Gene and Cell Therapies for CNS Disorders Set for Transformative Growth Through 2036, Driven by Pipeline Innovation, Rising Disease Burden, and Expanding Market Opportunities

CNS care is shifting toward one-time, disease-modifying cell and gene therapies, fueled by better delivery and approvals, while safety, cost and manufacturing remain barriers.

Dublin, Sept. 15, 2026 (GLOBE NEWSWIRE) -- "Gene and Cell Therapies Targeting CNS Disorders - Market Insight, Epidemiology, and Market Forecast - 2036" has been added to ResearchAndMarkets.com's offering.

The "Cell and Gene Therapies Targeting CNS Disorders - Market Insights, Epidemiology and Market Forecast - 2036" report provides a comprehensive assessment of the evolving treatment landscape, patient population, competitive environment, and commercial outlook across the United States, EU4 countries-Germany, France, Italy, and Spain-the United Kingdom, and Japan, collectively referred to as the 7MM.

Historically, central nervous system therapies relied heavily on small molecules and symptomatic management because of persistent challenges associated with delivering therapeutic agents to the brain. Advances in molecular biology, viral vector Engineering , stem cell research, gene silencing, and targeted administration have expanded the potential for disease-modifying treatments. Clinical success in rare monogenic disorders has further demonstrated the feasibility of durable and potentially one-time interventions.

The CNS cell and gene therapy market is expected to expand through 2036 as the prevalence of Alzheimer's disease, Parkinson's disease, spinal muscular atrophy, Huntington's disease, amyotrophic lateral sclerosis, multiple sclerosis, cerebral adrenoleukodystrophy, metachromatic leukodystrophy, and other neurological disorders continues to increase. Aging populations, improved diagnosis, longer survival, and substantial unmet medical need are strengthening demand for innovative CNS therapies.

Cell and Gene Therapies Targeting CNS Disorders Market Outlook

The report evaluates historical and forecast market performance from 2022 to 2036, including therapy-level revenue, market share, patient uptake, peak patient share, pricing trends, and growth opportunities. The United States accounted for the largest share of the CNS cell and gene therapy market in 2025, supported by established regulatory pathways, specialist treatment infrastructure, commercial availability of advanced therapies, and significant investment in neurological drug development.

Near-term market demand is being supported by approved treatments for rare neurological and genetic disorders, including spinal muscular atrophy, cerebral adrenoleukodystrophy, metachromatic leukodystrophy, aromatic L-amino acid decarboxylase deficiency, and Hunter syndrome with neurological manifestations. Longer-term growth is expected to be influenced by mid- and late-stage programs targeting Parkinson's disease, Alzheimer's disease, and Huntington's disease.

Approved CNS-focused cell and gene therapies include onasemnogene abeparvovec, elivaldogene autotemcel, atidarsagene autotemcel, eladocagene exuparvovec, and tividenofusp alfa-eknm. The report also assesses relevant regional brands, indications, regulatory status, clinical positioning, safety considerations, and commercial performance.

Market forecasts may change as clinical results, regulatory decisions, epidemiological estimates, pricing assumptions, and product development timelines are updated.

Competitive Landscape and Emerging CNS Therapies

The CNS cell and gene therapy pipeline includes programs based on adeno-associated viral vectors, ex vivo lentiviral platforms, gene silencing, gene replacement, gene editing, and stem cell-derived neuronal replacement. Leading investigational candidates include bemdaneprocel (BRT-DA01), AB-1005, LX1001, and AMT-130.

Bemdaneprocel, developed by BlueRock Therapeutics, is an investigational cell therapy for Parkinson's disease involving the implantation of stem cell-derived dopaminergic neuron precursors. The candidate has received Fast Track and Regenerative Medicine Advanced Therapy designations from the US Food and Drug Administration. Its pivotal Phase III exPDite-2 study is evaluating efficacy, safety, and overall clinical impact against a sham-surgery control.

