This Nucleic Acid Based Gene Therapy market report provides a comprehensive analysis of the market’s characteristics, size, and growth, including segmentation, regional and country-level breakdowns, competitive landscape, market shares, trends, and strategies. It also tracks historical and forecasted market growth across various geographies.
The nucleic acid based gene therapy market size has grown strongly in recent years. It will grow from $0.81 billion in 2024 to $0.88 billion in 2025 at a compound annual growth rate (CAGR) of 9.4%. The growth in the historic period can be attributed to growing understanding of human genetics, growing demand for personalized medicine, expanding pipeline of gene therapy products, growing government support for gene therapy, genetic disorders, cancer therapy.
The nucleic acid based gene therapy market size is expected to see rapid growth in the next few years. It will grow to $1.33 billion in 2029 at a compound annual growth rate (CAGR) of 10.7%. The growth in the forecast period can be attributed to expanding indications, increased funding, patient advocacy and awareness, growing investment in gene therapy research and development, expanding scope of gene therapy applications, rare diseases. Major trends in the forecast period include ex vivo gene editing, advances in in vivo gene editing, combination therapies, targeted delivery systems, gene editing for rare diseases, non-viral vectors.
The nucleic acid-based gene therapy market is expected to experience increased demand due to growing financial support from both governments and companies. Nucleic acid therapeutics, which mimic naturally occurring acids or proteins responsible for genetic expression, offer a promising approach to treating diseases such as cystic fibrosis, hemophilia, sickle cell anemia, thalassemia, and diabetes, for which traditional therapies lack a cure. These therapies target the genetic basis of diseases and hold the potential for permanent solutions. For example, the National Institute of Health (NIH) announced a $34 million annual investment over five years in August 2022 to advance HIV treatment research. Such government support and investments in gene therapies are expected to boost the nucleic acid-based gene therapy market.
The expanding healthcare expenditure is set to drive the future growth of the nucleic acid gene therapy market. Healthcare expenditure encompasses the total spending on healthcare-related goods and services within a healthcare system or economy and plays a critical role in supporting the development and accessibility of nucleic acid gene therapy. In 2022, healthcare expenditure in the United States increased by 0.8%, reaching $331 billion, up from 7.6% in 2021, as reported by the Canadian Institute for Health Information. This rise in healthcare spending contributes to the growth of the nucleic acid gene therapy market.
Companies operating in the nucleic acid-based gene therapy market are strategically pursuing partnerships and collaborations to enhance production and expand their market reach. Major players are joining forces to leverage their resources and explore new avenues for research and development. For example, Pfizer Inc. and Beam Therapeutics Inc. announced an exclusive four-year research collaboration in January 2022, focused on in vivo base editing programs targeting rare genetic diseases of the liver, muscle, and central nervous system. Similarly, in August 2022, Merck and Orna Therapeutics entered into a collaboration agreement to discover, develop, and commercialize programs, including vaccines and therapeutics, in the areas of infectious disease and oncology, utilizing Orna Therapeutics' engineered circular RNA (oRNA) therapies.
In March 2023, Moderna, Inc., a US-based biotechnology company, announced a partnership with Generation Bio. This collaboration aims to develop innovative nucleic acid therapeutics, including therapies designed to target immune cells, to advance both companies' pipelines of non-viral genetic medicines. Under the agreement, Moderna will fund all research and development activities related to this partnership, including a pre-payment to support research efforts. Generation Bio, also a US-based biotechnology company, specializes in nucleic acid-based gene therapies.
Major companies operating in the nucleic acid based gene therapy market include Celsion Corporation, Wave Life Sciences, Imugene Ltd., Caperna LLC, Phylogica Limited, Protagonist Therapeutics Inc., Benitec Biopharma Ltd., EGEN, BioMedica, Transgene SA, Copernicus Therapeutics Inc., 4D Molecular Therapeutics Inc., Abeona Therapeutics Inc., Alnylam Pharmaceuticals Inc., Amgen Inc., Beam Therapeutics Inc., BioNTech SE, Bluebird Bio Inc., CRISPR Therapeutics AG, Editas Medicine Inc., Genevant Sciences Inc., Homology Medicines Inc., Intellia Therapeutics Inc., Ionis Pharmaceuticals Inc., MaxCyte Inc., Moderna Inc., Novartis International AG, Orchard Therapeutics PLC, Precision BioSciences Inc., REGENXBIO Inc.
Gene therapy aims to correct faulty genes responsible for disease development, while nucleic acid-based therapeutics are employed to address genetic disorders and conditions without a definitive cure, such as anemia, sickle cell disease, cystic fibrosis, and thalassemia.
