RNA Therapeutics Market By Type (mRNA (Messenger RNA), ASO (Antisense Oligonucleotides), siRNA (Small Interfering RNA), Aptamers, Others); By Product (Vaccines, Drugs); By Indication (Rare Genetic Diseases, Infectious Diseases, Other Indications); By Geography, Segment Revenue Estimation, Forecast, 2024–2032.

RNA Therapeutics Market Introduction

The Global RNA Therapeutics Market is projected to grow from $13.5 billion in 2024 to $43.1 billion by 2030, registering a CAGR of 21.3% during the forecast period. RNA molecules have gained attention as powerful clinical therapeutics, offering precise targeting of previously “undruggable” proteins or molecules while minimizing side effects. RNA-based technologies have revolutionized modern medicine, reshaping vaccine development, gene regulation & precision therapeutics. By April 2024, the FDA had approved a total of 21 RNA-based therapies, comprising ten antisense oligonucleotides (ASOs), six small interfering RNAs (siRNAs), and five mRNA-based vaccines. Backed by regulatory successes such as Onpattro® (patisiran) and Spinraza® (nusinersen), RNA therapeutics are now expanding across a broad spectrum of indications, including oncology, cardiology, infectious disease, and rare genetic disorders.

 

RNA Therapeutics Market Dynamics:

Market Drivers

The RNA therapeutics industry is witnessing significant investment growth, driven by expanding collaborations between biotech startups & leading pharmaceutical firms. Strategic biopharma partnerships are accelerating RNA research, exemplified by Moderna & Merck’s joint efforts on mRNA-based cancer vaccines, as well as Alnylam & Novartis’s collaboration on RNA interference therapies. Eli Lilly & Co entered into a research collaboration with Genetic Leap, a startup specializing in AI & machine learning-driven drug discovery. The partnership, valued at $409 million, aims to advance the development of new drugs, with a strong emphasis on RNA-based therapies. Private investors & government agencies are significantly accelerating RNA innovation through venture capital investments and dedicated funding initiatives. Countries with advanced biotechnology capabilities, such as the US, China, and Germany, are at the forefront of RNA drug development, while emerging biotech hubs are actively advancing their own research programs in the field.

 

Market Opportunities

The RNA therapeutics market presents several compelling opportunities for growth & innovation. One of the most significant is the rising potential in personalized medicine, where RNA therapies can be tailored to individual genetic profiles, particularly in oncology & rare genetic disorders. There is also substantial opportunity in the expansion of mRNA technology beyond vaccines, with ongoing research into its use for cancer immunotherapy, autoimmune diseases & cardiovascular conditions. Partnerships between biotech firms and pharmaceutical giants are creating robust pipelines and accelerating commercialization timelines. Additionally, the increasing prevalence of rare & chronic diseases, coupled with favorable regulatory pathways like orphan drug designation & fast-track RNA approvals, offers a fertile environment for RNA-based drug development. Technological advancements in RNA delivery systems, such as lipid nanoparticles & targeted delivery mechanisms, continue to enhance the safety & efficacy of these therapies, opening the door for broader therapeutic applications.

 

Market Restraints

Despite its promising growth, the RNA therapeutics market faces several key restraints. One of the primary challenges is the delivery of RNA molecules into target cells, as RNA is inherently unstable & susceptible to degradation, requiring advanced & often costly delivery systems like lipid nanoparticles. Additionally, manufacturing complexities & high production costs pose significant barriers, particularly for smaller biotech companies trying to scale up. Regulatory uncertainty and evolving guidelines around relatively new RNA-based therapies can also delay approvals and market entry. Another restraint is the risk of unintended immune responses or off-target effects, which raises concerns about safety and limits broader clinical adoption in some cases. Furthermore, limited long-term efficacy & safety data, especially for newer modalities like RNA editing, can hinder physician and patient confidence. Lastly, market access and reimbursement challenges, particularly in developing regions, may restrict the widespread availability of RNA therapeutics, slowing global market penetration.

