Clinical Advancements in Gene Therapy for Neurological Diseases Market Outlook 2026–2035
Gene Therapy On Neurological Diseases Market Trends
The Business Research Company’s Gene Therapy for Neurological Diseases Global Market Report 2026 - Market Size, Trends, And Global Forecast 2026-2035
LONDON, GREATER LONDON, UNITED KINGDOM, March 31, 2026 /EINPresswire.com/ -- The gene therapy on neurological diseases market is dominated by a mix of global biotechnology companies, pharmaceutical manufacturers, and specialized gene therapy developers. Companies are focusing on adeno-associated virus (AAV) vector technologies, lentiviral delivery platforms, gene editing approaches such as CRISPR-based therapies, and advanced genomic engineering techniques to strengthen market presence and accelerate the development of therapies targeting neurological disorders such as Parkinson’s disease, Alzheimer’s disease, spinal muscular atrophy, and Huntington’s disease. Emphasis on expanding clinical pipelines for neurodegenerative and rare neurological diseases, advancements in targeted gene delivery to the central nervous system, increasing regulatory approvals for gene-based neurological treatments, and investments in scalable viral vector manufacturing for neurological therapeutics remains central to competitive positioning. Understanding the competitive landscape is essential for stakeholders seeking breakthrough neurological therapies, strategic research collaborations, and commercialization opportunities within the rapidly evolving gene therapy ecosystem for neurological diseases.
Which Market Player Is Leading The Gene Therapy On Neurological Diseases Market?
• According to our research, Novartis AG led global sales in 2024 with a 34% market share. The gene therapy division of the company, which is directly involved in the gene therapy on neurological diseases market, focuses on the development and commercialization of advanced viral vector–based gene therapies designed to treat rare and severe neurological disorders by delivering functional genes to affected cells, supporting disease-modifying treatment approaches, long-term therapeutic expression, and precision genetic medicine for conditions affecting the central nervous system.
Who Are The Major Players In The Gene Therapy On Neurological Diseases Market?
Major companies operating in the gene therapy on neurological diseases market are Novartis AG, Sarepta Therapeutics Inc., Kyowa Kirin, Bluebird Bio Inc., Daiichi Sankyo Company, Limited, PTC Therapeutics Inc., AviadoBio Ltd., Biogen Inc., Oxford Biomedica plc, Regenxbio Inc., Voyager Therapeutics Inc., Neurogene Inc., Intellia Therapeutics Inc., Sangamo Therapeutics Inc., Ionis Pharmaceuticals Inc., Wave Life Sciences Ltd., Capsida Biotherapeutics Inc., Taysha Gene Therapies Inc., Celosia Therapeutics, Editas Medicine Inc., GenSight Biologics, UniQure N.V., Abeona Therapeutics Inc.
How Concentrated Is The Gene Therapy On Neurological Diseases Market?
• The market is highly concentrated, with the top 5 players accounting for 58% of total market revenue in 2024. This level of concentration reflects high technological, regulatory, and clinical development entry barriers, driven by complex viral vector engineering requirements, stringent regulatory approvals, high research and development costs, specialized manufacturing infrastructure, long clinical trial timelines, and the need for proven safety and efficacy in treating complex neurological disorders. Leading players such as Novartis AG, Sarepta Therapeutics Inc., Kyowa Kirin, Bluebird Bio Inc., Daiichi Sankyo Company, Limited hold notable market shares through diversified gene therapy pipelines, strategic biotechnology collaborations, advanced AAV and gene-editing platforms, strong clinical development capabilities, and continuous innovation in neurological disease treatment technologies. As demand for targeted gene therapies, precision genetic medicine, and disease-modifying treatments for rare and inherited neurological disorders grows, strategic partnerships, technological advancements, and expanding clinical pipelines are expected to strengthen the competitive positioning of these leading companies in the market.
