PUBLISHER: The Business Research Company | PRODUCT CODE: 1826958
PUBLISHER: The Business Research Company | PRODUCT CODE: 1826958
Nucleic acid and gene therapies for neuromuscular disorders are innovative treatments designed to correct or compensate for genetic mutations that cause these conditions. Neuromuscular disorders (NMDs) affect the muscles and nerves, often due to inherited mutations. These therapies utilize nucleic acids or genetic material to repair, replace, or modulate gene expression, addressing the underlying cause of the disorder.
The primary disorders targeted by these therapies include motor neuron diseases, neuropathies, neuromuscular junction disorders, and myopathies such as muscular dystrophies. Motor neuron diseases are a group of progressive neurological conditions that impact motor neurons, leading to muscle weakness, atrophy, and loss of voluntary movement control. Treatment approaches include adeno-associated virus (AAV) gene therapy, postnatal gene therapy, and therapies for spinal muscular atrophy. These therapies are applied in various healthcare settings, including hospitals, specialty clinics, and ambulatory surgery centers.
Note that the outlook for this market is being affected by rapid changes in trade relations and tariffs globally. The report will be updated prior to delivery to reflect the latest status, including revised forecasts and quantified impact analysis. The report's Recommendations and Conclusions sections will be updated to give strategies for entities dealing with the fast-moving international environment.
The sharp rise in U.S. tariffs and the resulting trade tensions in spring 2025 are having a significant impact on the healthcare sector, especially in the supply of essential medical devices, diagnostic equipment, and pharmaceuticals. Hospitals and healthcare providers are grappling with higher costs for imported surgical tools, imaging systems, and consumables like syringes and catheters, many of which have limited domestic substitutes. These escalating expenses are putting pressure on healthcare budgets, prompting some providers to delay equipment upgrades or pass increased costs on to patients. Furthermore, tariffs on raw materials and components are disrupting the manufacturing of vital drugs and devices, leading to supply chain delays. In response, the industry is adopting diversified sourcing strategies, expanding local production where feasible, and pushing for tariff exemptions on critical medical products.
The nucleic acid and gene therapies in neuromuscular disorders market research report is one of a series of new reports from The Business Research Company that provides nucleic acid and gene therapies in neuromuscular disorders market statistics, including nucleic acid and gene therapies in neuromuscular disorders industry global market size, regional shares, competitors with a nucleic acid and gene therapies in neuromuscular disorders market share, detailed nucleic acid and gene therapies in neuromuscular disorders market segments, market trends and opportunities, and any further data you may need to thrive in the nucleic acid and gene therapies in neuromuscular disorders industry. This nucleic acid and gene therapies in neuromuscular disorders 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 and gene therapies in neuromuscular disorders market size has grown strongly in recent years. It will grow from $6.84 billion in 2024 to $7.5 billion in 2025 at a compound annual growth rate (CAGR) of 9.6%. The growth in the historic period can be attributed to increase in regulatory approvals, demand for nucleic acid therapeutics, demand for more precise and effective therapies, demand for effective gene therapies, and increase in the number of gene therapy-based discoveries.
The nucleic acid and gene therapies in neuromuscular disorders market size is expected to see strong growth in the next few years. It will grow to $10.78 billion in 2029 at a compound annual growth rate (CAGR) of 9.5%. The growth in the forecast period can be attributed to increasing prevalence of genetic disorders, increasing investments in research and development, growing demand for precision medicine, rising approval of gene therapy products, and increasing prevalence of chronic disorders. Major trends in the forecast period include advances in patient care, high performance chromatography, innovative solutions for conditions, advancements in gene editing technologies, and availability of nucleic acid extraction technology.
The forecast of 9.5% growth over the next five years reflects a slight reduction of 0.2% from the previous projection. This reduction is primarily due to the impact of tariffs between the US and other countries. The imposition of tariffs may disrupt U.S. medical innovation by increasing costs for Adeno-associated virus vector-based therapies and Clustered regularly interspaced short palindromic repeats -Cas9 components imported from Switzerland and South Korea, potentially raising specialty pharmacy costs and postponing rare disease treatments. The effect will also be felt more widely due to reciprocal tariffs and the negative effect on the global economy and trade due to increased trade tensions and restrictions.