AB-1005, being developed by AskBio and Bayer , is under evaluation in the Phase II REGENERATE-PD trial for moderate-stage Parkinson's disease. The program uses AAV-mediated delivery of the glial cell line-derived neurotrophic factor gene and has received regulatory designations in the United States, United Kingdom, and Japan.

Other prominent pipeline programs include LX1001 for Alzheimer's disease and AMT-130 for Huntington's disease. These candidates illustrate the industry's growing focus on complex neurodegenerative conditions that have historically experienced high clinical development failure rates.

Key companies active in the CNS cell and gene therapy market include Denali Therapeutics, Kyowa Kirin, PTC Therapeutics, Genetix Biotherapeutics, BlueRock Therapeutics, Bayer , AskBio, Lexeo Therapeutics, and uniQure.

Recent Cell and Gene Therapy Developments

• In March 2026, Denali Therapeutics announced accelerated FDA approval of AVLAYAH for neurological manifestations of Hunter syndrome.
• In March 2026, uniQure reported continued engagement with the FDA regarding Phase III development considerations for AMT-130 in Huntington's disease.
• In December 2025, Japan's Ministry of Health, Labour and Welfare granted SAKIGAKE designation to AskBio's AB-1005 program for Parkinson's disease.
• In December 2025, bemdaneprocel also received SAKIGAKE designation from Japan's Ministry of Health, Labour and Welfare.
• In August 2025, the FDA approved updated safety labeling for SKYSONA concerning the risk of hematologic malignancy.

CNS Disorders Epidemiology in the 7MM

The report provides diagnosed prevalence forecasts and indication-specific patient segmentation across the 7MM. According to the analysis, approximately 1.29 million diagnosed prevalent cases of Parkinson's disease were recorded in the United States in 2025, with the patient population expected to increase during the forecast period. The United States also recorded approximately 30,000 amyotrophic lateral sclerosis cases and 25,000 diagnosed prevalent Huntington's disease cases in 2025.

Across the 7MM, approximately 17 million diagnosed prevalent cases of Alzheimer's disease were estimated in 2025. The United States represented around 45% of these cases, making it the largest contributing market among the countries analyzed.

Market Drivers and Growth Opportunities

Growth in the cell and gene therapies targeting CNS disorders market is being driven by improvements in vector design, CNS-optimized delivery, molecular diagnostics, biomarker development, regenerative medicine, and manufacturing technologies. Intrathecal, intracerebral, intraputaminal, intracisternal, and stereotactic administration strategies are also expanding the range of neurological targets that may be addressed.

Regulatory incentives, strategic partnerships, licensing agreements, and increased investment are accelerating pipeline development. As additional clinical evidence becomes available, physician confidence and patient adoption may improve, particularly for therapies demonstrating durable Benefit in severe disorders with limited treatment alternatives.

Commercial uptake is expected to vary by indication and administration requirements. Approved therapies for rare genetic disorders may achieve steady adoption where diagnosis, referral, reimbursement, and treatment-center capacity are established. Emerging therapies for common neurodegenerative diseases may initially experience slower uptake because of complex patient selection, invasive delivery, long-term monitoring requirements, and the need for robust comparative evidence.

Unmet Needs and Market Barriers

• Limited and inconsistent delivery to targeted CNS regions
• High treatment costs and reimbursement challenges
• Immune responses, procedure-related risks, and long-term safety concerns
• Uncertainty regarding the durability of Benefit in some indications
• Limited clinical evidence in common, multifactorial neurological disorders
• Complex manufacturing, quality assurance, and supply-chain requirements
• Restricted access to qualified treatment centers and specialist infrastructure

Chemistry, manufacturing, and controls requirements remain a significant development risk. Regulatory delays associated with manufacturing data, product consistency, and Quality Systems have reinforced the need for early commercial-scale planning. Safety events in gene therapy studies have also increased regulatory scrutiny of AAV-based neurotherapies and long-term patient monitoring.

Report Coverage and Strategic Insights

The report includes epidemiology-based bottom-up forecasting, current treatment practices, therapy profiles, clinical development analysis, market access considerations, pricing and analogue assessment, patient uptake forecasts, and competitive benchmarking. It reviews mechanisms of Action , clinical trial findings, patents, regulatory milestones, collaborations, strategic partnerships, product advantages, limitations, and recent developments.

