The global antisense and RNAi therapeutics market size reached USD 5.20 billion in 2023 and is projected to hit around USD 30.03 billion by 2033, expanding at a CAGR of 19.12% during the forecast period from 2024 to 2033.
Due to advancements in RNA-based drug development and improved understanding of disease mechanism, the market of antisense and RNAi has seen significant growth.
Report Source: https://www.towardshealthcare.com/insights/antisense-and-rnai-therapeutics-market-sizing
Key Insights of the Antisense and RNAi Therapeutics Market
· By region, North America dominated the market by 62.10% in 2023.
· By region, Asia Pacific is expected to grow at the fastest rate during the forecast period.
· By technology, the antisense RNA segment held the largest share of the market in 2023.
· By application, the neurodegenerative disorders segment dominated the antisense and RNAi therapeutics market in 2023.
· By route of administration, the intrathecal injections segment held the largest share of the market in 2023.
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Antisense and RNAi Therapeutics Market: Overview & Factors
The modern treatment process that works at genetic level to treat and cure diseases are called Antisense and RNA interference (RNAi). In antisense therapy, synthetic strands of DNA or RNA are used to bind the messenger RNA, which is mRNA, offering a specific gene, blocking its ability to produce a harmful protein. RNAi, on the other hand, uses small RNA molecules to interfere with the mRNA, preventing the production of disease-causing proteins. Both these approaches are designed to target and silence specific genes, offering a more precise way to treat genetic disorders, cancers, and other diseases.
The antisense and RNAi therapeutics market is on to grow at a significant growth rate driven by advancements in RNA technology. Increased funding for rare disease research and development internationally is expanding the market.
Drivers
Technological Advancements in Drug Delivery Systems
Ongoing innovations and advancements in the drug delivery system the driving the growth of antisense and RNAi therapeutics market. Advanced drug delivery systems are revolutionizing how RNA therapies are administered, ensuring they reach their target cells more effectively and with greater precision. One significant breakthrough is the development of lipid nanoparticles, which act as tiny carriers for RNA molecules. They help to overcome the natural barriers that RNA faces, such as rapid degradation and difficulty penetrating cell membranes. By enveloping RNA in these lipid particles, the stability and delivery efficiency of the drugs are significantly enhanced.
· For instance, in July 2023, Novartis made headlines by acquiring DTx Pharma, a company specializing in short-interfering RNA (siRNA) therapies aimed at treating neurological conditions. This move highlights a growing confidence in the potential of lipid nanoparticle technologies and their role in advancing RNA-based treatments.
RNA Stability Issues Restraining the Antisense and RNAi Therapeutics Market
Antisense and RNAi therapies have groundbreaking potential yet one of the significant problems that they face is RNA stability which restraints the market. RNA molecules are unstable and can be quickly broken down by RNases enzymes found in cells and tissues that degrade RNA. Which makes is challenging to ensure that that RNA-based drugs remain intact long enough to reach their intended targets within cells. For these therapies to be effective, RNA molecules need to be delivered to the cytoplasm of target cells without being degraded or losing their functional integrity.
RNA molecules have a negative charge, making it difficult for them to pass through hydrophobic (water-repellent) lipid membranes of cells. Because of these barriers RNA molecules struggle to cross the cell membrane, where they need to be to perform their therapeutic functions. As a result, developing effective delivery systems that can protect RNA from degradation and facilitate its entry into cells is a major area of focus.
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Turning Challenges into Innovations
The high cost and complexity associated with developing and managing of RNAi therapies causes significant challenges for the antisense and RNAi therapeutics market. But the challenges also present a unique opportunity for the market. The complexity of these therapies drives innovation and fosters breakthroughs, especially in the realm of treating previously untreatable genetic diseases.
Using sophisticated technology and extensive research it is possible to develop RNAi and antisense based therapies. Which comparatively can be costly. The process involves creating precise RNA molecules that can interact with specific genes or gene product this task requires or demands high tech technology and higher expertise this complexity not only raises the cost but also presents an opportunity for the industry to innovate and reduce expenses in the long run.