 

RNA Therapeutics Market: Recent Developments

• On April 17, 2025, Hoth Therapeutics, Inc. announced that the Japan Patent Office granted it a patent, strengthening its global intellectual property in RNA-based cancer treatments. The patent covers antisense oligomers targeting the KIT gene, aimed at modifying pre-mRNA splicing or reducing KIT protein expression for precise cancer therapy.

• On April 4, 2025, the discreet Chinese biotech firm RiboX Therapeutics made history as the first to initiate clinical trials for an innovative genetic medicine called circular RNA (circRNA). Unlike conventional mRNA therapies, which degrade rapidly within the body, circRNA is engineered for greater stability, potentially enabling longer-lasting effects without the irreversible risks associated with full gene therapy. If proven effective, this breakthrough could pave the way for safer & more affordable treatments for chronic illnesses.

• On January 8, 2025, Angelini Ventures—the corporate venture arm of Angelini Industries, dedicated to supporting innovation in BioTech and Digital Health—announced its participation as a co-lead in a €20 million Series A funding round for Neumirna Therapeutics. Neumirna, an RNA-focused biotech firm, is working on cutting-edge therapeutics designed to address neurological disorders.

 

RNA Therapeutics Market: Pipeline Landscape

As of 2025, there are over 300 active RNA-based clinical trials, with approximately 65% focused on oncology and rare diseases. Notable pipeline assets include:

• SLN360 (Silence Therapeutics) – siRNA for cardiovascular risk

• Sepofarsen (ProQR Therapeutics) – ASO for Leber congenital amaurosis

• BNT122 (BioNTech/Genentech) – individualized mRNA cancer vaccine

 

Figure 1: Pipeline RNA-Based Therapies, by Phase of Clinical Development

BLA: Biologics License Application; NDA: New Drug Application

 

RNA-based therapies span all phases of clinical development, with a strong presence in early stages. As illustrated in Figure 1, 80% of these products are in early-phase trials (Phase I or Phase II), with Phase I alone accounting for one-third (33%). While later-stage development (Phase IIb or beyond) comprises a smaller portion at 20%, the number of late-stage products (29) exceeds the total number of RNA-based therapies currently available (21). This suggests a promising expansion of RNA-based treatments in the near future.

     

Figure 2: Pipeline RNA-Based Therapies, Based on Current Regulation Under NDA/BLA

BLA: Biologics License Application; NDA: New Drug Application

 

As shown in Figure 2, RNA-based therapies in Phase II or later primarily—or exclusively—comprise products expected to be regulated under NDAs. Based on current regulatory interpretations, a substantial portion of future RNA-based therapies is anticipated to enter the market through the NDA approval pathway.

 

FDA Approved Marketed Therapies:

N	Product (INN)	Company	Segment	Indication	Approved	Sales 2022, $m	Sales 2021, $m	Trend YoY
1	Amvuttra (vutrisiran)	Alnylam	siRNA	hATTR amyloidosis	2022	94	-
2	Leqvio (inclisiran)	Alnylam / Novartis	siRNA	Hypercholesterolemia	2021	112	12	833%
3	Amondys 45 (casimersen)	Sarepta	ASO	DMD	2021	215	69	212%
4	Comirnaty (tozinameran)	Pfizer / BioNTech	mRNA	COVID-19	2020	37,806	36,781	3%
5	Spikevax (elasomeran)	Moderna	mRNA	COVID-19	2020	18,400	17,700	4%
6	Oxlumo (lumasiran)	Alnylam	siRNA	Primary hyperoxaluria type 1	2020	70	60	17%
7	Viltepso (viltolarsen)	NS Pharma	ASO	DMD	2020	110	70	57%
8	Vyondys 53 (golodirsen)	Sarepta	ASO	DMD	2019	117	90	30%
9	Givlaari (givosiran)	Alnylam	siRNA	Acute hepatic porphyria	2019	173	128	35%
10	Onpattro (patisiran)	Alnylam	siRNA	hATTR amyloidosis	2018	558	475	17%
11	Tegsedi (inotersen sodium)	Ionis	ASO	hATTR amyloidosis	2018	30 **	56 **	-46%
12	Spinraza (nusinersen)	Ionis / Biogen	ASO	SMA	2016	1,794	1,905	-6%
13	Exondys 51 (eteplirsen)	Sarepta	ASO	DMD	2016	512	454	13%

