(Albany, New York) – October 14, 2026 – The market dynamics for gene and cell therapies targeting central nervous system (CNS) disorders are evolving rapidly, driven by the Rising Prevalence of Neurological Diseases, Advancements in Gene Delivery Technologies, Increasing Demand for Disease-Modifying Treatments, and Growing Investment in Regenerative Medicine. Additionally, the development of emerging therapies such as Bemdaneprocel (BRT-DA01), AB-1005, LX1001, AMT-130, and others is expected to influence the market.

DelveInsight, a leading market research firm, announces the release of its latest report, “DelveInsight’s Gene and Cell Therapies Targeting CNS Disorders Market Insights, Epidemiology, and Market Forecast 2036.” This comprehensive report provides an in-depth understanding of gene and cell therapies targeting CNS disorders, including historical and forecasted epidemiology, market trends, and treatment scenarios across the United States, EU4 (Germany, Spain, Italy, and France), the United Kingdom, and Japan.

Key Takeaways from the Gene and Cell Therapies Targeting CNS Disorders Market

• The market for gene and cell therapies targeting CNS disorders is expected to expand during 2026–2036, supported by advances in genetic medicine and regenerative therapies. The exact 2025 and projected 2036 market values, along with the forecast CAGR, are not publicly disclosed on the report page.

• The United States accounted for the largest market share for gene and cell therapies targeting CNS disorders in the 7MM in 2025.

• DelveInsight’s analysis indicates that approximately 1.29 million diagnosed prevalent cases of Parkinson’s disease were reported in the United States in 2025.

• In 2025, the United States had approximately 30,000 cases of amyotrophic lateral sclerosis (ALS) and 25,000 diagnosed prevalent cases of Huntington’s disease.

• The diagnosed prevalent population of Alzheimer’s disease across the 7MM was estimated at approximately 17 million in 2025, with the United States accounting for around 45% of cases.

• Other CNS conditions relevant to the gene and cell therapy landscape include spinal muscular atrophy (SMA), cerebral adrenoleukodystrophy (CALD), metachromatic leukodystrophy (MLD), aromatic L-amino acid decarboxylase (AADC) deficiency, and multiple sclerosis.

• Leading companies in this market include BlueRock Therapeutics, AskBio, uniQure, Lexeo Therapeutics, Voyager Therapeutics, Novartis, and other biotechnology and pharmaceutical developers.

• Promising emerging therapies include Bemdaneprocel (BRT-DA01), AB-1005, LX1001, AMT-130, and other candidates targeting neurodegenerative and genetic CNS disorders.

• Marketed therapies such as ZOLGENSMA, UPSTAZA/KEBILIDI, LIBMELDY/LENMELDY, and SKYSONA demonstrate the growing clinical application of gene therapy in selected neurological and genetic conditions.

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Key Factors Driving the Gene and Cell Therapies Targeting CNS Disorders Market

Rising Prevalence of Central Nervous System Disorders: The growing burden of neurological conditions, including Parkinson’s disease, Alzheimer’s disease, Huntington’s disease, ALS, and inherited neurological disorders, is increasing the need for effective treatment options. Aging populations, improved diagnostic capabilities, and greater recognition of genetic causes are contributing to demand for innovative therapies. Many CNS disorders continue to have substantial unmet medical needs because conventional treatments often manage symptoms without correcting the underlying biological cause.

Advancements in Gene Delivery Technologies: Improvements in adeno-associated viral (AAV) vectors, lentiviral vectors, gene replacement, and gene silencing technologies are supporting the development of targeted therapies for CNS disorders. Researchers are also exploring intrathecal and intracerebral administration to improve delivery to the brain and spinal cord. These developments may enable more precise treatment of genetic abnormalities, although delivery efficiency, immune responses, safety, and long-term durability remain important challenges.

Increasing Demand for Disease-Modifying and Regenerative Treatments: Many conventional neurological therapies focus on managing symptoms or slowing functional decline. Gene and cell therapies offer alternative approaches, including replacing missing or defective genes, reducing the expression of harmful genes, and introducing cells intended to restore or support neuronal function. These approaches are being investigated for selected rare genetic disorders and complex neurodegenerative diseases, with the potential to deliver sustained clinical benefits.

Gene and Cell Therapies Targeting CNS Disorders Competitive Landscape

Several gene and cell therapy candidates targeting CNS disorders are in development, including Bemdaneprocel (BRT-DA01) (BlueRock Therapeutics), AB-1005 (AskBio), LX1001 (Lexeo Therapeutics), and AMT-130 (uniQure).

These candidates use different mechanisms to address neurological disease. Bemdaneprocel is an investigational cell replacement therapy designed to deliver dopamine-producing neuronal cells for Parkinson’s disease. AB-1005 is an investigational AAV-based gene therapy being studied for Parkinson’s disease. LX1001 is an investigational AAV gene therapy targeting APOE-related mechanisms in Alzheimer’s disease, particularly in individuals carrying the APOE4 genotype. AMT-130 is an investigational gene therapy designed to reduce the production of huntingtin protein in Huntington’s disease.

Their potential clinical and commercial impact will depend on trial results, safety, durability of treatment effect, regulatory decisions, manufacturing capacity, and accessibility. In addition, the high cost and complex administration requirements of advanced therapies may influence market uptake.