Primary research incorporates perspectives from key opinion leaders and subject-matter experts across the 7MM. Interviews address treatment adoption, patient adherence, therapy switching, accessibility, diagnosis, epidemiology, prescription patterns, reimbursement, and pipeline prioritization. Expert outreach included institutions such as the University of North Carolina at Chapel Hill, the Berlin Institute of Health at Charite, and the University of Nottingham.

Qualitative assessment includes SWOT and conjoint analyses covering efficacy, safety, administration route, treatment frequency, market-entry timing, probability of success, and addressable patient population. These analyses support therapy ranking, competitive positioning, market attractiveness assessment, and evidence-based portfolio strategy.

Reasons to Access the Report

• Assess the CNS cell and gene therapy market size, market share, and growth outlook through 2036.
• Understand patient burden and epidemiological trends across the United States, Europe, and Japan.
• Compare approved products and emerging therapies by clinical, regulatory, and commercial attributes.
• Evaluate pricing, reimbursement, treatment access, adoption, and peak patient share.
• Identify unmet needs and high-value opportunities across rare and prevalent CNS disorders.
• Monitor leading companies, clinical programs, collaborations, and regulatory developments.
• Support market entry, product launch, investment, licensing, and pipeline prioritization decisions.

Despite delivery, manufacturing, safety, pricing, and access challenges, the long-term outlook for cell and gene therapies targeting CNS disorders remains positive. Continued innovation and a diversified clinical pipeline are expected to advance neurological care from predominantly symptomatic treatment toward durable, disease-modifying interventions.

Key Topics Covered:

1. Key Insights

2. Report Introduction

3. Executive Summary

4. Key Events
4.1. Upcoming Key Catalysts
4.2. Key Transactions And Collaborations
4.3. Key Conference Highlights
4.4. News Flow

5. Epidemiology and Market Forecast Methodology of Cell and Gene Therapies Targeting CNS Disorders

6. Cell and Gene Therapies Targeting CNS Disorders Market Overview at a Glance
6.1. Clinical Landscape Analysis (By Phase, Molecule Type, and RoA)
6.2. Market Share (%) Distribution of Indication-wise Cell and Gene Therapies Targeting CNS Disorders by Indication in the 7MM, in 2025
6.3. Market Share (%) Distribution of Indication-wise Cell and Gene Therapies Targeting CNS Disorders by Indication in the 7MM, in 2036

7. Disease Background and Overview on Cell and Gene Therapies Targeting CNS Disorders
7.1. Introduction
7.2. Types
7.3. Symptoms
7.4. Causes
7.5. Pathophysiology
7.6. Diagnosis
7.7. Treatment

8. Treatment Guidelines of Cell and Gene Therapies Targeting CNS Disorders

9. Epidemiology and Patient Population of Cell and Gene Therapies Targeting CNS Disorders
9.1. Key Findings
9.2. Assumptions and Rationale
9.3. Total Fina Elf Prevalent Cases of selected indications for Cell and Gene Therapies Targeting CNS Disorders

in the 7MM
9.4. The United States
9.4.1. Total Fina Elf Diagnosed Prevalent Cases of Selected Indications for Cell and Gene Therapies Targeting CNS Disorders in the US
9.4.2. Total Fina Elf Indication-wise Eligible Cases for Cell and Gene Therapies Targeting CNS Disorders in the US
9.4.3. Total Fina Elf Indication-wise Treated Cases of Cell and Gene Therapies Targeting CNS Disorders in the US
9.5. EU4 and the UK
9.5.1. Total Fina Elf Diagnosed Prevalent Cases of Selected Indications for Cell and Gene Therapies Targeting CNS Disorders in EU4 and the UK
9.5.2. Total Fina Elf Indication-wise Eligible Cases for Cell and Gene Therapies Targeting CNS Disorders in EU4 and the UK
9.5.3. Total Fina Elf Indication-wise Treated Cases of Cell and Gene Therapies Targeting CNS Disorders in EU4 and the UK
9.6. Japan
9.6.1. Total Fina Elf Diagnosed Prevalent Cases of Selected Indications for Cell and Gene Therapies Targeting CNS Disorders in Japan
9.6.2. Total Fina Elf Indication-wise Eligible Cases for Cell and Gene Therapies Targeting CNS Disorders in Japan
9.6.3. Total Fina Elf Indication-wise Treated Cases of Cell and Gene Therapies Targeting CNS Disorders in Japan

