Market report with the given market data, TechSci Research offers customizations according to a company's specific needs. The following customization options are available for the report:

Company Information

• Detailed analysis and profiling of additional market players (up to five).

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