Gene Therapy on Neurological Diseases Market Report 2026

The gene therapy on neurological diseases market size has grown rapidly in recent years. It will grow from $3.55 billion in 2025 to $4.01 billion in 2026 at a compound annual growth rate (CAGR) of 13%. The growth in the historic period can be attributed to limited gene therapy options for neurological diseases, growing prevalence of alzheimer's and parkinsons disease, reliance on conventional treatment methods, increasing awareness of genetic approaches, early clinical trials of viral vectors.

The gene therapy on neurological diseases market size is expected to see rapid growth in the next few years. It will grow to $6.48 billion in 2030 at a compound annual growth rate (CAGR) of 12.7%. The growth in the forecast period can be attributed to development of adeno associated virus and lentiviral therapies, expansion of non-viral delivery platforms, increasing regulatory approvals for neurological gene therapies, rising investment in r&d for targeted genetic interventions, integration of digital monitoring and AI-based patient management. Major trends in the forecast period include rising investment in viral and non-viral gene therapy platforms, expansion of gene therapy applications across neurological disorders, increasing focus on personalized and targeted genetic treatments, growth in hospital and clinic-based gene therapy programs, development of advanced delivery methods for neurological gene therapy.

The rising prevalence of neurological disorders is expected to drive the growth of the gene therapy for neurological diseases market in the coming years. Neurological disorders are medical conditions that affect the brain, spinal cord, and nerves, causing a variety of cognitive, motor, and sensory impairments. The increasing prevalence of these disorders is closely linked to aging, which gradually impairs neuronal function, reduces brain plasticity, and increases oxidative stress, contributing to disease onset and progression. Gene therapy for neurological diseases supports the management of these conditions by addressing underlying genetic causes, offering the potential for long-term or permanent treatment solutions. It improves patient outcomes by restoring or modifying gene function, alleviating symptoms, and minimizing the need for repeated interventions, thereby enhancing overall quality of life. For example, according to the National Health Service in the UK, by June 30, 2024, there were 487,432 patients diagnosed with dementia - a progressive brain disorder affecting memory, thinking, and daily activities - representing an increase of 3,155 cases compared to May 31, 2024. This growing prevalence of neurological disorders is thus supporting the expansion of the gene therapy for neurological diseases market.

Leading companies in this market are focusing on advanced viral delivery systems, such as adeno-associated virus serotype 2 (AAV2) vectors, to improve targeted gene delivery, enhance efficacy, reduce immune responses, and enable long-lasting treatments. AAV2 vectors are modified, non-pathogenic viral systems used in gene therapy to transport therapeutic genes into target cells, particularly neurons, for sustained expression. For instance, in November 2024, PTC Therapeutics Inc., a US-based biopharmaceutical company, received FDA approval for a gene therapy to treat aromatic L-amino acid decarboxylase (AADC) deficiency. This therapy uses an AAV2 vector to deliver a functional copy of the DDC gene into the putamen, restoring dopamine production and improving motor function. The therapy is administered as a one-time intraputaminal infusion, providing long-term therapeutic benefits and significantly reducing debilitating symptoms without repeated interventions.

In January 2023, Neurocrine Biosciences Inc., a US-based biotechnology company, partnered with Voyager Therapeutics Inc. to advance next-generation gene therapies for neurological diseases. Through this collaboration, Neurocrine and Voyager aim to accelerate development and commercialization by combining Voyager’s TRACER capsid platform-enabled GBA1 gene therapy and other CNS-targeted AAV programs with Neurocrine’s neuroscience and clinical expertise. The partnership allows for risk-sharing, cost and profit alignment, and the pursuit of novel treatments for Parkinson’s disease, Friedreich’s ataxia, and other rare CNS disorders. Voyager Therapeutics Inc., based in the US, specializes in developing AAV-based gene therapies with enhanced blood-brain barrier penetration for central nervous system conditions.

Major companies operating in the gene therapy on neurological diseases market are Novartis AG, Biogen Inc., Sarepta Therapeutics Inc., PTC Therapeutics Inc., Oxford Biomedica plc, Regenxbio Inc., Passage Bio Inc., Intellia Therapeutics Inc., Voyager Therapeutics Inc., UniQure N.V., AviadoBio Ltd., Capsida Biotherapeutics Inc., StrideBio Inc., Lysogene S.A., Wave Life Sciences Ltd., Taysha Gene Therapies Inc., Abeona Therapeutics Inc., CRISPR Therapeutics AG.

North America was the largest region in the gene therapy on neurological diseases market in 2025. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in the gene therapy on neurological diseases market report are Asia-Pacific, South East Asia, Western Europe, Eastern Europe, North America, South America, Middle East, Africa. The countries covered in the gene therapy on neurological diseases market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Taiwan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

Tariffs have impacted the gene therapy for neurological diseases market by increasing the cost of imported viral vectors, nanoparticles, and specialized delivery equipment, leading to higher treatment costs and supply chain disruptions. Segments like viral gene therapies and advanced non-viral delivery systems are most affected, particularly in North America and Europe. On the positive side, tariffs have encouraged domestic production of vectors and delivery technologies, promoted local innovation, and accelerated development of cost-effective gene therapy solutions.

The gene therapy on neurological diseases market research report is one of a series of new reports that provides gene therapy on neurological diseases market statistics, including gene therapy on neurological diseases industry global market size, regional shares, competitors with a gene therapy on neurological diseases market share, detailed gene therapy on neurological diseases market segments, market trends and opportunities, and any further data you may need to thrive in the gene therapy on neurological diseases industry. This gene therapy on neurological diseases 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.

Gene therapy for neurological diseases involves using genetic material to treat or prevent disorders that affect the brain, spinal cord, and nervous system. This approach delivers corrective genes into a patient’s cells using viral or non-viral vectors to address the underlying genetic causes of neurological conditions. It aims to restore normal gene function, slow disease progression, or alleviate symptoms by targeting the root genetic causes rather than merely managing symptoms.

The main types of gene therapy for neurological diseases are viral gene therapy and non-viral gene therapy. Viral gene therapy uses engineered viruses as delivery vectors to transport therapeutic genes into a patient’s cells for the treatment or prevention of disease. These therapies target a range of neurological conditions, including Alzheimer’s disease, Parkinson’s disease, amyotrophic lateral sclerosis (ALS), Huntington’s disease, and multiple sclerosis. They can be administered via various routes, such as intravenous, intrathecal, intranasal, and intracerebral methods, and are applied in different settings, including hospitals, clinics, and other healthcare facilities.

The gene therapy on neurological diseases market consists of revenues earned by entities by providing services such as gene delivery and vector development, clinical trial services, personalised therapy development, regulatory consulting and compliance, and manufacturing support. The market value includes the value of related goods sold by the service provider or included within the service offering. The gene therapy on neurological diseases market also includes sales of gene therapy vectors, plasmid deoxyribonucleic acid (DNA), ribonucleic acid (RNA)-based therapeutics, cell lines and reagents, and neurologically targeted gene therapies. 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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