Nucleic Acid Based Gene Therapy Global Market Report 2024

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.

The nucleic acid based gene therapy market size has grown strongly in recent years. It will grow from $0.75 billion in 2023 to $0.82 billion in 2024 at a compound annual growth rate (CAGR) of 9.6%. The growth observed during the historic period in the field of gene therapy and nucleic acid-based therapeutics can be attributed to several key factors, including the increasing comprehension of human genetics, a rising demand for personalized medicine, the expansion of pipelines for gene therapy products, and growing government support for gene therapy, particularly in addressing genetic disorders and 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 $1.2 billion in 2028 at a compound annual growth rate (CAGR) of 10.1%. The expected growth in the forecast period in the field of gene therapy and nucleic acid-based therapeutics can be attributed to several factors, including the broadening of therapeutic indications, increased funding for research and development, the influence of patient advocacy and growing awareness, rising investments in gene therapy research, and the expanding scope of applications for gene therapy, particularly in addressing rare diseases. Noteworthy trends anticipated during the forecast period include advancements in ex vivo gene editing, in vivo gene editing techniques, the exploration of combination therapies, the development of targeted delivery systems, the use of gene editing for rare diseases, and the adoption of non-viral vectors to enhance the effectiveness of these therapies.

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 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.

The stringent regulations imposed on gene therapies can lead to increased prices for these therapies, posing a hindrance to the demand for nucleic acid-based gene therapies. The complex regulatory oversight results in expensive and time-consuming approval processes, significantly raising costs. Gene therapies, unlike other FDA-regulated drugs, are subject to additional regulatory bodies, such as the Recombinant DNA Advisory Committee and Office of Biotechnology Activities. According to estimates by the Foundation for Economic Education (FEE), the cost of developing an approved gene therapy drug is nearly $5.0 billion, which is five times higher than the average cost of FDA-approved drugs. These high costs make gene therapeutics less accessible to the general population and are likely to limit the growth of the nucleic acid-based gene therapy market.

Leading companies in the nucleic acid drugs market are actively developing innovative therapies, such as Casimersen, to drive market revenues. Casimersen, an antisense oligonucleotide (ASO), is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients with a specific mutation amenable to exon 45 skipping. Sarepta Therapeutics Inc., a US-based biotechnology company, launched Casimersen (Amondys 45) in February 2021, following FDA approval. This innovation is based on a significant increase in dystrophin production observed in patients treated with AMONDYS 45, with the potential for clinical benefits in those with exon 45 amenable mutations.

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.

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.

North America was the largest region in the nucleic acid-based gene therapy market in 2023. 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, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Italy, Spain, Canada.

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.


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