Nucleic Acid Therapeutics Market by Type (Antisense Oligonucleotides, RNA Interference, Small Interfering RNA And Others), Application (Monogenetic Disorders & Multi-Genetic Disorders) End User (Hospitals & Clinics, Academic & Research Institutes, Others) And Region – Global Industry Analysis & Forecast (2024-2030)

The Nucleic Acid Therapeutics Market size was valued at USD 5.23 Billion in 2023 and the total Nucleic Acid Therapeutics Market size is expected to grow at a CAGR of 12.71% from 2024 to 2030, reaching nearly USD 12.09 Billion by 2030.
Nucleic Acid Therapeutics Market Overview
Nucleic acid therapeutics are a type of drug that uses nucleic acids or similar chemical compounds to treat various diseases such as infectious diseases, protein and gene deficiencies, and many types of cancers. Nucleic acid therapeutics can be applied in areas like protein replacement therapy, vaccines, gene editing, and cancer immunotherapy. The nucleic acid therapeutics market is experiencing significant growth driven by the increasing prevalence of genetic disorders and chronic diseases. Nucleic acid therapies offer innovative solutions for conditions like cystic fibrosis, spinal muscular atrophy (SMA), and various cancers, addressing the root causes rather than just managing symptoms. The success of therapies such as Spinraza and Onpattro highlights the market’s potential and the growing demand for precision medicine.

Looking ahead, the nucleic acid therapeutics market holds promising opportunities due to ongoing advancements in technology and increasing investments in research and development. Innovations such as targeting mRNA to prevent disease progression and the expansion of therapies into common diseases like cancer and infectious diseases are expected to drive future growth. Enhanced manufacturing techniques and the exploration of new applications will likely position nucleic acid therapies as a major solution in medicine. Success stories like the development of patisiran for hereditary amyloidosis highlight the potential of these therapies.

Regionally, North America leads the nucleic acid therapeutics market, due to its strong focus on research and development and the presence of major pharmaceutical companies. The region's strong pipeline of clinical trials and regulatory support from institutions like the U.S. Food and Drug Administration (FDA) further cement its leadership. The competitive landscape features key players such as Novartis Pharma AG, Agilent Technologies, and Nippon Shinyaku, all investing heavily in advancing nucleic acid-based therapies and expanding their global reach.

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Nucleic Acid Therapeutics Market Dynamics
Nucleic Acid Therapeutics Market Drivers

Rising Prevalence of Genetic Disorders & Chronic Diseases Drives Nucleic Acid Therapeutics Market Growth

The rising prevalence of genetic disorders and chronic diseases such as Cancer and neurodegenerative diseases significantly drives the growth of the Nucleic Acid Therapeutics Market, as these therapies offer innovative solutions where traditional treatments often fail. Genetic disorders such as cystic fibrosis, spinal muscular atrophy (SMA), and Duchenne muscular dystrophy are caused by specific genetic mutations, and conventional therapies generally focus on symptom management rather than the root cause. Nucleic Acid Therapeutics (NATs), such as antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs), are designed to specifically target and modify the expression of these faulty genes, offering the potential for more effective and lasting treatments. For instance, Spinraza, an ASO, has revolutionized the treatment of SMA by targeting and modifying the splicing of the SMN2 gene, leading to increased production of functional SMN protein, which is essential for motor neuron survival. By 2021, Spinraza had treated more than 11,000 patients globally. Biogen, the manufacturer of Spinraza, reported $1.7 billion in revenue from Spinraza sales.

Spinal Muscular Atrophy (SMA) affects 1 in every 8,000 to 10,000 people worldwide. In the United States, approximately 10,000 to 25,000 children and adults are currently living with SMA.

Chronic diseases including various types of cancer, cardiovascular diseases, and neurodegenerative diseases such as Alzheimer's disease and Parkinson's disease are also driving the demand for Nucleic Acid Therapeutics (NAT). These diseases often involve complex genetic components, which make them difficult to treat with standard therapies. NATs offer a promising approach for directly targeting mutations or genetic pathways involved in these diseases. For instance, Onpattro, an RNA interference (RNAi) therapy, targets and silences the gene responsible for producing the abnormal transthyretin protein in transthyretin-mediated hereditary amyloidosis, thereby treating the underlying disease cause.

As the incidence of these chronic and genetic diseases continues to rise globally, the demand for more precise and effective therapies such as NAT is expected to increase, fueling nucleic acid therapeutics market growth.