One of the most promising areas of opportunity lies in the development of new, cost-effective drug discovery methods. Advances in technology and research are making it possible to identify and develop RNA-based therapies more efficiently.
· In 2024, this partnership announced promising results from their AI-driven platform, which successfully identified novel RNA targets for several rare genetic diseases. This kind of technological advancement not only speeds up the discovery process but also has the potential to lower costs by streamlining research and development.
RNA Interference, to Become Widely Used Technology
By technology, RNA interference dominated the antisense and RNAi therapeutics market in 2023. RNAi is useful for gene silencing, allowing for precise targeting and suppression of specific genes associated with various diseases. RNAi has become popular due to its ability to selectively inhibit the expression of disease-causing genes, making it a promising approach for treating conditions like cancer and genetic disorders. For instance, companies are exploring new nanoparticle-based delivery methods to enhance the targeted delivery of RNAi agents, thereby increasing their effectiveness and reducing side effects.
· In December 2023, Atomic AI, a U.S.-based biotech startup, introduced ATOM-1, a new platform that leverages large language models (LLMs) to predict RNA structure and function. This innovation aims to address challenges in developing RNA-based treatments by improving the accuracy of RNA-targeted small molecules.
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Advancements to Support Neurodegenerative Disorder Segment
The neurodegenerative disorders segment holds a notable share in applications for antisense and RNAi therapeutics. The conditions, such as Parkinson’s diseases and Alzheimer’s have long been challenging to treat due to their complex and progressive nature. Recent Progress in neurodegenerative disorders includes development of RNS-based therapies that target specific genetic mutations linked to these diseases. For example, several companies are advancing antisense oligonucleotides aimed at modifying the progression of neurodegenerative diseases by targeting faulty gene expressions. Innovations in this field are making it easier to address these debilitating conditions more effectively which offers hope for patients
In May 2024, Isarna Therapeutics, a German biotech company, unveiled new antisense therapies targeting transforming growth factor beta (TGF-B) signaling pathways. These treatments are designed to modulate TGF-B pathways more precisely, potentially offering safer and more effective options for neurodegenerative diseases and other conditions.
Intrathecal Injections Segment to Hold Lion’s Share
The intrathecal injections segment dominated the antisense and RNAi therapeutics market by route of administration in 2023. Intrathecal injections involve delivering medication directly into the spinal canal. Which allows for targeted treatment of central nervous system disorders. It’s particularly useful for conditions where precise delivery is crucial. Recent advancements in this includes the development of improved delivery systems that reduce invasiveness and increase the efficacy of intrathecal injections.
· In April 2024, A breakthrough in intrathecal drug delivery technology was achieved with the development of a new, less invasive delivery system. This system enhances the precision of drug administration into the spinal canal, improving outcomes for patients with central nervous system disorders.
Tech Innovations: North America’s Projection as a Leader
In 2023 North America dominated the antisense and RNAi therapeutics market, thanks to its advanced healthcare infrastructure and significant investment in research and development. The United States is at the forefront, with a substantial number of clinical trials and FDA approvals. For instance, Alnylam Pharmaceuticals received FDA approval for Givlaari, an RNAi therapeutic for acute hepatic porphyria. This region’s strong support for biotech innovation and continuous investments ensures it remains a leader in this space.
· In June 2024, The U.S. National Science Foundation awarded nearly $12.7 million to nine research teams under the Molecular Foundations for Biotechnology (MFB) program. This funding aims to explore the potential of RNA for a range of applications, from cancer treatments to agricultural improvements, highlighting North America’s role as a leader in RNA-based therapeutic advancements.
· In August 2022, Cure SMA announced the addition of 9 new spinal muscular atrophy screening centers to its Care Center Network. In addition, currently, there are 27 such care centers in this network across the United States. Thus, this leads to a surge in early screening in the region which in turn leads to a surge in demand for antisense and RNAi therapeutics.
Driven by supportive European government policies and increasing funding for biotechnological research, Europe is also making significant strides. Countries like Germany, the UK, and France are key players. The European Medicines Agency (EMA) recently approved Leqvio, an RNAi therapy developed by Novartis to lower cholesterol, marking a significant advancement in cardiovascular treatments.
