Nucleic Acid and Gene Therapies in Neuromuscular Disorders Market Report 2026

The nucleic acid and gene therapies in neuromuscular disorders market size has grown strongly in recent years. It will grow from $7.5 billion in 2025 to $8.22 billion in 2026 at a compound annual growth rate (CAGR) of 9.7%. The growth in the historic period can be attributed to limited therapeutic options for nmds, low awareness of gene therapies, high cost of gene therapy development, lack of specialized treatment centers, slow regulatory approvals.

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 $11.76 billion in 2030 at a compound annual growth rate (CAGR) of 9.4%. The growth in the forecast period can be attributed to advancements in adeno-associated virus and postnatal gene therapies, increasing r&d investments in nmds, growing number of hospitals and specialty clinics offering gene therapies, rising prevalence of neuromuscular disorders, increasing patient awareness and advocacy initiatives. Major trends in the forecast period include rising adoption of gene therapy techniques, increasing development of nucleic acid therapeutics, growing focus on rare neuromuscular disorders, rising investment in personalized medicine approaches, increasing integration of advanced diagnostic and monitoring tools.

The rising prevalence of genetic disorders is expected to drive the growth of the nucleic acid and gene therapies in neuromuscular disorders market in the coming years. Genetic disorders are caused by abnormalities or mutations in an individual’s DNA, which may be inherited or arise spontaneously. Factors such as genetic mutations, environmental influences, lifestyle changes, and improved diagnostic capabilities are contributing to the increasing incidence of these conditions. Nucleic acid and gene therapies offer targeted solutions by correcting or replacing defective genes in neuromuscular disorders, thereby restoring normal cellular function. For instance, 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 living with Spinal Muscular Atrophy (SMA), with 37% of cases classified as Type 2, and the condition occurs in an estimated 1 in 15,000 births. Therefore, the rising prevalence of genetic disorders is fueling the growth of this market.

Major companies in the nucleic acid and gene therapies market are developing innovative treatments such as gene therapy to address the underlying causes of neuromuscular disorders through precise gene correction or replacement. Gene therapy involves introducing, altering, or repairing genetic material within a patient’s cells to correct defective genes. For example, 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 delivered as a single intravenous infusion and provides a gene coding for micro-dystrophin, a shortened but functional version of the dystrophin protein, which is critical for muscle function. This therapy directly targets the genetic defect, potentially halting or reversing disease progression.

In November 2024, Novartis AG, a Switzerland-based pharmaceutical company, acquired Kate Therapeutics Inc. for $1.1 billion to strengthen its gene therapy pipeline for inherited neuromuscular diseases. Through this acquisition, Novartis integrates Kate Therapeutics’ AAV-based DELIVER gene therapy platform and preclinical candidates for Duchenne muscular dystrophy, facioscapulohumeral dystrophy, and myotonic dystrophy type 1 into its portfolio. Kate Therapeutics Inc., a US-based biotechnology company, specializes in developing adeno-associated virus (AAV)-based gene therapies for genetically defined muscle and heart disorders, positioning Novartis to expand its leadership in neuromuscular gene therapy.

Major companies operating in the nucleic acid and gene therapies in neuromuscular disorders market are Pfizer Inc, F. Hoffmann-La Roche Ltd, Sanofi S.A, Novartis AG, Biogen Inc, BioMarin Pharmaceuticals Inc, Sarepta Therapeutics Inc, Ionis Pharmaceuticals Inc, Ultragenyx Pharmaceutical Inc, Regenxbio Inc, UniQure N.V, Généthon S.A, Axovant Gene Therapies Inc, PTC Therapeutics Inc, Wave Life Sciences Ltd, CRISPR Therapeutics AG, Editas Medicine Inc, Helixmith Co Ltd, MeiraGTx Ltd, Cure Rare Disease, Beam Therapeutics Inc, Intellia Therapeutics Inc, Prime Medicine Inc, Arcturus Therapeutics Holdings Inc, Takeda Pharmaceutical Company Ltd.

North America was the largest region in the nucleic acid and gene therapies in neuromuscular disorders market in 2025. The regions covered in the nucleic acid and gene therapies in neuromuscular disorders market report are Asia-Pacific, South East Asia, Western Europe, Eastern Europe, North America, South America, Middle East, 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, Taiwan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

Tariffs have impacted the nucleic acid and gene therapies market by raising costs of imported viral vectors, nucleic acid materials, and specialized equipment, particularly affecting hospitals and specialty clinics in regions like north america, europe, and asia-pacific. These tariffs can slow the deployment of advanced therapies, though they also encourage local manufacturing and innovation, helping to establish domestic production of gene therapy components and reduce long-term dependence on imports.

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.

Nucleic acid and gene therapies for neuromuscular disorders are innovative treatments designed to correct or compensate for the genetic mutations that cause these conditions. Neuromuscular disorders (NMDs) affect muscles and nerves, often due to inherited genetic defects. These therapies use nucleic acids or genetic material to repair, replace, or modulate gene expression, targeting the underlying cause of the disorder.

The main disorders addressed by nucleic acid and gene therapies in neuromuscular conditions include motor neuron diseases, neuropathies, neuromuscular junction disorders, and myopathies such as muscular dystrophies. Motor neuron diseases are a group of progressive neurological disorders that damage motor neurons, resulting in muscle weakness, atrophy, and loss of voluntary movement control. Therapeutic approaches include adeno-associated virus (AAV) gene therapy, postnatal gene therapy, and treatments for spinal muscular atrophy. These therapies are applied in settings such as hospitals, specialty clinics, and ambulatory surgery centers.

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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