Visão geral do mercado

The Nucleic Acid Based Therapeutics Market is poised to experience substantial growth, with its size expected to expand from USD 4,895 million in 2023 to USD 9,545 milhão por 2028, at a Compound Annual Growth Rate (CAGR) of 14.29% during the forecast period (2023-2028).

This market revolves around nucleic acid therapeutics, which are based on nucleic acids or related chemical compounds utilized in the treatment of various diseases. Notable examples include aptamers, antisense oligonucleotides (ASOs), and small interfering RNAs (siRNAs), which are often employed when precise suppression of a specific gene’s function related to a disease is deemed therapeutically beneficial. ASOs and siRNAs are particularly effective in silencing genes by interfering with mRNA translation into proteins and the transcription of nuclear DNA into mRNA without modifying them.

Several factors contribute to the anticipated growth of the nucleic acid-based therapeutics market. These include the escalating prevalence of genetic diseases, growing investments in the healthcare sector, and the pharmaceutical industry’s rapid shift towards innovative biologics.

The rising prevalence of genetic diseases such as cancer, cystic fibrosis, sickle cell anemia, Duchenne muscular dystrophy, thalassemia, and others plays a pivotal role in driving the demand for nucleic acid-based therapeutics. These therapeutics hold the potential to treat diseases by correcting the abnormal expression of specific genes. For example, data from the Cystic Fibrosis Foundation published in July 2022 indicated that in the United States, 40,000 children and adults are affected by cystic fibrosis (CF). Globally, an estimated 105,000 people were diagnosed with CF in 94 countries in 2022. Consequently, the significant burden of cystic fibrosis among the population is expected to positively impact the market.

Increasing investments in nucleic acid therapeutics, driven by the high prevalence of chronic, genetic, and other diseases, are expected to further boost market growth. Notably, in March 2023, Switch Therapeutics raised USD 52 million in a Series A financing round co-led by Insight Partners and UCB Ventures. The company utilized these funds to advance the development of siRNA therapeutic candidates for the treatment of a central nervous system disease and further their RNAi technology. Market players are actively investing in nucleic acid-based therapy research and development to expand their product portfolios in this field. For instance, in January 2023, Agilent Technologies Inc. invested USD 725 million in nucleic acid-based therapeutics, with the aim of doubling their manufacturing capacity for active pharmaceutical ingredients (APIs).

Companies’ increasing focus on collaborations, partnerships, new product launches, and other initiatives enhances the availability of novel therapeutic drugs in the market, thereby contributing to its growth. For example, in February 2023, the CMT Research Foundation partnered with Nanite Inc. to enhance the therapeutic efficacy of antisense oligonucleotides in Charcot-Marie-Tooth disease. In October 2022, Neuway Pharma GmbH and Wacker collaborated on a research project to identify and manufacture RNA-based therapeutics for the treatment of central nervous system disorders using EnPC (Engineered Protein Capsules) protein-based drug delivery technology.

Segmentação de mercado

The market is segmented based on various factors, including product type, application, end-user, and geography.

Segmentation by Product Type
RNA interference [RNAi] and short interfering RNAs [siRNAs]
Antisense Oligonucleotides (ASOs)
Other Product Types

Segmentation by Application
Autoimmune Disorders
Infectious Diseases
Genetic Disorders
Cancer
Other Applications

Segmentation by End-User
Hospitals and Clinics
Academic and Research Institutes

Segmentation by Geography
North America – Estados Unidos, Canadá, México
Europe – Reino Unido, Alemanha, França, Itália, Espanha, and Rest of Europe
Asia-Pacific – China, Japão, Índia, Austrália, South Korea, and Rest of Asia-Pacific
Latin America – Brasil, Argentina, and Rest of Latin America
Middle East and Africa – GCC, África do Sul, and Rest of Middle East and Africa

Antisense oligonucleotides (ASOs) are short, single-stranded DNA or RNA molecules that possess the capability to selectively bind to specific messenger RNA (mRNA) sequences, resulting in the degradation of the targeted mRNA or the inhibition of its translation into protein. This unique property has rendered ASOs a highly promising therapeutic strategy for a wide range of diseases, encompassing genetic disorders, infectious diseases, and cancer. ASOs can effectively target disease-causing genes, including those responsible for the production of aberrant proteins or enzymes that contribute to disease progression.

Several approved ASO therapies stand as examples of this therapeutic approach’s success. Notable among them are Spinraza (nusinersen), utilized for the treatment of spinal muscular atrophy; Exondys51 (eteplirsen), employed for Duchenne muscular dystrophy treatment; Onpattro (patisiran), indicated for hereditary transthyretin-mediated amyloidosis treatment; and Tegsedi (inotersen), utilized for hereditary transthyretin-mediated amyloidosis treatment.

The ASOs segment is expected to witness significant growth during the forecast period due to the escalating demand for antisense oligonucleotide drugs, an upsurge in research and development activities by key industry players, and the introduction of new products to the market.

Companies’ increasing focus on adopting diverse business strategies, such as partnerships, collaborations, acquisitions, and other initiatives, to accelerate the research and development of novel therapeutics for rare and genetic disorders is anticipated to drive the segment’s growth. For instance, in September 2022, Vanda Pharmaceuticals Inc. and OliPass Corporation entered into an R&D collaboration agreement to jointly develop a set of antisense oligonucleotide (ASO) molecules based on modified peptide nucleic acids. In February 2021, the US FDA approved Sarepta’s Amondys 45 (casimersen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation of the DMD gene.