The key technologies include anti-sense and anti-gene strategies, short inhibitory sequences, gene transfer therapy, nucleoside analogues, ribozymes, aptamers, and other innovative approaches. Nucleoside analogues, a significant class of antiviral agents, are commonly utilized to combat conditions like human immunodeficiency virus (HIV), hepatitis B virus (HBV), hepatitis C virus (HCV), and cytomegalovirus (CMV) infections. These therapeutic approaches find applications in various medical fields, including oncology, the management of conditions like muscular dystrophy and muscular disorders, and the treatment of rare diseases. They are implemented by end-users, including hospitals, clinics, academic institutions, and research centers, where cutting-edge research and development efforts are often carried out.
The nucleic acid-based gene therapy market research report is one of a series of new reports that provides nucleic acid-based gene therapy market statistics, including nucleic acid-based gene therapy industry global market size, regional shares, competitors with a nucleic acid-based gene therapy market share, detailed nucleic acid-based gene therapy market segments, market trends and opportunities, and any further data you may need to thrive in the nucleic acid-based gene therapy industry. This nucleic acid-based gene therapy market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenario of the industry.
North America was the largest region in the nucleic acid-based gene therapy market in 2024. Western Europe was the second-largest region in the nucleic acid-based gene therapy market report. The regions covered in the nucleic acid based gene therapy market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, and Africa. The countries covered in the nucleic acid based gene therapy market report are Australia, China, India, Indonesia, Japan, South Korea, Bangladesh, Thailand, Vietnam, Malaysia, Singapore, Philippines, Hong Kong, New Zealand, USA, Canada, Mexico, Brazil, Chile, Argentina, Colombia, Peru, France, Germany, UK, Austria, Belgium, Denmark, Finland, Ireland, Italy, Netherlands, Norway, Portugal, Spain, Sweden, Switzerland, Russia, Czech Republic, Poland, Romania, Ukraine, Saudi Arabia, Israel, Iran, Turkey, UAE, Egypt, Nigeria, South Africa.
The nucleic acid based gene therapy market consists of sales of nucleic acid based gene therapy technologies such as anti-sense and anti-gene, short inhibitory sequences, gene transfer therapy, nucleoside analogs, ribozymes, aptamers, and other technologies. Values in this market are factory gate values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors, and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.
The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).
The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.
This product will be delivered within 3-5 business days.
The nucleic acid based gene therapy market size has grown strongly in recent years. It will grow from $0.81 billion in 2024 to $0.88 billion in 2025 at a compound annual growth rate (CAGR) of 9.4%. The growth in the historic period can be attributed to growing understanding of human genetics, growing demand for personalized medicine, expanding pipeline of gene therapy products, growing government support for gene therapy, genetic disorders, cancer therapy.
The nucleic acid based gene therapy market size is expected to see rapid growth in the next few years. It will grow to $1.33 billion in 2029 at a compound annual growth rate (CAGR) of 10.7%. The growth in the forecast period can be attributed to expanding indications, increased funding, patient advocacy and awareness, growing investment in gene therapy research and development, expanding scope of gene therapy applications, rare diseases. Major trends in the forecast period include ex vivo gene editing, advances in in vivo gene editing, combination therapies, targeted delivery systems, gene editing for rare diseases, non-viral vectors.
The nucleic acid-based gene therapy market is expected to experience increased demand due to growing financial support from both governments and companies. Nucleic acid therapeutics, which mimic naturally occurring acids or proteins responsible for genetic expression, offer a promising approach to treating diseases such as cystic fibrosis, hemophilia, sickle cell anemia, thalassemia, and diabetes, for which traditional therapies lack a cure. These therapies target the genetic basis of diseases and hold the potential for permanent solutions. For example, the National Institute of Health (NIH) announced a $34 million annual investment over five years in August 2022 to advance HIV treatment research. Such government support and investments in gene therapies are expected to boost the nucleic acid-based gene therapy market.
The expanding healthcare expenditure is set to drive the future growth of the nucleic acid gene therapy market. Healthcare expenditure encompasses the total spending on healthcare-related goods and services within a healthcare system or economy and plays a critical role in supporting the development and accessibility of nucleic acid gene therapy. In 2022, healthcare expenditure in the United States increased by 0.8%, reaching $331 billion, up from 7.6% in 2021, as reported by the Canadian Institute for Health Information. This rise in healthcare spending contributes to the growth of the nucleic acid gene therapy market.
Companies operating in the nucleic acid-based gene therapy market are strategically pursuing partnerships and collaborations to enhance production and expand their market reach. Major players are joining forces to leverage their resources and explore new avenues for research and development. For example, Pfizer Inc. and Beam Therapeutics Inc. announced an exclusive four-year research collaboration in January 2022, focused on in vivo base editing programs targeting rare genetic diseases of the liver, muscle, and central nervous system. Similarly, in August 2022, Merck and Orna Therapeutics entered into a collaboration agreement to discover, develop, and commercialize programs, including vaccines and therapeutics, in the areas of infectious disease and oncology, utilizing Orna Therapeutics' engineered circular RNA (oRNA) therapies.