• Leading companies include:
o Novartis AG (34%)
o Sarepta Therapeutics Inc. (23%)
o Kyowa Kirin (Orchard Therapeutics plc) (1%)
o Bluebird Bio Inc. (0.3%)
o Daiichi Sankyo Company, Limited (0.1%)
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Who Are The Key Raw Material Suppliers In The Gene Therapy On Neurological Diseases Market?
• Major raw material suppliers in the gene therapy on neurological diseases market include Thermo Fisher Scientific Inc., Merck KGaA, Danaher Corporation, Lonza Group AG, Catalent Inc., Sartorius AG, Fujifilm Holdings Corporation, WuXi AppTec Co. Ltd., Oxford Biomedica plc, Charles River Laboratories International Inc., Samsung Biologics Co. Ltd., AGC Biologics, Takara Bio Inc., Bio-Rad Laboratories Inc., GenScript Biotech Corporation, Aldevron LLC, Polyplus, Cobra Biologics, Miltenyi Biotec GmbH, Creative Biogene, VGXI Inc., elyn Biosciences Inc.
Who Are The Major Wholesalers And Distributors In The Gene Therapy On Neurological Diseases Market?
• Major wholesalers or distributors in the gene therapy on neurological diseases market include AmerisourceBergen Corporation, McKesson Corporation, Cardinal Health Inc., Zuellig Pharma Corporation, Phoenix Pharmahandel GmbH & Co KG, Medipal Holdings Corporation, Sinopharm Group Co. Ltd., Shanghai Pharmaceuticals Holding Co. Ltd., Alliance Healthcare, FFF Enterprises Inc., Morris & Dickson Co. LLC, Anda Inc., H D Smith LLC, Dakota Drug Inc., BioCare Copenhagen A/S, Uniphar Group plc, Clinigen Group plc, EVERSANA, Biocair International Ltd., Marken Ltd.
Who Are The Major End Users Of The Gene Therapy On Neurological Diseases Market?
• Major end users in the gene therapy on neurological diseases market include Novartis AG, Sarepta Therapeutics Inc., Biogen Inc., PTC Therapeutics Inc., Daiichi Sankyo Company Limited, Bluebird Bio Inc., REGENXBIO Inc., Voyager Therapeutics Inc., Neurogene Inc., Sangamo Therapeutics Inc., Intellia Therapeutics Inc., Ionis Pharmaceuticals Inc., Wave Life Sciences Ltd., Ultragenyx Pharmaceutical Inc., Passage Bio Inc., Asklepios BioPharmaceutical Inc., Denali Therapeutics Inc., Abeona Therapeutics Inc., Taysha Gene Therapies Inc., MeiraGTx Holdings plc, Solid Biosciences Inc., Krystal Biotech Inc.
What Are The Major Competitive Trends In The Market?
• CRISPR-enabled gene-editing platforms are transforming the gene therapy for neurological diseases market by enabling precise correction of disease-causing mutations, improving therapeutic targeting within the central nervous system, and accelerating the development of next-generation one-time genetic treatments for rare neurological disorders.
• Example: In February 2026, Neurogene Inc. announced that the U.S. Food and Drug Administration granted breakthrough therapy designation to its investigational gene therapy NGN-401 for the treatment of Rett Syndrome.
• It is designed as a one-time treatment that delivers a functional MECP2 gene to address the underlying genetic cause of the disease, supported by promising interim Phase 1/2 clinical data demonstrating meaningful improvements in multiple functional domains among treated patients, highlighting the growing role of precision genome-editing approaches in advancing neurological gene therapy development.
Which Strategies Are Companies Adopting To Stay Ahead?
• Targeted Gene Therapies Advancing Treatment For Rare Neurological Disorders
• Non-Viral Gene Delivery Systems Enabling Safer And Repeatable Gene Therapies
• Advancements In Viral Vector Technologies Strengthening Neurological Gene Therapy Development
• Strategic Collaborations Accelerating Innovation In Neurological Gene Therapy Research
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