Gene Therapy on Neurological Diseases Market Report 2025

The gene therapy on neurological diseases market size has grown rapidly in recent years. It will grow from $3.13 billion in 2024 to $3.55 billion in 2025 at a compound annual growth rate (CAGR) of 13.2%. The growth during the historic period can be attributed to the increasing prevalence of neurological disorders, rising demand for personalized medicine, growing investments in biotechnology research and development, expanding clinical trial activities, and a rising number of approvals for gene therapies.

The gene therapy on neurological diseases market size is expected to see rapid growth in the next few years. It will grow to $5.76 billion in 2029 at a compound annual growth rate (CAGR) of 12.9%. The growth during the forecast period can be attributed to rising global clinical trial activity, expanding healthcare infrastructure, increasing awareness of rare diseases, a growing pipeline of gene therapies, and a surge in partnerships and collaborations. Key trends in the forecast period include the integration of technology in remote patient monitoring, innovations in vector manufacturing platforms, automation driven by advanced technologies in biomanufacturing, development of gene therapies for ultra-rare pediatric central nervous system (CNS) disorders, and the incorporation of genomic screening in clinical settings.

The increasing prevalence of neurological disorders is expected to drive growth in the gene therapy for neurological diseases market in the coming years. Neurological disorders affect the brain, spinal cord, and nerves, leading to cognitive, motor, and sensory impairments. Their prevalence is rising largely due to the aging population, as aging diminishes neuronal function, reduces brain plasticity, and increases oxidative stress, all of which contribute to the onset and progression of these conditions. Gene therapy offers a transformative approach by targeting the underlying genetic causes of neurological diseases, providing potential long-term or permanent treatment solutions. By restoring or modifying gene function, it helps reduce symptoms, improve outcomes, and minimize the need for repetitive treatments, thereby enhancing quality of life. For example, in July 2024, the National Health Service in the UK reported 487,432 patients diagnosed with dementia by June 30, 2024, an increase of 3,155 cases compared to May 31, 2024. This growing disease burden is fueling the expansion of the gene therapy for neurological diseases market.

Leading companies in this market are developing advanced viral delivery systems, such as adeno-associated virus serotype 2 (AAV2) vectors, to improve targeted gene delivery, enhance treatment efficacy, reduce immune responses, and enable durable therapeutic effects. AAV2 vectors are non-pathogenic viral carriers engineered to deliver therapeutic genes into neurons for long-term expression. For instance, in November 2024, PTC Therapeutics Inc., a US-based biopharmaceutical company, received FDA approval for a gene therapy treating aromatic L-amino acid decarboxylase (AADC) deficiency. This therapy uses an AAV2 vector to deliver a functional DDC gene into the putamen, restoring dopamine production and improving motor function. Administered as a one-time intraputaminal infusion, it provides sustained benefits, significantly improving mobility and reducing severe symptoms without the need for repeated interventions.

In November 2022, Kriya Therapeutics Inc., a US-based biopharmaceutical company, acquired Redpin Therapeutics Inc. for an undisclosed amount. This acquisition expanded Kriya’s neurology-focused gene therapy pipeline, adding Redpin’s chemogenetics platform and lead programs targeting epilepsy and trigeminal neuralgia. Redpin Therapeutics is a US-based biotechnology company developing innovative gene therapies for neurological diseases.

Major players in the gene therapy on neurological diseases market are Novartis AG, Biogen Inc., Neurocrine Biosciences Inc., Sarepta Therapeutics Inc., PTC Therapeutics Inc., Amicus 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 2024. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in gene therapy on neurological diseases market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa. The countries covered in the gene therapy on neurological diseases market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

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 sudden escalation of U.S. tariffs and the consequent trade frictions in spring 2025 are severely impacting the pharmaceutical companies contend with tariffs on APIs, glass vials, and lab equipment inputs with few alternative sources. Generic drug makers, operating on razor-thin margins, are especially vulnerable, with some reducing production of low-profit medicines. Biotech firms face delays in clinical trials due to tariff-related shortages of specialized reagents. In response, the industry is expanding API production in India and Europe, increasing inventory stockpiles, and pushing for trade exemptions for essential medicines.

Gene therapy for neurological diseases involves using genetic material to treat or prevent disorders affecting the brain, spinal cord, and nervous system. This approach delivers corrective genes into a patient’s cells through 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 primary types of gene therapy for neurological diseases include viral gene therapy and non-viral gene therapy. Viral gene therapy utilizes engineered viruses as delivery vectors to transport therapeutic genes into a patient’s cells to treat or prevent diseases. These therapies target a range of neurological disorders, 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, intracerebral, and others, and are applied across different settings, including hospitals and clinics.

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 the gene therapy on neurological diseases industry global market size, regional shares, competitors with the 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 scenarios of the industry.

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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