10. Patient Journey of Cell and Gene Therapies Targeting CNS Disorders

11. Marketed Therapies
11.1. Marketed Competitive Landscape of Cell and Gene Therapies Targeting CNS Disorders
11.2. Tividenofusp alfa-eknm (AVLAYAH): Denali Therapeutics
11.2.1. Drug Description
11.2.2. Regulatory Milestones
11.2.3. Other Developmental Activities
11.2.4. Summary of Pivotal Trials
11.2.5. Clinical Development
11.2.5.1. Clinical Trial Information
11.2.6. Analyst Views
11.3. Elivaldogene autotemcel (SKYSONA): Kyowa Kirin
11.3.1. Drug Description
11.3.2. Regulatory Milestones
11.3.3. Other Developmental Activities
11.3.4. Summary of Pivotal Trials
11.3.5. Clinical Development
11.3.5.1. Clinical Trial Information
11.3.6. Analyst Views
List to be continued...

12. Emerging Therapies
12.1. Emerging Competitive Landscape of Cell and Gene Therapies Targeting CNS Disorders
12.2. Bemdaneprocel (BRT-DA01): BlueRock Therapeutics ( Bayer subsidiary)
12.2.1. Drug Description
12.2.2. Other Developmental Activities
12.2.3. Clinical Development
12.2.3.1. Clinical Trials Information
12.2.4. Safety and Efficacy
12.2.5. Analyst Views
12.3. AB-1005: Bayer and AskBio
12.3.1. Drug Description
12.3.2. Other Developmental Activity
12.3.3. Clinical Development
12.3.3.1. Clinical Trials Information
12.3.4. Safety and Efficacy
12.3.5. Analyst Views
List to be continued...

13. Cell and Gene Therapies Targeting CNS Disorders: 7MM Analysis
13.1. Key Findings
13.2. Market Outlook of Cell and Gene Therapies Targeting CNS Disorders
13.3. Key Market Forecast Assumptions
13.3.1. Cost Assumptions
13.3.2. Pricing Trends
13.3.3. Analogue Assessment
13.3.4. Launch Year and Therapy Uptakes
13.4. Conjoint Analysis of Cell and Gene Therapies Targeting CNS Disorders
13.5. Total Fina Elf Market Size of Cell and Gene Therapies Targeting CNS Disorders by Indications in the 7MM
13.6. Total Fina Elf Market Size of Cell and Gene Therapies Targeting CNS Disorders by Therapies in the 7MM
13.7. The United States
13.7.1. Total Fina Elf Market Size of Cell and Gene Therapies Targeting CNS Disorders by Indications in the United States
13.7.2. Total Fina Elf Market Size of Cell and Gene Therapies Targeting CNS Disorders by Therapies in the United States
13.8. EU4 and the UK
13.8.1. Total Fina Elf Market Size of Cell and Gene Therapies Targeting CNS Disorders by Indications in EU4 and the UK
13.8.2. Total Fina Elf Market Size of Cell and Gene Therapies Targeting CNS Disorders by Therapies in EU4 and the UK
13.9. Japan
13.9.1. Total Fina Elf Market Size of Cell and Gene Therapies Targeting CNS Disorders by Indications in Japan
13.9.2. Total Fina Elf Market Size of Cell and Gene Therapies Targeting CNS Disorders by Therapies in Japan