Future Opportunities in the Nucleic Acid Therapeutics Market

The future of the nucleic acid therapeutics market is bright and offers significant opportunities for advances in patient care. These therapies are not limited to targeting abnormal proteins but aim to attack the causes of genetic diseases. For instance, by targeting mRNA before it begins to produce abnormal proteins, these treatments could stop disease progression at the source. This innovative approach opens up new possibilities for the treatment of various genetic disorders and chronic diseases. Thanks to increased investments in research and development and an increase in regulatory approvals, the market for nucleic acid therapy is expanding rapidly. This growth is reflected in the success of treatments such as patisiran for hereditary amyloidosis and the continuous development of new therapies for other diseases.

The opportunities for the application of nucleic acid therapies are evolving at the pace of technology. The field has already made considerable progress, with early successes such as DNA-based therapy formivirsen, and RNA-based therapy, demonstrating its potential. In the future, the capabilities of nucleic acid therapies are expected to expand beyond rare diseases to include more common pathologies, such as infectious diseases and cancer. Advanced manufacturing techniques, including high-performance chromatography and mass spectrometry, are essential to ensure the quality and effectiveness of these treatments. As researchers continue to explore new applications and improve delivery methods, nucleic acid therapies are poised to become a major solution in medicine, addressing a wide range of health challenges and opening new avenues for therapeutic development.

Nucleic Acid Therapeutics Market Challenge

The nucleic acid therapeutics market faces several major challenges that affect the effectiveness and development of these treatments. One of the main problems is the susceptibility of nucleic acids to degradation by cellular enzymes. Enzymes such as endonucleases and exonucleases can cleave DNA and RNA molecules, rendering the unmodified nucleotides ineffective for therapy. To address this problem, nucleic acid therapies must be designed to resist these enzymes and maintain their stability in cells. This requires advanced technology to ensure that the therapeutic nucleotides can survive long enough to be effective. Another major challenge is getting these therapies into the right cells. Nucleic acids, like RNA drugs, are heavy and negatively charged, preventing them from crossing cell membranes. They need special transport agents to help them enter the cells and avoid being filtered by the kidneys or taken up by non-target cells. Targeting these therapies to specific tissues, especially those that are not the liver, remains difficult and can lead to unwanted side effects. Although there are distribution systems available, the reliable operation of these systems on a large scale remains a challenge in the nucleic acid therapeutics market.
Nucleic Acid Therapeutics Market Segment Analysis
Based On Types, the Nucleic Acid Therapeutics Market is segmented into Antisense oligonucleotides (ASOs), RNA interference (RNAi), Small interfering RNA (siRNA), and Others. In 2023, the Antisense oligonucleotides (ASOs) dominated the nucleic acid therapeutics market with a share of xx% and are expected to maintain their dominance during the forecasted period (2024-2030). Antisense oligonucleotides (ASOs) are synthetic single-stranded nucleic acid polymers used in nucleic acid therapies to modulate gene expression. ASOs are short single-stranded DNA or RNA molecules designed to bind to specific RNA (mRNA) sequences. This can inhibit protein synthesis, degrade the target mRNA, and prevent translation. ASOs can also modify protein expression and have shown potential in the treatment of genetic disorders and hereditary diseases. ASOs are approved to treat rare neurological disorders such as spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD).

As of February 2024, the US Food and Drug Administration and the European Medicines Agency have approved 16 ASOs for the treatment of rare and common diseases affecting the retina, blood, muscles, nerves, and heart.


Nucleic Acid Therapeutics Market Regional Insight
North America dominated the Global Nucleic Acid Therapeutics Market with the largest market share of XX% in 2023. This is due to the region’s strong focus on R&D, and the presence of major pharmaceutical and biotechnology companies. The increasing prevalence of genetic disorders and chronic diseases such as cancer and neurodegenerative conditions in this region further fueled the demand for nucleic acid therapeutics. Nearly 60% of American adults live with at least one chronic disease. According to the U.S. Department of Health and Human Services, about 129 million people in the U.S. have at least one major chronic disease, such as heart disease, cancer, diabetes, obesity, or high blood pressure.

North America is expected to maintain its dominance in the nucleic acid therapeutics market due to significant funding for genetic research, support from the U.S. National Institutes of Health (NIH), and regulatory approvals from the FDA for therapies like Spinraza and Onpattro. Additionally, the region has a strong pipeline of over 500 clinical trials for gene and RNA-based treatments, which keeps it at the forefront. Companies like Biogen, based in Massachusetts, have achieved major success, with Spinraza generating billions in revenue and treating thousands of patients, solidifying North America's leadership in the nucleic acid therapeutics market.