 

RNA Therapeutics Market: Strategic Partnerships in RNA Space

• On January 22, 2025, Altamira Therapeutics Ltd., a company focused on developing and commercializing RNA delivery technology for applications beyond the liver, announced a collaboration agreement with a radiopharmaceutical sector company. Altamira and its partner will test RNA-loaded nanoparticles with a proprietary radiopharmaceutical in vitro and in vivo. If successful, the partner may negotiate a license and supply agreement to develop and commercialize the technology for cancer treatment.

• On January 15, 2025, Orna Therapeutics, Inc. and Shanghai Simnova Biotech Co., Ltd. announced the expansion of their strategic collaboration to focus on BCMA (B-cell maturation antigen) as a biological target for RNA-based therapeutics. This partnership combines Orna’s innovative circular RNA (oRNA®) technology with Simnova’s cell therapy expertise to develop groundbreaking treatments for patients globally.

• On August 28, 2024, Bayer and NextRNA Therapeutics, a biotech company dedicated to pioneering treatments for diseases driven by long non-coding RNA (lncRNA), announced a collaboration and licensing agreement. Their partnership aims to advance small molecule therapeutics that target lncRNAs in oncology. lncRNAs serve as a diverse group of therapeutic targets by recruiting RNA-binding proteins (RBPs) to influence disease-related processes. The approach of disrupting lncRNA-RBP interactions with small molecules represents a novel strategy for developing a new class of therapeutic agents.

 

RNA Therapeutics Market: Cross Modality CDMO Deals

• On January 9, 2025, Kindeva Drug Delivery, a leading CDMO in drug delivery, and Emervax, the creator of the proprietary circular RNA-based vaccine platform emxRNA™, unveiled an exclusive partnership. Their collaboration focuses on developing a cutting-edge solution for administering vaccines against emerging viruses. As part of the agreement, Kindeva has granted Emervax exclusive rights to incorporate its solid-coated microneedle array patch into the next-generation emxRNA™ platform, marking a significant advancement in RNA vaccine technology.

 

• On January 8, 2025, Evonik partnered with ST Pharma, a CDMO based in South Korea. Through this collaboration, Evonik offers customized nucleic acids from ST Pharm alongside its lipid and lipid nanoparticle (LNP) drug development services. This integrated approach helps pharmaceutical companies streamline processes, reducing complexity and accelerating the market entry of nucleic acid therapeutics.

 

RNA Therapeutics Market: Mergers & Acquisitions

Companies	Country	Acquirer	Founded	Deal year	Deal size, $m
The Medicines Company	US	Novartis	1996	2020	9,700
IVERIC bio	US	Astellas	2007	2023	5,900
Dicerna	US	Novo Nordisk	2006	2021	3,300
Translate Bio	US	Sanofi	2010	2021	3,200
DTx Pharma	US	Novartis	2017	2023	1000
GeneTx	US	Ultragenyx	2017	2022	75
Trasir Tx	US	Auris	2014	2021	3.5

Big Pharma acquires 1-2 RNA therapy companies per year

 

RNA Therapeutics Market Segmentation: A Comprehensive Overview

By Type

• mRNA (Messenger RNA)

• ASO (Antisense oligonucleotides)

• siRNA (Small interfering RNA)

• Aptamers

• Others

 

By Product

• Vaccines

• Drugs

 

