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Nucleic Acid-based Therapeutics Companies

Nucleic acid-based therapeutics involve the use of genetic material, such as DNA or RNA, to treat or prevent diseases. These therapies can target specific genes or cellular processes, offering potential treatments for various medical conditions, including genetic disorders, cancers, and infectious diseases.

Nucleic Acid-based Therapeutics Key Companies


Latest Nucleic Acid-based Therapeutics Companies Update

Nov 2023Moderna (US) Received FDA approval for their second-generation COVID-19 booster vaccine (mRNA-1273.214) offering broader protection against circulating variants.Advanced clinical trials for their mRNA-based therapies for HIV, cytomegalovirus, and various types of cancer.

BioNTech (Germany) Partnered with Genmab to develop mRNA-based cancer vaccines targeting specific tumor antigens.Initiated Phase 1 trials for their mRNA therapy for Duchenne muscular dystrophy.

Alnylam Pharmaceuticals (US) Received FDA approval for their Onpattro® (patisiran) for the treatment of hereditary ATTR amyloidosis, expanding their RNAi therapy portfolio.Announced positive results from Phase 2a trials for their RNAi therapy for alpha-1 antitrypsin deficiency, a genetic liver disease.

Editas Medicine (US) Dosing of the first patient in their Phase 1/2 clinical trial for their CRISPR gene editing therapy for Leber congenital amaurosis, a genetic eye disease.Collaborated with Roche to develop CRISPR-based therapies for various rare genetic diseases.

Wave Life Sciences (US) Filed an IND application with the FDA for their mRNA therapy for Duchenne muscular dystrophy, entering the race for this promising market.Collaborated with Pfizer to develop mRNA-based therapies for rare neuromuscular diseases.

List of Nucleic Acid-based Therapeutics Key Companies in the Market

  • Wave Life Sciences Ltd

  • Copernicus Therapeutics Inc

  • Imugene

  • Caperna

  • Phylogica

  • Protagonist Therapeutics

  • Benitec Biopharma

  • EGEN (Expression Genetics)

  • Benitec Biopharma

  • BioMedica (Oxford BioMedica)

  • Transgene

Global Nucleic Acid-based Therapeutics Market Overview

The Nucleic Acid-based Therapeutics Market Size was valued at USD 0.35 billion in 2022. The nucleic acid-based therapeutics market industry is projected to grow from USD 0.6 Billion in 2023 to USD 0.86 billion by 2032, exhibiting a compound annual growth rate (CAGR) of 4.1% during the forecast period (2023 - 2032). Technology advancements and an understanding of the human genome have facilitated the creation of several nucleic acid-based therapies. Even the US FDA and EMA's approvals show the promise for genetically treating disorders are the key market drivers enhancing the market growth.Nucleic Acid-based Therapeutics Market Overview

Source Secondary Research, Primary Research, MRFR Database and Analyst Review

Nucleic Acid-based Therapeutics Market Trends

  • Rising Investments in Healthcare Sectors to boost the market growth

The increased investments in the healthcare industry have also accelerated research into several illnesses and ailments. Research efforts to improve healthcare environments ly have increased due to the rising prevalence of genetic abnormalities and chronic illnesses among the population.

The growing frequency of chronic diseases, including cancer and cardiovascular disorders, and the rising investments in nucleic acid treatments further drive market growth. The Soros Economic Development Fund (SEDF) announced in May 2022 that it would contribute USD 100 million to COVAX's efforts to increase the distribution of COVID-19 vaccinations to low- and middle-income nations. For instance, the Kingdom of Saudi Arabia (KSA) declared in October 2022 that it would spend USD 36.8 billion or around 14.4% of its 2022 budget, on healthcare development. As a result, the increase in investments in healthcare development is anticipated to present profitable opportunities for market CAGR.

However, the creation of gene treatments for development and clinical use permission has increased over the past several years due to the growing understanding of the human genome, which is another factor driving the growth of the nucleic acid-based therapeutics market revenue.

Nucleic Acid-based Therapeutics Market Segment Insights

Nucleic Acid-based Therapeutics Technology Insights

Based on technology, the nucleic acid-based therapeutics market segmentation includes anti-sense and anti-gene, short inhibitory sequences, gene transfer therapy, nucleoside analogs, ribozymes, and aptamers. In terms of technology segmentation, the anti-sense and anti-gene segment was expected to dominate the market in 2022, accounting for the largest share of the market of the nucleic acid-based therapeutics market revenue. This is due to the increasing prevalence of chronic diseases such as cancer and genetic disorders, major therapeutic targets for anti-sense and anti-gene therapies.