14. Unmet Needs of Cell and Gene Therapies Targeting CNS Disorders

15. SWOT Analysis of Cell and Gene Therapies Targeting CNS Disorders

16. KOL Views of Cell and Gene Therapies Targeting CNS Disorders

17. Market Access and Reimbursement of Cell and Gene Therapies Targeting CNS Disorders
17.1. The United States
17.2. EU4 and the UK
17.2.1. Germany
17.2.2. France
17.2.3. Italy
17.2.4. Spain
17.2.5. United Kingdom
17.3. Japan
17.4. Summary and comparison of Market Access and Pricing Policy Developments in 2025
17.5. Market Access and Reimbursement of Cell and Gene Therapies Targeting CNS Disorders

18. Appendix
18.1. Bibliography
18.2. Report Methodology

19. Analyst's Capabilities

20. Disclaimer

21. About the Publisher

List of Tables
Table 1: 7MM Gene and Cell Therapies Targeting CNS Disorders Epidemiology (2022-2036)
Table 2: 7MM Gene and Cell Therapies Targeting CNS Disorders Diagnosed and Treatable Cases (2022-2036)
Table 3: Gene and Cell Therapies Targeting CNS Disorders Epidemiology in the United States (2022-2036)
Table 4: Gene and Cell Therapies Targeting CNS Disorders Diagnosed and Treatable Cases in the United States (2022-2036)
Table 5: Gene and Cell Therapies Targeting CNS Disorders Epidemiology in Germany (2022-2036)
Table 6: Gene and Cell Therapies Targeting CNS Disorders Diagnosed and Treatable Cases in Germany (2022-2036)
Table 7: Gene and Cell Therapies Targeting CNS Disorders Epidemiology in France (2022-2036)
Table 8: Gene and Cell Therapies Targeting CNS Disorders Diagnosed and Treatable Cases in France (2022-2036)
Table 9: Gene and Cell Therapies Targeting CNS Disorders Epidemiology in Italy (2022-2036)
Table 10: Gene and Cell Therapies Targeting CNS Disorders Diagnosed and Treatable Cases in Italy (2022-2036)
Table 11: Gene and Cell Therapies Targeting CNS Disorders Epidemiology in Spain (2022-2036)
Table 12: Gene and Cell Therapies Targeting CNS Disorders Diagnosed and Treatable Cases in Spain (2022-2036)
Table 13: Gene and Cell Therapies Targeting CNS Disorders Epidemiology in the UK (2022-2036)
Table 14: Gene and Cell Therapies Targeting CNS Disorders Diagnosed and Treatable Cases in the UK (2022-2036)
Table 15: Gene and Cell Therapies Targeting CNS Disorders Epidemiology in Japan (2022-2036)
Table 16: Gene and Cell Therapies Targeting CNS Disorders Diagnosed and Treatable Cases in Japan (2022-2036)
Table 17: Drug Name , Clinical Trials by Recruitment status
Table 18: Drug Name , Clinical Trials by Zone
Table 19: Total Fina Elf Seven Major Market Size in USD, Million (2022-2036)
Table 20: Region-wise Market Size in USD, Million (2022-2036)
Table 21: 7MM-Market Size by Therapy in USD, Million (2022-2036)
Table 22: United States Market Size in USD, Million (2022-2036)
Table 23: United States Market Size by Therapy in USD, Million (2022-2036)
Table 24: Germany Market Size in USD, Million (2022-2036)
Table 25: Germany Market Size by Therapy in USD, Million (2022-2036)
Table 26: France Market Size in USD, Million (2022-2036)
Table 27: France Market Size by Therapy in USD, Million (2022-2036)
Table 28: Italy Market Size in USD, Million (2022-2036)
Table 29: Italy Market Size by Therapy in USD, Million (2022-2036)
Table 30: Spain Market Size in USD, Million (2022-2036)
Table 31: Spain Market Size by Therapy in USD, Million (2022-2036)
Table 32: United Kingdom Market Size in USD, Million (2022-2036)
Table 33: United Kingdom Market Size by Therapy in USD, Million (2022-2036)
Table 34: Japan Market Size in USD, Million (2022-2036)
Table 35: Japan Market Size by Therapy in USD, Million (2022-2036)
*The list of tables is not exhaustive; the final content may vary

