(Albany, US), November 11, 2026 – DelveInsight’s ‘Cell and Gene Therapies Targeting CNS Disorders Market Insights, Epidemiology and Market Forecast – 2036’ report delivers an in-depth understanding of the cell and gene therapies targeting CNS disorders, historical and forecasted epidemiology, as well as the gene and cell therapies targeting CNS disorders market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.
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Key Takeaways from the Gene And Cell Therapies Targeting CNS Disorders Market Report
In March 2026, Denali Therapeutics announced that the US FDA had granted accelerated approval for AVLAYAH (tividenofusp alfa-eknm), the first FDA-approved biologic specifically designed to cross the blood-brain barrier and reach the whole body, including the brain, for neurologic manifestations of Hunter syndrome.
In March 2026, uniQure announced it had received final meeting minutes from the US FDA regarding a Type A meeting held on January 30, 2026, to discuss AMT-130 for Huntington’s disease. The company plans to request a Type B meeting in the second quarter of 2026 to discuss potential Phase III study design approaches.
In December 2025, AskBio announced that Japan’s MHLW had granted the Pioneering Regenerative Medical Product designation (SAKIGAKE) for AB-1005 for the treatment of Parkinson’s disease.
In December 2025, Bayer and BlueRock Therapeutics announced that bemdaneprocel had received SAKIGAKE designation from Japan’s MHLW.
CNS therapies were historically limited to small molecules because large molecules penetrate the brain poorly. Early monoclonal antibody attempts in neurological diseases showed limited success, underscoring persistent delivery challenges.
Efficient delivery to specific brain regions remained a key obstacle when gene therapy was first explored for CNS diseases. These CNS drug delivery challenges and early safety and vector setbacks shaped gene therapy timelines, and they led to improved AAV-based delivery systems with better efficacy and tolerability.
Gene and cell therapies for CNS disorders are advancing rapidly. The drivers are the rising prevalence of Alzheimer’s disease, Parkinson’s disease, Spinal Muscular Atrophy (SMA), Huntington’s disease, Amyotrophic Lateral Sclerosis (ALS), multiple sclerosis, Cerebral Adrenoleukodystrophy (CALD), and others.
Approved cell and gene therapies for CNS disorders include onasemnogene abeparvovec (ZOLGENSMA), intrathecal onasemnogene abeparvovec (ITVISMA), elivaldogene autotemcel (SKYSONA), atidarsagene autotemcel (LENMELDY), eladocagene exuparvovec (KEBILIDI), and tividonofusp alfa-eknm (AVLAYAH).
According to DelveInsight’s analysis, there were 1,290,000 diagnosed prevalent cases of Parkinson’s disease in the US in 2025, and the cases are expected to increase at a CAGR of ~3%.
In 2025, the total number of diagnosed prevalent cases of Alzheimer’s disease in the 7MM was 17,000,000. The US accounted for around 45% of these cases.
The leading Gene And Cell Therapies Targeting CNS Disorders Companies include Denali Therapeutics, Kyowa Kirin, PTC Therapeutics, Genetix Biotherapeutics, BlueRock Therapeutics, Bayer, AskBio, Lexeo Therapeutics, uniQure, and others.
Promising Gene And Cell Therapies Targeting CNS Disorders include Tividenofusp alfa-eknm (AVLAYAH), Atidarsagene autotemcel (LENMELDY/LIBMELDY), Eladocagene exuparvovec-tneq (KEBILIDI/UPSTAZA), Elivaldogene autotemcel (SKYSONA), Bemdaneprocel (BRT-DA01), AB-1005, LX1001, AMT-130, and others.
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Gene And Cell Therapies Targeting CNS Disorders Overview
Cell and gene therapies targeting CNS disorders represent a rapidly evolving field focused on the underlying genetic and cellular causes of neurological diseases. These approaches include gene replacement, gene silencing, and cell-based regenerative strategies to restore or modify neuronal function. Advances in viral vectors (e.g., AAV) and improved delivery methods such as intrathecal and intracerebral administration have enhanced access to the brain. Several therapies have already achieved regulatory approval, particularly for rare genetic disorders, demonstrating strong clinical potential. Overall, the field is shifting CNS treatment from symptomatic management to long-term, potentially curative solutions. Gene therapy also carries risks, which depend on the type of gene therapy, type of vector, and administration method, and some can be serious.