However, the gene transfer therapy segment was projected to be the fastest-growing segment during the forecast period, with a high CAGR. This is due to the increasing focus on personalized medicine and the potential of gene transfer therapy to treat a range of genetic diseases, including rare genetic disorders.

October 2022 EnPC (Engineered Protein Capsules), a protein-based drug delivery technology, and Neuway Pharma GmbH and Wacker have announced the beginning of a research collaboration to discover and produce RNA-based therapies for the treatment of illnesses of the central nervous system. 

Nucleic Acid-based Therapeutics Application Insights

Based on application, the nucleic acid-based therapeutics market segmentation includes Monogenetic disorders, Thalassemia, Sickle cell anemia, Hemophilia, Cystic Fibrosis, Multi-genetic disorders, Cancer, Diabetes, Neurodegenerative Diseases, Cardiovascular Diseases etc. Since most diseases treated by nucleic acid therapies are hereditary and extremely common, the monogenetic disorders sector had the biggest revenue contributor to the market in 2022. The significant nucleic acid therapeutics market growth is attributable to newly developed and authorized medicines by the industry players to cure single gene problems. Industry leaders are concentrating on creating innovative methods to get around problems, including off-targets, stability, and gene quality in multi-genetic illnesses will likely drive the demand for nucleic acid-based therapeutics market growth.

For instance, Breastcancer.org predicted that in 2022 there would be 51,400 new instances of non-invasive (in situ) breast cancer and 287,850 new cases of invasive breast cancer in women. Those elements will probably encourage the use of nucleic acid-based medicines, which will help the market under study develop.

Nucleic Acid-based Therapeutics End Users Insights

The nucleic acid-based therapeutics market data has been bifurcated by surgery into hospitals and academic & research institutes. Hospitals and academic & research institutes are expected to play important roles in developing and adopting nucleic acid-based therapeutics. Hospitals are likely to be the primary administration sites for these therapies. At the same time, academic and research institutes will likely continue to play a key role in the research and development of new therapies. Overall, as the market for nucleic acid-based therapeutics continues to grow, hospitals and academic & research institutes are expected to see increased demand for their services. However, the extent of growth in each segment may vary depending on factors such as regulatory approval, clinical trial success, and patient acceptance.

Figure 1 Nucleic Acid-based Therapeutics Market, by End User, 2022 & 2030 (USD Million)Nucleic Acid-based Therapeutics Market, by End User, 2022 & 2030

Source Secondary Research, Primary Research, MRFR Database and Analyst Review

Nucleic Acid-based Therapeutics Regional Insights

By Region, the study provides the Nucleic Acid-based Therapeutics market insights into North America, Europe, Asia-Pacific and Rest of the World. Due to factors including the high incidence of chronic illnesses like cancer and diabetes, the rising need for targeted and customized therapy, and supportive government efforts, North America is predicted to lead the market. For instance, more than 130 people in the United States presently have diabetes, according to the CDC National Diabetes Statistical Report published in January 2022. The United States has dominated North American markets. The rising investment in R&D activities leading to an increase in developments in gene therapy and the rising incidence of target diseases are the main drivers of market growth.

Further, the major countries studied in the market report are The U.S, Canada, Germany, France, UK, Italy, Spain, China, Japan, India, Australia, South Korea, and Brazil.


Source Secondary Research, Primary Research, MRFR Database and Analyst Review

Europe nucleic acid-based therapeutics market accounts for the second-largest market share due to the nucleic acid testing market in Europe being anticipated to benefit during the projected period due to rising investments in the diagnostics and research sectors and the rising occurrence of infectious disorders. Further, the Germany nucleic acid-based therapeutics market held the largest market share, and the UK nucleic acid-based therapeutics market was the fastest-growing market in the European region

The Asia-Pacific Nucleic Acid-based Therapeutics Market is expected to grow at the fastest CAGR from 2022 to 2030. Due to factors including growing knowledge of nucleic acid testing and a transfer in research institutes and contract research companies from developed western nations to emerging countries in Asia Pacific, the region is predicted to have a greater CAGR than other regions. Moreover, China nucleic acid-based therapeutics market held the largest market share, and the India nucleic acid-based therapeutics market was the fastest-growing market in the Asia-Pacific region

For instance, the American biotechnology business Ionis Pharmaceuticals, Inc. reported in January 2022 that Tofersen, an experimental antisense drug for treating superoxide dismutase one amyotrophic lateral sclerosis, had received FDA clearance (SOD1-ALS).