List of Figures
Figure 1: 7MM Gene and Cell Therapies Targeting CNS Disorders Epidemiology (2022-2036)
Figure 2: 7MM Gene and Cell Therapies Targeting CNS Disorders Diagnosed and Treatable Cases (2022-2036)
Figure 3: Gene and Cell Therapies Targeting CNS Disorders Epidemiology in the United States (2022-2036)
Figure 4: Gene and Cell Therapies Targeting CNS Disorders Diagnosed and Treatable Cases in the United States (2022-2036)
Figure 5: Gene and Cell Therapies Targeting CNS Disorders Epidemiology in Germany (2022-2036)
Figure 6: Gene and Cell Therapies Targeting CNS Disorders Diagnosed and Treatable Cases in Germany (2022-2036)
Figure 7: Gene and Cell Therapies Targeting CNS Disorders Epidemiology in France (2022-2036)
Figure 8: Gene and Cell Therapies Targeting CNS Disorders Diagnosed and Treatable Cases in France (2022-2036)
Figure 9: Gene and Cell Therapies Targeting CNS Disorders Epidemiology in Italy (2022-2036)
Figure 10: Gene and Cell Therapies Targeting CNS Disorders Diagnosed and Treatable Cases in Italy (2022-2036)
Figure 11: Gene and Cell Therapies Targeting CNS Disorders Epidemiology in Spain (2022-2036)
Figure 12: Gene and Cell Therapies Targeting CNS Disorders Diagnosed and Treatable Cases in Spain (2022-2036)
Figure 13: Gene and Cell Therapies Targeting CNS Disorders Epidemiology in the UK (2022-2036)
Figure 14: Gene and Cell Therapies Targeting CNS Disorders Diagnosed and Treatable Cases in the UK (2022-2036)
Figure 15: Gene and Cell Therapies Targeting CNS Disorders Epidemiology in Japan (2022-2036)
Figure 16: Gene and Cell Therapies Targeting CNS Disorders Diagnosed and Treatable Cases in Japan (2022-2036)
Figure 17: Drug Name , Clinical Trials by Recruitment status
Figure 18: Drug Name , Clinical Trials by Zone
Figure 19: Total Fina Elf Seven Major Market Size in USD, Million (2022-2036)
Figure 20: Region-wise Market Size in USD, Million (2022-2036)
Figure 21: 7MM-Market Size by Therapy in USD, Million (2022-2036)
Figure 22: United States Market Size in USD, Million (2022-2036)
Figure 23: United States Market Size by Therapy in USD, Million (2022-2036)
Figure 24: Germany Market Size in USD, Million (2022-2036)
Figure 25: Germany Market Size by Therapy in USD, Million (2022-2036)
Figure 26: France Market Size in USD, Million (2022-2036)
Figure 27: France Market Size by Therapy in USD, Million (2022-2036)
Figure 28: Italy Market Size in USD, Million (2022-2036)
Figure 29: Italy Market Size by Therapy in USD, Million (2022-2036)
Figure 30: Spain Market Size in USD, Million (2022-2036)
Figure 31: Spain Market Size by Therapy in USD, Million (2022-2036)
Figure 32: United Kingdom Market Size in USD, Million (2022-2036)
Figure 33: United Kingdom Market Size by Therapy in USD, Million (2022-2036)
Figure 34: Japan Market Size in USD, Million (2022-2036)
Figure 35: Japan Market Size by Therapy in USD, Million (2022-2036)
*The list of figures is not exhaustive; the final content may vary

A selection of companies mentioned in this report includes, but is not limited to:

• Denali Therapeutics
• Kyowa Kirin
• PTC Therapeutics
• Genetix Biotherapeutics
• BlueRock Therapeutics
• Bayer
• AskBio
• Lexeo Therapeutics
• uniQure

For more information about this report visit https://www.researchandmarkets.com/r/4p70gh

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