In March 2023, Moderna, Inc., a US-based biotechnology company, announced a partnership with Generation Bio. This collaboration aims to develop innovative nucleic acid therapeutics, including therapies designed to target immune cells, to advance both companies' pipelines of non-viral genetic medicines. Under the agreement, Moderna will fund all research and development activities related to this partnership, including a pre-payment to support research efforts. Generation Bio, also a US-based biotechnology company, specializes in nucleic acid-based gene therapies.
Major companies operating in the nucleic acid based gene therapy market include Celsion Corporation, Wave Life Sciences, Imugene Ltd., Caperna LLC, Phylogica Limited, Protagonist Therapeutics Inc., Benitec Biopharma Ltd., EGEN, BioMedica, Transgene SA, Copernicus Therapeutics Inc., 4D Molecular Therapeutics Inc., Abeona Therapeutics Inc., Alnylam Pharmaceuticals Inc., Amgen Inc., Beam Therapeutics Inc., BioNTech SE, Bluebird Bio Inc., CRISPR Therapeutics AG, Editas Medicine Inc., Genevant Sciences Inc., Homology Medicines Inc., Intellia Therapeutics Inc., Ionis Pharmaceuticals Inc., MaxCyte Inc., Moderna Inc., Novartis International AG, Orchard Therapeutics PLC, Precision BioSciences Inc., REGENXBIO Inc.
Gene therapy aims to correct faulty genes responsible for disease development, while nucleic acid-based therapeutics are employed to address genetic disorders and conditions without a definitive cure, such as anemia, sickle cell disease, cystic fibrosis, and thalassemia.
The key technologies include anti-sense and anti-gene strategies, short inhibitory sequences, gene transfer therapy, nucleoside analogues, ribozymes, aptamers, and other innovative approaches. Nucleoside analogues, a significant class of antiviral agents, are commonly utilized to combat conditions like human immunodeficiency virus (HIV), hepatitis B virus (HBV), hepatitis C virus (HCV), and cytomegalovirus (CMV) infections. These therapeutic approaches find applications in various medical fields, including oncology, the management of conditions like muscular dystrophy and muscular disorders, and the treatment of rare diseases. They are implemented by end-users, including hospitals, clinics, academic institutions, and research centers, where cutting-edge research and development efforts are often carried out.
The nucleic acid-based gene therapy market research report is one of a series of new reports that provides nucleic acid-based gene therapy market statistics, including nucleic acid-based gene therapy industry global market size, regional shares, competitors with a nucleic acid-based gene therapy market share, detailed nucleic acid-based gene therapy market segments, market trends and opportunities, and any further data you may need to thrive in the nucleic acid-based gene therapy industry. This nucleic acid-based gene therapy market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenario of the industry.
North America was the largest region in the nucleic acid-based gene therapy market in 2024. Western Europe was the second-largest region in the nucleic acid-based gene therapy market report. The regions covered in the nucleic acid based gene therapy market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, and Africa. The countries covered in the nucleic acid based gene therapy market report are Australia, China, India, Indonesia, Japan, South Korea, Bangladesh, Thailand, Vietnam, Malaysia, Singapore, Philippines, Hong Kong, New Zealand, USA, Canada, Mexico, Brazil, Chile, Argentina, Colombia, Peru, France, Germany, UK, Austria, Belgium, Denmark, Finland, Ireland, Italy, Netherlands, Norway, Portugal, Spain, Sweden, Switzerland, Russia, Czech Republic, Poland, Romania, Ukraine, Saudi Arabia, Israel, Iran, Turkey, UAE, Egypt, Nigeria, South Africa.
The nucleic acid based gene therapy market consists of sales of nucleic acid based gene therapy technologies such as anti-sense and anti-gene, short inhibitory sequences, gene transfer therapy, nucleoside analogs, ribozymes, aptamers, and other technologies. Values in this market are factory gate values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors, and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.
The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).
The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.
This product will be delivered within 3-5 business days.
Executive Summary
Nucleic Acid Based Gene Therapy Global Market Report 2025 provides strategists, marketers and senior management with the critical information they need to assess the market.This report focuses on nucleic acid based gene therapy market which is experiencing strong growth. The report gives a guide to the trends which will be shaping the market over the next ten years and beyond.
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Description
Where is the largest and fastest growing market for nucleic acid based gene therapy ? How does the market relate to the overall economy, demography and other similar markets? What forces will shape the market going forward? The nucleic acid based gene therapy market global report answers all these questions and many more.The report covers market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the market’s historic and forecast market growth by geography.
- The market characteristics section of the report defines and explains the market.
- The market size section gives the market size ($b) covering both the historic growth of the market, and forecasting its development.