The increasing prevalence of genetic disorders is expected to drive the growth of the nucleic acid and gene therapies in the neuromuscular disorders market. Genetic disorders arise from abnormalities in an individual's DNA, either inherited or resulting from mutations. The rise in these disorders is attributed to factors such as genetic mutations, environmental influences, lifestyle changes, and advancements in diagnostic technologies. Nucleic acid and gene therapies play a crucial role in addressing genetic neuromuscular disorders by correcting or replacing defective genes to restore normal function. For example, in May 2024, according to Cure SMA, a US-based non-profit organization, approximately 9,000 to 9,500 individuals in the United States are currently living with Spinal Muscular Atrophy (SMA), with Type 2 SMA accounting for 37% of cases. The estimated incidence rate of SMA is approximately 1 in 15,000 births. As a result, the growing prevalence of genetic disorders is fueling the demand for nucleic acid and gene therapies in the neuromuscular disorders market.
Leading companies in the market are advancing gene therapy solutions to directly target the root causes of genetic neuromuscular conditions through precise gene correction or replacement. Gene therapy involves modifying or repairing genetic material within a patient's cells to address defective genes. For instance, in June 2023, Sarepta Therapeutics Inc., a US-based biotechnology company, received FDA approval for Elevidys, the first gene therapy for Duchenne Muscular Dystrophy (DMD) in ambulatory pediatric patients aged 4 to 5 years. Elevidys is administered as a single intravenous infusion, offering a simplified alternative to ongoing infusion-based therapies. This therapy delivers a gene encoding a shortened form of dystrophin, known as micro-dystrophin, which is crucial for muscle function. By enabling muscle cells to produce a functional dystrophin protein, Elevidys aims to slow or potentially reverse disease progression.
In November 2024, Novartis AG, a Switzerland-based pharmaceutical company, acquired Kate Therapeutics Inc. for $1.1 billion. This acquisition aims to strengthen Novartis' gene therapy portfolio for inherited neuromuscular diseases. By integrating Kate Therapeutics' AAV-based gene therapy platform DELIVER and its preclinical candidates for Duchenne muscular dystrophy, facioscapulohumeral dystrophy, and myotonic dystrophy type 1, Novartis is expanding its capabilities in developing targeted therapies for genetic muscle and heart diseases. Kate Therapeutics Inc. is a US-based biotechnology company specializing in adeno-associated virus (AAV)-based gene therapies for genetically defined neuromuscular conditions.
Major players in the nucleic acid and gene therapies in neuromuscular disorders market are Pfizer Inc., F. Hoffmann-La Roche Ltd., Bayer AG, Sanofi S.A, Novartis AG, Eli Lilly and Company, Astellas Pharma Inc, Biogen Inc, Vertex Pharmaceuticals Inc, BioMarin Pharmaceuticals Inc, Nippon Shinyaku Co Ltd, Sarepta Therapeutics Inc, Ionis Pharmaceuticals, Inc., Ultragenyx Pharmaceutical Inc, Amicus Therapeutics Inc, Arrowhead Pharmaceuticals Inc, Regenxbio Inc, UniQure N.V., Genethon S.A, Axovant Gene Therapies Inc .
North America was the largest region in the nucleic acid and gene therapies in neuromuscular disorders market in 2024. The regions covered in nucleic acid and gene therapies in neuromuscular disorders report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa.
The countries covered in the nucleic acid and gene therapies in neuromuscular disorders market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.
The nucleic acid and gene therapies in neuromuscular disorders market consist of revenue earned by entities by providing services such as genetic diagnostics, personalized therapeutics, and gene delivery. The market value includes the value of related goods sold by the service provider or included within the service offering. The nucleic acid and gene therapies in neuromuscular disorders market also include sales of oligonucleotides, gene therapy vectors, and diagnostic kits. 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.
Nucleic Acid And Gene Therapies In Neuromuscular Disorders Global Market Report 2025 from The Business Research Company provides strategists, marketers and senior management with the critical information they need to assess the market.
This report focuses on nucleic acid and gene therapies in neuromuscular disorders 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.
Where is the largest and fastest growing market for nucleic acid and gene therapies in neuromuscular disorders ? How does the market relate to the overall economy, demography and other similar markets? What forces will shape the market going forward, including technological disruption, regulatory shifts, and changing consumer preferences? The nucleic acid and gene therapies in neuromuscular disorders market global report from the Business Research Company 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 forecasts are made after considering the major factors currently impacting the market. These include the technological advancements such as AI and automation, Russia-Ukraine war, trade tariffs (government-imposed import/export duties), elevated inflation and interest rates.