Diagnosis of CNS disorders combines clinical evaluation, neurological examination, and advanced diagnostic tools. Key methods include neuroimaging (MRI and CT scans), electrophysiological tests such as EEG, and laboratory tests including cerebrospinal fluid (CSF) analysis and genetic testing. Increasing use of biomarkers and molecular diagnostics is improving early detection and disease characterization.
Gene And Cell Therapies Targeting CNS Disorders Epidemiology Segmentation in the 7MM
Total Diagnosed Prevalent Cases of Selected Indications for Cell and Gene Therapies Targeting CNS Disorders
Total Indication-wise Eligible Cases for Cell and Gene Therapies Targeting CNS Disorders
Total Indication-wise Treated Cases for Cell and Gene Therapies Targeting CNS Disorders
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Gene And Cell Therapies Targeting CNS Disorders Epidemiology Key Findings
In 2025, there were 1,290,000 diagnosed prevalent cases of Parkinson’s disease in the US, with cases expected to increase at a CAGR of ~3%.
In 2025, the number of Amyotrophic Lateral Sclerosis (ALS) cases in the United States was ~30,000.
In the United States, diagnosed prevalent cases of Huntington’s disease were 25,000 in 2025, and they are expected to increase during the forecast period, i.e., 2026–2036.
In 2025, diagnosed prevalent cases of Alzheimer’s disease in the 7MM totaled 17,000,000. The US accounted for the highest number, at around 45%, while the UK reported the lowest.
Estimated diagnosed prevalence in the US in 2025 for other indications includes ~13,000 cases of Spinal Muscular Atrophy and ~86,000 cases of Multiple Sclerosis.
Approved Gene And Cell Therapies Targeting CNS Disorders
The approved therapies chapter provides a detailed review of marketed therapies, covering mechanism of action, regulatory approvals, and advantages and limitations.
Tividenofusp alfa-eknm (AVLAYAH): Denali Therapeutics
AVLAYAH is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme indicated for the treatment of neurologic manifestations of Hunter syndrome (Mucopolysaccharidosis type II, MPS II). It is indicated when initiated in presymptomatic or symptomatic pediatric patients weighing at least 5 kg prior to advanced neurologic impairment. The indication is approved under accelerated approval based on the reduction of CSF heparan sulfate, and continued approval may be contingent upon verification of clinical benefit in a confirmatory trial.
Elivaldogene autotemcel (SKYSONA): Kyowa Kirin
SKYSONA is an autologous hematopoietic stem cell-based gene therapy indicated to slow the progression of neurologic dysfunction in boys 4–17 years of age with early, active cerebral adrenoleukodystrophy (CALD) without an available HLA-matched donor for allogeneic hematopoietic stem cell transplant. Early, active CALD refers to asymptomatic or mildly symptomatic boys (neurologic function score, NFS ≤ 1) who have gadolinium enhancement on brain MRI and Loes scores of 0.5–9.
Other marketed therapies include LENMELDY/LIBMELDY (metachromatic leukodystrophy; restores functional ARSA enzyme production) and KEBILIDI/UPSTAZA (PTC Therapeutics; AADC deficiency; delivers a functional copy of the human DDC gene directly into the putamen).
Gene And Cell Therapies Targeting CNS Disorders Pipeline Analysis
Bemdaneprocel (BRT-DA01): BlueRock Therapeutics (Bayer subsidiary)
Bemdaneprocel (BRT-DA01) is an investigational cell therapy designed to replace the dopamine-producing neurons lost in Parkinson’s disease. The dopaminergic neuron precursors are derived from human embryonic pluripotent stem cells and continue developing into mature dopamine neurons after implantation. The therapy received Fast Track Designation in 2021 and a Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA in 2024. A pivotal Phase III trial (exPDite-2) comparing bemdaneprocel with sham surgery is currently enrolling participants.
AB-1005: Bayer and AskBio
AB-1005 is an investigational gene therapy using AAV vector-mediated delivery of the glial cell line-derived neurotrophic factor (GDNF) gene for participants with moderate-stage Parkinson’s disease, and it is being evaluated in the Phase II REGENERATE-PD trial. It previously received US FDA RMAT and FTD designations, as well as the UK MHRA Innovation Passport.
