Press release
Gene and Cell Therapies Targeting CNS Disorders Market to Reach USD 22.4 Billion by 2034
Gene and Cell Therapies Targeting CNS Disorders
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Key Market Insights
• Market Size (2024): USD 5.9 billion
• Forecast Size (2034): USD 22.4 billion
• CAGR (2025-2034): 16.5%
• Leading Region: North America
• Major Drivers: Breakthroughs in viral vectors, CRISPR technologies, rising burden of CNS diseases, and strong biotech R&D investment.
Market Drivers
Neurological and neurodegenerative diseases such as Parkinson's, Alzheimer's disease, Huntington's disease, spinal muscular atrophy (SMA), and rare pediatric CNS conditions require highly targeted and restorative therapeutic approaches.
Advances in AAV vectors, lentiviral vectors, gene silencing technologies, CRISPR gene editing, neuro-regenerative stem cell therapies, and personalized medicine are enabling transformative treatment options.
Growing regulatory support-including orphan drug designations, breakthrough therapy approvals, and accelerated clinical pathways-further boosts market momentum.
Market Segmentation
By Therapy Type
• Gene Therapy - AAV vectors, lentiviral vectors, CRISPR-based editing, RNA interference therapies.
• Cell Therapy - Neural stem cells, induced pluripotent stem cells (iPSCs), mesenchymal stem cells (MSCs).
• Combination Therapies - Gene-modified cell therapies.
By Application
• Parkinson's Disease
• Alzheimer's Disease
• Spinal Muscular Atrophy
• Huntington's Disease
• Amyotrophic Lateral Sclerosis (ALS)
• Rare Pediatric CNS Disorders
• Traumatic Brain & Spinal Cord Injury
By Vector Type
• Adeno-associated Virus (AAV)
• Lentivirus
• Retrovirus
• Non-viral Vectors (Nanoparticles, Liposomes)
By End User
• Hospitals & Neurology Centers
• Gene Therapy Treatment Centers
• Academic & Research Institutes
• Biopharmaceutical Companies
Explore Full Report here: https://exactitudeconsultancy.com/reports/70785/gene-and-cell-therapies-targeting-cns-disorders-market
Regional Outlook
• North America dominates due to strong regulatory support, high clinical trial participation, and a robust ecosystem for gene and cell therapy manufacturing.
• Europe shows significant growth driven by global biotech investments and expanding rare disease programs.
• Asia-Pacific is the fastest-growing region, supported by increasing research funding, improved genetic diagnostics, and emerging biotech hubs in China, Japan, and South Korea.
• Latin America & Middle East/Africa are developing markets with rising neurological disease burden and improving healthcare capabilities.
Industry Trends & Opportunities
• Increasing development of AAV capsid-engineering for targeted CNS delivery.
• Growth in CRISPR-based therapies for genetic neurodegenerative disorders.
• Expansion of iPSC-derived neural cell therapies for regenerative CNS repair.
• Rising biotech-pharma collaborations for scaling advanced gene therapy manufacturing.
• Strong investment pipeline driven by rare neurological disease programs.
Key Competitors
Leading companies in the CNS gene and cell therapy landscape include:
Biogen, Novartis AG, Bluebird Bio, Roche/Genentech, Spark Therapeutics, Sangamo Therapeutics, Neurogene Inc., Passage Bio, Voyager Therapeutics, Ionis Pharmaceuticals, and several emerging regenerative medicine innovators.
Challenges
• High treatment costs and complex manufacturing processes for gene and cell therapies.
• Regulatory challenges related to long-term safety verification.
• Risks associated with viral vector delivery and immune response.
• Limited availability of specialized treatment centers.
This report is also available in the following languages : Japanese (中枢神経系疾患市場をターゲットとした遺伝子・細胞治療), Korean (중추신경계 질환을 표적으로 하는 유전자 및 세포 치료 시장), Chinese (针对中枢神经系统疾病的基因和细胞疗法市场), French (Marché des thérapies géniques et cellulaires ciblant les troubles du SNC), German (Markt für Gen- und Zelltherapien zur Behandlung von ZNS-Erkrankungen), and Italian (Mercato delle terapie geniche e cellulari mirate ai disturbi del sistema nervoso centrale), etc.
Request for a sample of this research report at (Use Corporate Mail ID for Quick Response) @ https://exactitudeconsultancy.com/request-sample/70785
Our More Reports:
Biologics Safety Testing Market
https://exactitudeconsultancy.com/reports/28396/biologics-safety-testing-market
Bioreactors Market
https://exactitudeconsultancy.com/reports/29878/bioreactors-market
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https://exactitudeconsultancy.com/reports/30235/gene-panel-market
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https://exactitudeconsultancy.com/reports/31143/microbiome-sequencing-services-market
About Us
Exactitude Consultancy is a market research & consulting services firm which helps its client to address their most pressing strategic and business challenges. Our market research helps clients to address critical business challenges and also helps make optimized business decisions with our fact-based research insights, market intelligence, and accurate data.
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