For instance, In January 2024, The European Medicines Agency (EMA) granted conditional approval for an RNA-based therapy targeting a rare form of cancer. This development highlights Europe’s active role in bringing innovative RNA therapies to patients with limited treatment options.
Advancements in Therapies to Create Potential for Asia
Due to its increasing healthcare expenditure and rising awareness of advanced therapies, the region Asia Pacific is on to grow at a rapid growth rate in the forecast period. China and Japan are leading the charge, investing heavily in RNA-based drug development. Suzhou Ribo Life Science Co. in China recently completed Phase II trials for an RNAi-based hepatitis B treatment, while Japan’s Takeda Pharmaceuticals has partnered with Arrowhead Pharmaceuticals to develop RNAi therapeutics for liver diseases.
· For instance, In March 2024, Japan’s Ministry of Health, Labour and Welfare approved a new RNAi therapeutic for treating liver diseases. This approval is part of Japan’s broader strategy to enhance its biotech sector and provide cutting edge treatments to its population.
With Time and technological advancements in biotechnology India is also an emerging market in Asia Pacific. The country is ramping up its investments in biotechnology, supported by government initiatives like the Biotechnology Industry Research Assistance Council (BIRAC). Indian pharmaceutical companies are increasingly focusing on RNAi and antisense technologies. For instance, Dr. Reddy’s Laboratories recently announced a collaboration with a US-based biotech firm to develop RNAi-based treatments for chronic liver diseases. For fostering innovation and market growth, the Indian government has also invested and launched new funding schemes go support biotechnology and biotech startups.
New Advancements in Antisense and RNAi Therapeutics Market
· In June 2024, Deep Genomics expanded its operations and opened a new lab in Cambridge, Massachusetts. The company focuses on using machine learning to create RNA therapeutics for hereditary illnesses.
· In June 2024, Exsilio Therapeutics raised $82 million in a series A funding round led by Novartis Venture Fund and Delos Capital, aiming to develop RNA therapeutics using mobile genetic elements.
· In April 2024, Alnylam Pharmaceuticals, Inc. and Medison Pharma announced an expansion of their existing partnership in Central & Eastern Europe and Israel, to include certain territories in LATAM and APAC regions, as well as other global markets. The partnership will ensure expansion and commercialization of RNA-based therapeutics such as ONPATTRO (patisiran), AMVUTTRA (vutrisiran), GIVLAARI (givosiran), and OXLUMO (lumasiran) globally.
· In April 2024, Chugai Pharmaceutical Co., Ltd. announced a license agreement with Alnylam Pharmaceuticals, Inc. and F. Hoffmann-La Roche AG to obtain commercialization rights in Japan for zilebesiran, an investigational RNAi therapeutic indicated for hypertension.
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Top Companies in Antisense and RNAi Therapeutics Market
· GSK plc
· Deep Genomics
· Percheron Therapeutics
· Sanofi
· Silence Therapeutics
· Exsilio Therapeutics
· Benitec Biopharma
· Arbutus Biopharma
· Ionis Pharmaceuticals
· Alnylam Pharmaceuticals
· Olix Pharmaceuticals
· Sarepta Therapeutics
Segments Covered in the Report
By Technology
- RNA Interference
- Antisense RNA
By Application
- Genetic Disease
- Cancer
- Infectious Disease
- Neurodegenerative Disorders
- Cardiometabolic & Renal Disorders
- Ocular Disorders
- Respiratory Disorders
- Skin Disorders
By Route of Administration
- Intravenous Injections
- Intrathecal Injections
- Subcutaneous Injections
- Other Delivery Methods
By Region
- North America
- U.S.
- Canada
- Europe
- Germany
- UK
- France
- Italy
- Spain
- Sweden
- Denmark
- Norway
- Asia Pacific
- China
- Japan
- India
- South Korea
- Thailand
- Latin America
- Brazil
- Mexico
- Argentina
- Middle East and Africa (MEA)
- South Africa
- UAE
- Saudi Arabia
- Kuwait
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