Discover more about therapies set to influence the gene and cell therapies targeting CNS disorders market @ Gene and Cell Therapies Targeting CNS Disorders Treatment Landscape

Recent Developments in the Gene and Cell Therapies Targeting CNS Disorders Market

• In December 2025, Bayer and its wholly owned, independently operated subsidiary BlueRock Therapeutics announced that the investigational cell therapy bemdaneprocel had received Pioneering Regenerative Medical Product designation (SAKIGAKE) from Japan’s Ministry of Health, Labour and Welfare.

• In August 2025, the US FDA approved safety labeling changes for SKYSONA (elivaldogene autotemcel) to reflect an increased risk of hematologic malignancy. The update highlights the importance of long-term safety monitoring for gene therapy recipients.

• Gene and cell therapy developers continued to advance clinical programs targeting Parkinson’s disease, Alzheimer’s disease, Huntington’s disease, and other neurological conditions, with a focus on improved CNS delivery, treatment durability, and disease-modifying potential.

What Are Gene and Cell Therapies Targeting CNS Disorders?

Gene and cell therapies targeting CNS disorders are advanced treatment approaches designed to address the genetic or cellular mechanisms underlying diseases of the brain and spinal cord. Unlike conventional treatments that often focus on symptom control, these therapies aim to modify disease processes by introducing functional genetic material, reducing the expression of disease-associated genes, or delivering cells that may replace or support damaged neuronal populations.

Gene therapy may involve replacing a missing or defective gene, introducing genetic instructions to produce a therapeutic protein, or reducing the expression of a harmful gene. Cell therapy involves administering living cells with the intention of replacing damaged cells, restoring specific biological functions, or supporting tissue repair.

These approaches are being investigated across a broad range of conditions, including Parkinson’s disease, Alzheimer’s disease, Huntington’s disease, SMA, CALD, MLD, AADC deficiency, and other neurological disorders. Some gene and cell therapies have already received regulatory approval for specific rare genetic diseases, while many treatments for neurodegenerative disorders remain investigational.

Treatment development faces important challenges, including delivery across or around the blood–brain barrier, immune responses, precise targeting of affected cells, long-term safety, manufacturing complexity, and the need for sustained clinical benefit.

Gene and Cell Therapies Targeting CNS Disorders Epidemiology Segmentation

The epidemiology section provides insights into the historical and current patient populations associated with CNS disorders relevant to gene and cell therapies, alongside forecasted trends across the leading markets. The report covers the study period 2022–2036 and includes the following segments:

• Total Diagnosed Prevalent Cases of Selected CNS Disorders

• Indication-specific Diagnosed Prevalent Cases

• Indication-specific Eligible Cases for Gene and Cell Therapies

• Indication-specific Treated Cases

• Country-specific Patient Populations Across the 7MM

The indications evaluated include Parkinson’s disease, Alzheimer’s disease, Huntington’s disease, ALS, SMA, multiple sclerosis, and selected rare genetic CNS disorders. Eligibility for a specific therapy depends on the relevant diagnosis, genetic profile where applicable, disease stage, clinical criteria, and approved treatment indication.

Scope of the Gene and Cell Therapies Targeting CNS Disorders Market Report

• Therapeutic Assessment: Current marketed gene and cell therapies, emerging candidates, and their mechanisms of action

• Market Dynamics: Historical and forecast market size, therapy uptake, market share, and forecast assumptions across the 7MM

• Key Companies: BlueRock Therapeutics, AskBio, uniQure, Lexeo Therapeutics, Novartis, and other developers

• Key Therapies: Bemdaneprocel (BRT-DA01), AB-1005, LX1001, AMT-130, ZOLGENSMA, UPSTAZA/KEBILIDI, LIBMELDY/LENMELDY, SKYSONA, and other relevant therapies

• Competitive Intelligence Analysis: Clinical development, mechanism of action, regulatory status, collaborations, partnerships, and SWOT analysis

• Epidemiology: Diagnosed prevalent cases, eligible patient populations, treated cases, and geographical distribution

• Unmet Needs and Market Access: Key opinion leader perspectives, treatment accessibility, pricing, reimbursement, patient uptake, and future market opportunities

To know more about companies working in the gene and cell therapies targeting CNS disorders market, visit @ Gene and Cell Therapies Targeting CNS Disorders Clinical Trials and Therapeutic Assessment

Table of Contents

1. Gene and Cell Therapies Targeting CNS Disorders Market Report Introduction

2. Executive Summary

3. Key Insights and Market Events

4. Gene and Cell Therapies Targeting CNS Disorders Market Overview

5. Clinical Landscape Analysis by Phase, Molecule Type, and Route of Administration

6. Disease Background and Overview

7. Types, Symptoms, Causes, Pathophysiology, and Diagnosis

8. Gene and Cell Therapies Targeting CNS Disorders Epidemiology and Patient Population

9. Country-Specific Patient Population Across the 7MM

10. Current Treatment Practices and Medical Management

11. Marketed Therapies and Emerging Pipeline Candidates

12. Gene and Cell Therapies Targeting CNS Disorders Market Outlook

13. Country-Wise Market Analysis (2022–2036)

14. Therapy Uptake, Pricing, and Market Access

15. Unmet Needs and Future Opportunities

16. SWOT Analysis

17. Key Opinion Leader Perspectives

18. Market Access and Reimbursement

19. Appendix and Report Methodology

20. Disclaimer

21. About DelveInsight

About DelveInsight

DelveInsight is a leading market research and consulting firm specializing in the life sciences and healthcare industries. Founded in 2014, the company provides comprehensive market intelligence, epidemiology, and insights across pharmaceuticals, biotechnology, medical devices, and emerging therapies. DelveInsight helps healthcare organizations make informed strategic decisions through data-driven research and industry expertise.

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