Nucleic Acid And Gene Therapies In Neuromuscular Disorders Global Market Report 2025

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 $7.52 billion in 2025 at a compound annual growth rate (CAGR) of 9.9%. The growth during the historic period can be attributed to an increase in regulatory approvals, rising demand for nucleic acid therapeutics, a growing need for more precise and effective treatments, heightened demand for gene therapies, and an increasing 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 $10.87 billion in 2029 at a compound annual growth rate (CAGR) of 9.7%. The growth during the forecast period can be driven by the rising prevalence of genetic disorders, increased investments in research and development, growing demand for precision medicine, a surge in gene therapy product approvals, and a higher incidence of chronic disorders. Key trends in this period include advancements in patient care, high-performance chromatography, innovative treatment solutions, progress in gene editing technologies, and the availability of advanced nucleic acid extraction technology.

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., Généthon 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.

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.

The nucleic acid and gene therapies in neuromuscular disorders market research report is one of a series of new reports 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 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.

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