In 2022, cancer prevalence varied significantly by region. North America had the highest rate, with 595 cases per 100,000 people. Europe follows with 469.5 cases per 100,000 people.



Nucleic Acid Therapeutics Market Competitive Landscape

The competitive landscape for the nucleic acid therapeutics market is defined by major players such as Novartis Pharma AG, Agilent Technologies, Nippon Shinyaku and other prominent players. These companies are at the forefront of developing nuclear acid-based drugs and invest heavily in research and development. Companies focused on developing effective treatments for genetic and chronic diseases. Competition is intense among these key players as they try to bring new therapies to market and expand their global reach.

1. In 2022, Agilent invested USD 725 million in nucleic acid-based therapeutics to expand its production capacity. This investment is aimed at doubling the company's ability to manufacture active pharmaceutical ingredients (APIs) used in various drugs, specifically focusing on therapeutic nucleic acids, which are short DNA and RNA molecules critical for these treatments.

2. In February 2024, UT Southwestern partnered with Pfizer to develop advanced RNA delivery technologies for genetic medicine therapies. This partnership aims to combine Pfizer's expertise in chemistry and engineering with UT Southwestern's research to enhance the understanding of genetic medicines, utilize AI design methods, and create new delivery technologies for future therapies.

3. In May 2024, Aldevron announced a strategic partnership with Acuitas Therapeutics to enhance its capabilities in mRNA lipid nanoparticle (LNP) encapsulation. This collaboration allows Aldevron, a global leader in manufacturing DNA, RNA, and proteins, to integrate Acuita's specialized LNP encapsulation technology into its services. This partnership builds on Aldevron's recent expansion of mRNA production and will significantly boost its ability to provide comprehensive mRNA drug product services, including supporting Acuitas LNP formulations and licensees.
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Global Nucleic Acid Therapeutics Market


Report Coverage
Details


Base Year:
2023
Forecast Period:
2024-2030


Historical Data:
2018 to 2023
Market Size in 2023:
USD 5.23 Bn.


Forecast Period 2024 to 2030 CAGR:
12.71%
Market Size in 2030:
USD 12.09 Bn.


 Segments Covered:
by Type
Antisense oligonucleotides (ASOs)
RNA interference (RNAi)
Small interfering RNA (siRNA)
Others


by Application
Monogenetic disorders
Multi-genetic disorders


by End User
Hospitals and Clinics
Academic and Research Institutes
Others



Nucleic Acid Therapeutics Market, by Region
North America (United States, Canada and Mexico)
Europe (UK, France, Germany, Italy, Spain, Sweden, Austria and Rest of Europe)
Asia Pacific (China, South Korea, Japan, India, Australia, Indonesia, Malaysia, Vietnam, Taiwan, Bangladesh, Pakistan and Rest of APAC)
Middle East and Africa (South Africa, GCC, Egypt, Nigeria and Rest of ME&A)
South America (Brazil, Argentina Rest of South America)
Nucleic Acid Therapeutics Market, Key Players
1. Novartis Pharma AG
2. Ionis Pharmaceuticals, Inc.
3. Arbutus Biopharma
4. Thermo Fisher Scientific
5. Agilent
6. Alynylam Pharmaceuticals
7. Nippon Shinyaku
8. Silence Therapeutics plc
9. Pfizer
10. Sarepta Therapeutics
11. Roche Holding AG
12. Eli Lilly and Company
13. Copernicus Therapeutics Inc.
14. Sumitomo Chemical Co., Ltd.
15. Aldevron
16. Genzyme Sanofi
17. lonis Pharmaceuticals, Inc.
18. Moderna

Frequently Asked Questions:

1. Which region has the largest share in the Nucleic Acid Therapeutics Market?
Ans: The North American region held the largest share in 2023.

2. What is the growth rate of the Nucleic Acid Therapeutics Market?
Ans: The Nucleic Acid Therapeutics Market is expected to grow at a CAGR of 12.71% during the forecast period 2024-2030.

3. What is the market size of the Nucleic Acid Therapeutics Market?
Ans. The Nucleic Acid Therapeutics Market size was valued at USD 5.23 billion in 2023 reaching nearly USD 12.09 billion in 2030.

4. What are the different segments of the Nucleic Acid Therapeutics Market?
Ans. The Nucleic Acid Therapeutics Market is divided by Type, application, and End-User.

5. What is the scope of the Nucleic Acid Therapeutics Market report?
Ans: The Nucleic Acid Therapeutics Market report helps with the PESTLE, PORTER, Recommendations for Investors & Leaders, and market estimation of the forecast period.