Dublin, Oct. 06, 2025 (GLOBE NEWSWIRE) — The “Gene Therapy on Neurological Diseases Market Report 2025” has been added to ResearchAndMarkets.com’s offering.
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
The report covers market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the market’s historic and forecast market growth by geography.
Key Attributes:
| Report Attribute | Details |
| No. of Pages | 250 |
| Forecast Period | 2025 – 2029 |
| Estimated Market Value (USD) in 2025 | $3.55 Billion |
| Forecasted Market Value (USD) by 2029 | $5.76 Billion |
| Compound Annual Growth Rate | 12.9% |
| Regions Covered | Global |
Key Topics Covered:
Markets Covered:
- By Type: Viral Gene Therapy; Non-Viral Gene Therapy
- By Target Disease: Alzheimer’s Disease; Parkinsons Disease; Amyotrophic Lateral Sclerosis (ALS); Huntington’s Disease; Multiple Sclerosis
- By Administration Route: Intravenous; Intrathecal; Intranasal; Intracerebral; Other Administration Routes
- By Application: Hospital; Clinic; Other Applications
Subsegments:
- By Viral Gene Therapy Type: Adeno Associated Virus; Lentivirus; Retrovirus; Herpes Simplex Virus; Adenovirus
- By Non-Viral Gene Therapy Type: Liposomes; Nanoparticles; Electroporation; Gene Gun; Naked Plasmid Deoxyribonucleic Acid (DNA)
Gene Therapy on Neurological Diseases Market Regional and Country Analysis
Gene Therapy on Neurological Diseases Market Competitive Landscape and Company Profiles
- Novartis AG Overview, Products and Services, Strategy and Financial Analysis
- Biogen Inc. Overview, Products and Services, Strategy and Financial Analysis
- Neurocrine Biosciences Inc. Overview, Products and Services, Strategy and Financial Analysis
- Sarepta Therapeutics Inc. Overview, Products and Services, Strategy and Financial Analysis
- PTC Therapeutics Inc. Overview, Products and Services, Strategy and Financial Analysis
Gene Therapy on Neurological Diseases Market Other Major and Innovative Companies
- 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
Global Gene Therapy on Neurological Diseases Market Competitive Benchmarking and Dashboard
Key Mergers and Acquisitions in the Gene Therapy on Neurological Diseases Market
Recent Developments in the Gene Therapy on Neurological Diseases Market
Gene Therapy on Neurological Diseases Market High Potential Countries, Segments and Strategies
- Countries Offering Most New Opportunities
- Segments Offering Most New Opportunities
- Growth Strategies
For more information about this report visit https://www.researchandmarkets.com/r/jziw2i
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- Gene Therapy on Neurological Diseases Market
