Gene Therapy in CNS Disorder Market Expected to Achieve a Strong 14.2% CAGR, to Reach USD 25.0 Billion by 2035

WiseGuy Reports, Sept. 2025 (Press Release) – The Gene Therapy in CNS Disorder Market Global Outlook:

Gene Therapy in CNS Disorder Market Size, Share & Industry Analysis By Application (Neurodegenerative Diseases, Demyelinating Diseases, Psychiatric Disorders, Traumatic Brain Injury, Stroke), By Type of Therapy (Adeno-Associated Virus, Lentiviral Vector, Zinc Finger Nucleases, CRISPR-Cas9, RNA Interference), By Route of Administration (Intravenous, Intrathecal, Intracranial, Intraparenchymal, Intra-arterial), By Target Patient Population (Pediatric, Adult, Geriatric) and Regional Forecasts, 2025-2035

Key Profits for Gene Therapy in CNS Disorder Market Industry Top Members and Stakeholders:

1. The Gene Therapy in CNS Disorder Market report includes a plethora of information such as market dynamics scenario and opportunities during the forecast period.

2. Which regulatory trends at corporate-level, business-level, and functional-level strategies.

3. Which are the End-User technologies being used to capture new revenue streams in the near future.

4. The competitive landscape comprises share of key players, new developments, and strategies in the last three years.

5. One can increase a thorough grasp of Gene Therapy in CNS Disorder Market dynamics by looking at prices as well as the actions of producers and users.

6 Comprehensive companies offering products, relevant financial information, recent developments, various Industry analysis methods, and strategies by these top players.

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Gene Therapy in CNS Disorder Market: Growth, Trends, and Strategic Outlook

The Gene Therapy in CNS Disorder Market is emerging as one of the fastest-growing segments in the global life sciences industry. With neurological diseases accounting for a high proportion of global disease burden, the demand for innovative therapies is intensifying. Gene therapy, offering the ability to modify or correct defective genes, is becoming a game-changing approach for central nervous system (CNS) disorders. According to recent industry insights, the market was valued at USD 5.8 billion in 2024 and is projected to reach USD 25.0 billion by 2035, growing at a CAGR of 14.2% between 2025 and 2035.

Market Dynamics Driving Growth

The Gene Therapy in CNS Disorder Market is propelled by multiple converging factors. The increasing prevalence of CNS disorders, including Parkinson’s disease, Alzheimer’s disease, Huntington’s disease, and rare genetic disorders, is creating a high unmet medical need. At the same time, advancements in gene editing technologies such as CRISPR and AAV-based delivery vectors are enhancing the precision, safety, and efficiency of gene therapies.

Rising investments from both public and private stakeholders are further fueling R&D activity. Pharmaceutical leaders like Novartis, Biogen, and Roche are joined by biotech innovators including CRISPR Therapeutics, Editas Medicine, and bluebird bio in expanding their CNS-focused pipelines. Supportive regulatory frameworks across regions—particularly the FDA in the U.S. and EMA in Europe—are accelerating the approval pathways for breakthrough therapies.

Market Size and Regional Outlook

The Gene Therapy in CNS Disorder Market was valued at USD 6.7 billion in 2025 and is forecast to expand significantly, reaching USD 25.0 billion by 2035. North America remains the dominant region, driven by strong clinical trial infrastructure, robust funding, and high adoption of advanced therapies in the U.S. and Canada. Europe follows closely with Germany, the UK, and France leading investments in gene therapy platforms.

Asia-Pacific is set to witness rapid growth, with China, Japan, and India focusing on expanding biopharma capabilities and encouraging global collaborations. Emerging markets in South America and MEA, particularly Brazil, South Africa, and GCC countries, are gradually increasing adoption, supported by regulatory harmonization and healthcare modernization.

Segmentation Analysis

The Gene Therapy in CNS Disorder Market can be analyzed across applications, therapy types, routes of administration, and target patient populations. By therapy type, viral vector-based approaches dominate, though non-viral alternatives are gaining traction. Intrathecal and intravenous routes remain the most commonly adopted due to the complexity of crossing the blood-brain barrier.

Patient demographics show growing adoption across both pediatric and adult populations, as many CNS disorders have early onset while others are age-related. Applications extend from rare orphan disorders, such as spinal muscular atrophy, to more prevalent diseases like Alzheimer’s and multiple sclerosis, signaling broader commercial potential.

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Key Companies and Competitive Landscape

Competition in the Gene Therapy in CNS Disorder Market is intensifying. Global pharmaceutical giants such as Novartis, Roche, Biogen, Amgen, and GSK are leveraging their resources and regulatory expertise to scale commercialization. Innovative biotechs like Axovant Gene Therapies, Orchard Therapeutics, and Sarepta Therapeutics are focusing on specialized indications and cutting-edge technologies.

CRISPR-based innovators, including CRISPR Therapeutics and Editas Medicine, are pushing the boundaries of precision medicine, while companies like Regeneron Pharmaceuticals and Astellas Pharma are diversifying their CNS gene therapy pipelines. Strategic partnerships, licensing deals, and mergers are becoming common to strengthen market presence.

Growth Opportunities and Future Outlook

The Gene Therapy in CNS Disorder Market holds significant opportunities over the next decade. Key drivers include the rising awareness of personalized medicine, increasing patient acceptance of advanced therapies, and growing institutional investments in gene therapy infrastructure. The potential to address high unmet needs in CNS disorders positions this market for transformative impact.

However, challenges such as high treatment costs, complex manufacturing processes, and long-term safety considerations must be addressed to ensure scalability and patient accessibility. Companies that can optimize manufacturing, ensure regulatory compliance, and demonstrate long-term efficacy are expected to lead.

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Avail This Gene Therapy in CNS Disorder Market Language Pages Here:

中枢神経疾患における遺伝子治療の市場規模 | Marktanteil der Gentherapie bei ZNS-Erkrankungen | Analyse du marché de la thérapie génique pour les troubles du système nerveux central | 중추신경계 질환 유전자 치료 시장 분석 | 中枢神经系统疾病基因治疗市场概况 | Tendencias del mercado de la terapia génica en trastornos del sistema nervioso central

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