- The forecasts are made after considering the major factors currently impacting the market. These include: the Russia-Ukraine war, rising inflation, higher interest rates, and the legacy of the COVID-19 pandemic.
- Market segmentations break down the market into sub markets.
- The regional and country breakdowns section gives an analysis of the market in each geography and the size of the market by geography and compares their historic and forecast growth. It covers the growth trajectory of COVID-19 for all regions, key developed countries and major emerging markets.
- The competitive landscape chapter gives a description of the competitive nature of the market, market shares, and a description of the leading companies. Key financial deals which have shaped the market in recent years are identified.
- The trends and strategies section analyses the shape of the market as it emerges from the crisis and suggests how companies can grow as the market recovers.
Scope
Markets Covered:
1) By Technology: Anti-Sence and Anti-Gene; Short Inhibitory Sequences; Gene Transfer Therapy; Nucleoside Analogs; Ribozymes; Aptamers; Other Technologies2) By Application: Oncology; Muscular Dystrophy/ Muscular Disorders; Rare Diseases
3) By End User: Hospitals and Clinics; Academic and Research Institutes
Subsegments:
1) By Anti-Sense and Anti-Gene: Oligonucleotide Therapeutics; Peptide Nucleic Acids (PNA); Morpholino Oligomers (MO)2) By Short Inhibitory Sequences (siRNA): Synthetic siRNA; Encapsulation/Delivery Systems for siRNA; Target-specific siRNA Therapies
3) By Gene Transfer Therapy: Viral Vectors (Adenovirus, Lentivirus, Retrovirus, AAV); Non-Viral Vectors (Liposomes, Electroporation, Gene Gun)
4) By Nucleoside Analogs: Purine Analogs; Pyrimidine Analogs
5) By Ribozymes: Trans-Splicing Ribozymes; Hammerhead Ribozymes
6) By Aptamers: RNA Aptamers; DNA Aptamers; Small Molecule Binding Aptamers
7) By Other Technologies: CRISPR-based Gene Editing; Zinc Finger Nucleases (ZFN); Transcription Activator-Like Effector Nucleases (TALENs)
Key Companies Mentioned: Celsion Corporation; Wave Life Sciences; Imugene Ltd.; Caperna LLC; Phylogica Limited
Countries: Australia; China; India; Indonesia; Japan; South Korea; Bangladesh; Thailand; Vietnam; Malaysia; Singapore; Philippines; Hong Kong; New Zealand; USA; Canada; Mexico; Brazil; Chile; Argentina; Colombia; Peru; France; Germany; UK; Austria; Belgium; Denmark; Finland; Ireland; Italy; Netherlands; Norway; Portugal; Spain; Sweden; Switzerland; Russia; Czech Republic; Poland; Romania; Ukraine; Saudi Arabia; Israel; Iran; Turkey; UAE; Egypt; Nigeria; South Africa
Regions: Asia-Pacific; Western Europe; Eastern Europe; North America; South America; Middle East; Africa
Time Series: Five years historic and ten years forecast.
Data: Ratios of market size and growth to related markets, GDP proportions, expenditure per capita.
Data Segmentation: Country and regional historic and forecast data, market share of competitors, market segments.
Sourcing and Referencing: Data and analysis throughout the report is sourced using end notes.
Delivery Format: PDF, Word and Excel Data Dashboard.
Companies Mentioned
The major companies featured in this Nucleic Acid Based Gene Therapy market report include:- Celsion Corporation
- Wave Life Sciences
- Imugene Ltd.
- Caperna LLC
- Phylogica Limited
- Protagonist Therapeutics Inc.
- Benitec Biopharma Ltd.
- EGEN
- BioMedica
- Transgene SA
- Copernicus Therapeutics Inc.
- 4D Molecular Therapeutics Inc.
- Abeona Therapeutics Inc.
- Alnylam Pharmaceuticals Inc.
- Amgen Inc.
- Beam Therapeutics Inc.
- BioNTech SE
- Bluebird Bio Inc.
- CRISPR Therapeutics AG
- Editas Medicine Inc.
- Genevant Sciences Inc.
- Homology Medicines Inc.
- Intellia Therapeutics Inc.
- Ionis Pharmaceuticals Inc.
- MaxCyte Inc.
- Moderna Inc.
- Novartis International AG
- Orchard Therapeutics plc
- Precision BioSciences Inc.
- REGENXBIO Inc.
Table Information
| Report Attribute | Details |
|---|---|
| No. of Pages | 250 |
| Published | April 2025 |
| Forecast Period | 2025 - 2029 |
| Estimated Market Value ( USD | $ 0.88 Billion |
| Forecasted Market Value ( USD | $ 1.33 Billion |
| Compound Annual Growth Rate | 10.7% |
| Regions Covered | Global |
| No. of Companies Mentioned | 31 |