By Indication

• Rare Genetic Diseases

• Infectious Diseases

• Other Indications

 

By Region

• North America

• Asia-Pacific

• Europe

• Latin America

• Middle East Africa

 

Segmentation Analysis – RNA Targeted Therapeutics Market

By Type, mRNA leads the market due to the success of mRNA-based COVID-19 vaccines from companies like Pfizer-BioNTech and Moderna. Its scalability, rapid development cycle, and potential in cancer immunotherapy and rare diseases drive continued interest. Antisense oligonucleotides (ASOs) follow closely, with strong demand in treating genetic disorders such as spinal muscular atrophy & Duchenne muscular dystrophy. ASOs benefit from advanced targeting technologies and increasing regulatory approvals. Small interfering RNA (siRNA) has gained traction, particularly with Alnylam’s commercial success in rare liver diseases, and is expected to expand into CNS and other systemic disorders. Aptamers, while still niche, are being explored for use in diagnostics and targeted delivery due to their high specificity and low immunogenicity. Other emerging RNA types, including CRISPR-related RNA editing tools, represent a highly innovative space with long-term potential.

 

 

By Product, RNA-based vaccines hold the largest market share, propelled by pandemic-driven adoption and pipeline development for flu, RSV, and cancer vaccines. RNA-based drugs, however, are gaining momentum with approvals for therapies targeting rare genetic and metabolic diseases, and expanding research into chronic conditions and oncology.

 

By Indication, rare genetic diseases dominate due to the unmet medical need and regulatory incentives for orphan drugs, followed by infectious diseases, where vaccines and antivirals are in high demand. Other indications include cancer, cardiovascular & neurological diseases, representing a growing field of clinical research and investment.

 

By Region, North America remains the largest market, driven by strong biotech infrastructure, funding, and regulatory support. In the US, there are at least 21 U.S FDA approved, on-market, RNA-based therapies, as well as a strong pipeline of products in clinical development. As of January, 2024, at least 131 RNA-based therapies were being studied in clinical trials, with many more in pre-clinical development. Europe follows, with increasing government and private investments in biotech. Asia-Pacific is emerging as a fast-growing region, fueled by rising healthcare investments, clinical trials, and domestic biotech players in countries like China, Japan, and South Korea. Latin America and the Middle East & Africa currently represent smaller shares but show potential for growth due to improving healthcare access and international collaborations.

 

Key Players in the RNA Targeted Therapeutics Market:

• Moderna, Inc.

• Pfizer, Inc.

• Alnylam Pharmaceuticals, Inc.

• Novartis AG

• Ionis Pharmaceuticals, Inc.

• BioNTech SE

• Sarepta Therapeutics, Inc.

• Orna Therapeutics

• CRISPR Therapeutics

• Astellas Pharma Inc.

 

RNA Therapeutics Market Report Coverage

Report Attribute	Details
Forecast Period	2024 – 2032
Market Size Value in 2024	USD 13.5 Billion
Revenue Forecast in 2030	USD 43.1 Billion
Overall Growth Rate	CAGR of 21.3% (2024 – 2030)
Base Year for Estimation	2023
Historical Data	2017 – 2021
Unit	USD Million, CAGR (2024 – 2030)
Segmentation	By Type, By Product, By Indication, By Geography
By Type	mRNA (Messenger RNA), ASO (Antisense Oligonucleotides), siRNA (Small Interfering RNA), Aptamers, Others
By Product	Vaccines, Drugs
By Indication	Rare Genetic Diseases, Infectious Diseases, Other Indications
By Region	North America, Europe, Asia-Pacific, Latin America, Middle East & Africa
Country Scope	U.S., Germany, China, Japan, India, UK, Brazil, etc.
Market Drivers	Advancements in RNA-based drug delivery, rising investment in biotech R&D, and demand for personalized medicine
Pricing and Purchase Options	Customization available upon request