As a result, the segment is projected to grow over the forecast period due to the availability of several antisense oligonucleotide products in the market, increased company activities, and the introduction of new products.

In North America, the market is expected to hold a significant share, attributed to the growing research on various nucleic acid-based therapies, an increasing prevalence of genetic disorders and other chronic disorders, and escalating research and development investments in the region.

The rising prevalence of autoimmune diseases among the population contributes to the demand for nucleic acid-based therapies, thus augmenting market growth. For example, as per the Autoimmune Association’s data in June 2022, autoimmune diseases comprise approximately 80-150 unique, chronic conditions, affecting more than 31 million Americans annually. This highlights the substantial burden of autoimmune disorders in the target population. Furthermore, an article published in the Clinical Rheumatology Journal in February 2023 indicated a large burden of systemic autoimmune rheumatic diseases (SARDs) in Canada, impacting between 2 e 5 cases per 1,000 residents.

The increasing burden of cancer also drives the demand for effective and innovative drugs that can impede the progression of cancer cells, further boosting the demand for nucleic acid-based drugs in the region. According to the Cancer Facts and Figures 2023, approximately 1.9 million new cancer cases are estimated to be diagnosed in the United States in 2023. Similarly, as per statistics updated by the Government of Canada in June 2022, nearly 233,900 people in Canada were projected to be diagnosed with cancer by the end of 2022, with lung, breast, prostate, and colorectal cancers anticipated to be the most commonly diagnosed cancers in the Canadian population.

The region’s market growth is also fueled by innovative product launches and approvals, partnerships, acquisitions, expansions, and collaborations. For instance, in September 2022, Next Generation Manufacturing Canada (NGen) invested USD 10.5 million in a USD 34.8 million project led by OmniaBio Inc. and partners ExCellThera, MorphoCell Technologies, Aspect Biosystems, and Canadian Advanced Therapies Training Institute (CATTI). Additionally, in December 2021, Novartis AG received approval from the FDA for Leqvio (inclisiran), the first and only small interfering RNA (siRNA) therapy designed to lower low-density lipoprotein cholesterol (LDL-C or bad cholesterol) with just two doses a year, after an initial dose and one at three months.

Cenário competitivo

The nucleic acid-based therapeutics market exhibits moderate fragmentation, attributed to the presence of numerous small and large players on both a global and regional scale. These companies are actively involved in researching and developing nucleic acid-based therapeutics to address various chronic, genetic, and infectious diseases. Notable participants in the market include Silence Therapeutics PLC, Ionis Pharmaceuticals Inc., Sarepta Therapeutics, Novartis Pharma AG, Alnylam Pharmaceuticals Inc., and Biogen Inc., among others.

Key companies profiled in this report include Silence Therapeutics PLC, Ionis Pharmaceuticals Inc., Novartis Pharma AG, Arrowhead Pharmaceuticals Inc., Stoke Therapeutics Inc., Moderna Inc., Alnylam Pharmaceuticals Inc., Biogen Inc., Wave Life Sciences, Sarepta Therapeutics Inc.

Recent Industry Developments

In March 2023, Ionis Pharmaceuticals received unanimous approval from the Food and Drug Administration (FDA) advisory committee for the potential accelerated approval of Tofersen, intended for SOD1-ALS treatment. Tofersen is an antisense oligonucleotide that facilitates the degradation of superoxide dismutase 1 (SOD1) messenger RNA, thereby reducing SOD1 protein synthesis.

In February 2023, Myeloid Therapeutics Inc. entered into a collaboration with the New South Wales (NSW) Government in Australia to establish a state-of-the-art GMP (Good Manufacturing Practice) manufacturing facility, dedicated to the development of RNA immunotherapies. This new facility aims to expedite the commercialization of Myeloid’s RNA therapeutics and foster the growth of an RNA ecosystem in the region of NSW.

Key Questions Answered

What is the study period of this market?
The study period of the Nucleic Acid Based Therapeutics Market is from 2018 to 2028.

What is the growth rate of the Nucleic Acid Based Therapeutics Market?
The Nucleic Acid Based Therapeutics Market is experiencing a growth rate of 14.29% over the next 5 years.

What is the size of the Nucleic Acid Based Therapeutics Market?
The Nucleic Acid Based Therapeutics Market is expected to reach USD 4,895 million in 2023 and is projected to grow at a CAGR of 14.29% to reach USD 9,545 milhão por 2028.

Which region has the highest growth rate in the Nucleic Acid Based Therapeutics Market?
The Asia-Pacific region is witnessing the highest CAGR over the period of 2018 to 2028.

Which region has the largest share in the Nucleic Acid Based Therapeutics Market?
North America currently holds the highest market share as of 2022.

Who are the key players in the Nucleic Acid Based Therapeutics Market?
The major companies operating in the Nucleic Acid Based Therapeutics Market are Silence Therapeutics plc, Ionis Pharmaceuticals, Inc., Novartis Pharma AG, Sarepta Therapeutics, Inc., and Alnylam Pharmaceuticals, Inc.