LX1001 (Lexeo Therapeutics) and AMT-130 (uniQure)
LX1001 is a Phase I/II gene therapy for Alzheimer’s disease that expresses the protective APOE2 protein in the CNS of APOE4 homozygous patients. AMT-130 is a Phase I/II gene therapy for Huntington’s disease that silences the huntingtin gene by targeting and suppressing messenger RNA.
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Gene And Cell Therapies Targeting CNS Disorders Market Outlook
Gene and cell therapies for CNS disorders offer potential one-time, disease-modifying treatments in place of symptomatic management. Improvements in delivery technologies, such as viral vectors and intrathecal administration, are enhancing effectiveness in the brain. Increasing clinical success and regulatory approvals are further accelerating investment and development. The report maps this competitive and clinical landscape, including the Spain gene therapy market within the EU4, and provides cell and gene therapy insights across the 7MM, including patient burden trends, revenue and market share dynamics, and peak patient share and therapy uptake analysis.
Gene And Cell Therapies Targeting CNS Disorders Market Drivers
Rising prevalence of CNS disorders due to aging populations, lifestyle risk factors, improved diagnosis, and increased survival
Rising opportunities in therapies that target the root causes of neurological disorders
Improvements in delivery technologies such as viral vectors and intrathecal administration
Increasing clinical success and regulatory approvals accelerating investment and development
Emerging therapies such as bemdaneprocel (BRT-DA01), AB-1005, LX1001, and AMT-130, supported by advances in gene delivery, neuronal replacement, and gene silencing technologies
Gene And Cell Therapies Targeting CNS Disorders Market Barriers
Limited delivery across the blood–brain barrier, reducing treatment effectiveness
High treatment costs, restricting patient access and reimbursement
Safety concerns, including immune responses and long-term effects
Durability uncertainty of therapeutic benefit in some conditions
Limited clinical data for common and complex CNS disorders
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Scope of the Gene And Cell Therapies Targeting CNS Disorders Market Report
Coverage- 7MM
Study Period- 2022-2036
Forecast Period- 2026-2036
Gene And Cell Therapies Targeting CNS Disorders Companies- Denali Therapeutics, Kyowa Kirin, PTC Therapeutics, Genetix Biotherapeutics, BlueRock Therapeutics, Bayer, AskBio, Lexeo Therapeutics, uniQure, and others.
Gene And Cell Therapies Targeting CNS Disorders Therapies- Tividenofusp alfa-eknm (AVLAYAH), Atidarsagene autotemcel (LENMELDY/LIBMELDY), Eladocagene exuparvovec-tneq (KEBILIDI/UPSTAZA), Elivaldogene autotemcel (SKYSONA), Bemdaneprocel (BRT-DA01), AB-1005, LX1001, AMT-130, and others.
Gene And Cell Therapies Targeting CNS Disorders Market Dynamics: market drivers and market barriers
Gene And Cell Therapies Targeting CNS Disorders Competitive Intelligence Analysis: SWOT Analysis, Conjoint Analysis
Gene And Cell Therapies Targeting CNS Disorders Unmet Needs, KOL Views, Reimbursement
Table of Contents
Key Insights
Report Introduction
Executive Summary
Key Events
Epidemiology and Market Forecast Methodology of Cell and Gene Therapies Targeting CNS Disorders
Cell and Gene Therapies Targeting CNS Disorders Market Overview at a Glance
Disease Background and Overview on Cell and Gene Therapies Targeting CNS Disorders
Treatment Guidelines of Cell and Gene Therapies Targeting CNS Disorders
Epidemiology and Patient Population of Cell and Gene Therapies Targeting CNS Disorders
Patient Journey of Cell and Gene Therapies Targeting CNS Disorders
Marketed Therapies
Emerging Therapies
Cell and Gene Therapies Targeting CNS Disorders: 7MM Analysis
Unmet Needs of Cell and Gene Therapies Targeting CNS Disorders
SWOT Analysis of Cell and Gene Therapies Targeting CNS Disorders
KOL Views of Cell and Gene Therapies Targeting CNS Disorders
Market Access and Reimbursement of Cell and Gene Therapies Targeting CNS Disorders
Appendix
DelveInsight Capabilities
Disclaimer
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