Nucleic Acid-based Therapeutics Key Market Players & Competitive Insights

The Major market players for nucleic acid-based medicines will expand even more as major manufacturers invest heavily in R&D to expand their product ranges. With significant market developments like the introduction of new products, contractual agreements, mergers and acquisitions, increased investments, and collaboration with other organizations, market participants are also undertaking a variety of strategic initiatives to expand their footprint. Competitors in the nucleic acid-based therapeutics industry must offer cost-effective items to expand and survive in an increasingly competitive and rising market environment.

One of the most common business methods used by firms in the nucleic acid-based therapeutics industry to benefit clients and expand the market sector is manufacturing locally to reduce operating costs. In recent years, the nucleic acid-based therapeutics industry has provided medicine with some of the most significant benefits. In the nucleic acid-based therapeutics market, major players such as Wave Life Sciences Ltd, Copernicus Therapeutics Inc, Imugene, Caperna, Phylogica, Protagonist Therapeutics, and others are engaging in research and development operations to increase the market demand.

Wave Life Sciences Ltd is a biotechnology company that develops nucleic acid-based therapeutics to treat genetic diseases. The company's proprietary platform allows for designing and optimizing stereo pure nucleic acid therapies designed to target disease-causing genes while leaving healthy genes unaffected selectively. For instance, in December 2022, GSK plc and Wave Life Sciences Ltd. (Nasdaq WVE), a clinical-stage genetic medicine company dedicated to providing life-altering treatments for patients battling devastating diseases, today announced a strategic partnership to advance oligonucleotide therapeutics, including Wave's preclinical RNA editing program targeting alpha-1 antitrypsin deficiency (AATD), WVE-006.

Also, Copernicus Therapeutics Inc is a biotechnology company focused on developing nucleic acid-based therapeutics for treating genetic and acquired diseases. The company's proprietary platform is based on a non-viral gene delivery technology that allows for the efficient and targeted delivery of nucleic acid-based therapies to specific cells and tissues.

Key Companies in the nucleic acid-based therapeutics market includes

  • Wave Life Sciences Ltd

  • Copernicus Therapeutics Inc

  • Imugene

  • Caperna

  • Phylogica

  • Protagonist Therapeutics

  • Benitec Biopharma

  • EGEN (Expression Genetics)

  • Benitec Biopharma

  • BioMedica (Oxford BioMedica)

  • Transgene

Nucleic Acid-based Therapeutics Industry Developments

August 2022 Merck & Co. and Orna Therapeutics, Inc. will develop and commercialize a novel class of therapeutic RNA for the treatment of infectious and oncological disorders.

January 2022 The bivalent COVID-19 vaccine modified for Omicron BA.4/BA.5 has begun a study for the treatment of infection, according to Pfizer Inc. and BioNTech SE.

Nucleic Acid-based Therapeutics Market Segmentation

Nucleic Acid-based Therapeutics Technology Outlook

  • Anti-Sense and Anti-Gene

  • Short Inhibitory Sequences

  • Gene Transfer Therapy

  • Nucleoside Analogs

  • Ribozymes

  • Aptamers

Nucleic Acid-based Therapeutics Application Outlook

  • Monogenetic disorders

  • Thalassemia

  • Sickle cell anemia

  • Hemophilia

  • Cystic Fibrosis

  • Multi-genetic disorders

  • Cancer

  • Diabetes

  • Neurodegenerative Diseases

  • cardiovascular Diseases etc.

Nucleic Acid-based Therapeutics End Users Outlook

  • Hospitals

  • Academic & Research Institutes

Nucleic Acid-based Therapeutics Regional Outlook

  • North America

    • US

    • Canada

  • Europe

    • Germany

    • France

    • UK

    • Italy

    • Spain

    • Rest of Europe

  • Asia-Pacific

    • China

    • Japan

    • India

    • Australia

    • South Korea

    • Australia

    • Rest of Asia-Pacific

  • Rest of the World

    • Middle East

    • Africa

    • Latin America

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