(Albany, US), November 3, 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 gene and cell therapies targeting CNS disorders market, historical and forecasted epidemiology, as well as the gene therapy in CNS disorder 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 granted accelerated approval to AVLAYAH (tividenofusp alfa-eknm), the first FDA-approved biologic designed to cross the blood-brain barrier for treating neurological manifestations of Hunter syndrome.
In March 2026, uniQure received final FDA meeting minutes regarding AMT-130, its investigational gene therapy for Huntington’s disease. The company plans further discussions with the FDA on Phase III development and intends to request a Type B meeting in Q2 2026.
According to DelveInsight’s analysis, the United States captured the largest market size of cell and gene therapies targeting CNS disorders in 2025.
In 2025, the market was driven by key indications such as SMA, CALD, MLD, and AADC deficiency, supported by high unmet need and the availability of disease-modifying therapies.
In 2025, there were 1,290,000 diagnosed prevalent cases of Parkinson’s disease in the US, 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, with the US contributing around 45%, while the UK reported the lowest number of cases.
CNS therapies were historically limited to small molecules due to the poor ability of large molecules to penetrate the brain, and efficient delivery to specific brain regions remained a key obstacle for gene therapy.
Improved AAV-based delivery systems, along with progress in stem cell research, have enhanced efficacy and tolerability and introduced regenerative potential.
The approved cell and gene therapies for CNS disorders include ZOLGENSMA, ITVISMA, SKYSONA, LENMELDY, KEBILIDI, and AVLAYAH.
Manufacturing and quality (CMC) issues have emerged as a major bottleneck, with multiple regulatory delays and FDA Complete Response Letters (CRLs) in 2025 linked to gaps in chemistry, manufacturing, and controls data.
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 addressing 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, this field is shifting CNS treatment from symptomatic management to long-term, potentially curative solutions. Diagnosis of CNS disorders involves clinical evaluation, neurological examination, neuroimaging such as MRI and CT, electrophysiological tests like EEG, and laboratory tests including cerebrospinal fluid (CSF) analysis and genetic testing, with increasing use of biomarkers and molecular diagnostics improving early detection. Gene therapy also comes with risks, which depend on the type of gene therapy, type of vector, and administration method, and some of which can be serious.
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, the number of Amyotrophic Lateral Sclerosis (ALS) cases in the United States was ~30,000.
In the United States, the total number of diagnosed prevalent cases of Huntington’s disease was 25,000 in 2025, which is expected to increase during the forecast period, i.e., 2026–2036.
In 2025, the estimated diagnosed prevalent cases in the US were ~7,600,000 for Alzheimer’s disease, ~86,000 for multiple sclerosis, and ~13,000 for spinal muscular atrophy.
The prevalence of CNS disorders is rising globally due to aging populations, lifestyle risk factors, improved diagnosis, and increased survival, making them a leading cause of disability and disease burden.
Approved Therapies for Gene And Cell Therapies Targeting CNS Disorders
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) when initiated in presymptomatic or symptomatic pediatric patients weighing at least 5 kg prior to advanced neurologic impairment. This indication is approved under accelerated approval based on the reduction of CSF heparan sulfate observed in patients treated with AVLAYAH. Continued approval for this indication 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 human leukocyte antigen (HLA)-matched donor for allogeneic hematopoietic stem cell transplant. Early, active CALD refers to asymptomatic or mildly symptomatic (neurologic function score, NFS ≤ 1) boys who have gadolinium enhancement on brain magnetic resonance imaging (MRI) and Loes scores of 0.5-9.
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 that are lost in Parkinson’s disease. These dopaminergic neuron precursors are derived from human embryonic pluripotent stem cells that continue developing into mature dopamine neurons after implantation. In 2021, bemdaneprocel received Fast Track Designation and in 2024 a Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA. A pivotal Phase III clinical trial (exPDite-2) to assess the efficacy, safety, and overall impact of bemdaneprocel compared to sham surgery control is currently enrolling participants.
AB-1005: Bayer and AskBio
AB-1005, currently being evaluated in the Phase II REGENERATE-PD trial, is an investigational gene therapy with adeno-associated viral (AAV) vector-mediated delivery of the glial cell line-derived neurotrophic factor (GDNF) gene for participants with moderate-stage PD. The therapy aims to restore neuronal function and potentially slow disease progression for people with limited treatment options. AB-1005 previously received US FDA RMAT, FDA FTD, and UK MHRA Innovation Passport designations.
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Gene And Cell Therapies Targeting CNS Disorders Market Outlook
The market for cell and gene therapies targeting CNS disorders is poised for significant growth, driven by the rising prevalence of neurological conditions and the increasing need for disease-modifying treatments. As traditional therapies largely provide symptomatic relief, the shift toward gene replacement, gene editing, and cell-based regeneration is gaining strong momentum. Advancements in vector technologies, improved understanding of disease biology, and expanding clinical pipelines are supporting the transition of these therapies from experimental stages to commercialization, while increased investment, strategic collaborations, and supportive regulatory pathways are further accelerating development. Emerging therapies such as bemdaneprocel (BRT-DA01), AB-1005, LX1001, and AMT-130 highlight the growing focus on AAV-mediated gene delivery and stem cell-derived neuronal replacement in Parkinson’s disease, Alzheimer’s disease, and Huntington’s disease. However, CNS drug delivery challenges gene therapy timelines, with limitations such as delivery across the blood–brain barrier, high treatment costs, manufacturing complexity, and long-term safety considerations continuing to affect adoption. Clinical trial safety concerns, including patient deaths in gene therapy studies, have further raised scrutiny around AAV-based neurotherapies, and major neurodegenerative diseases such as Alzheimer’s and Parkinson’s continue to show high failure rates due to complex disease biology and late-stage intervention challenges. Despite these limitations, the outlook remains positive, as the entry of mid- to late-stage therapies is expected to increase competition and approvals. The US, Germany, France, Italy, the UK, Japan, and the Spain gene therapy market are all covered within the 7MM scope of the report.
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 cell and gene therapies that target the root causes of neurological disorders, offering potential one-time, disease-modifying treatments
Improvements in delivery technologies, such as viral vectors and intrathecal administration, enhancing effectiveness in the brain
Increasing clinical success and regulatory approvals accelerating investment and development
Emerging therapies such as bemdaneprocel (BRT-DA01), AB-1005, LX1001, and AMT-130 offering potential disease-modifying or curative treatments
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
To know more about Gene And Cell Therapies Targeting CNS Disorders Companies working in the treatment market, visit @ CNS Drug Delivery Study Providers Preclinical Gene Therapy
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: Gene And Cell Therapies Targeting CNS Disorders 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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