Gene Therapy In CNS Disorder Market Summary

• CNS disorders encompass a wide range of neurological conditions, including neurodegenerative diseases, movement disorders, and demyelinating diseases, that affect the brain and spinal cord. These disorders disrupt neural function, often resulting in progressive disability and, in severe cases, can be life-threatening without ongoing medical care and management.
• Gene therapies for CNS disorders target faulty or missing genes in the brain and spinal cord to restore or protect neural function, aiming to slow or halt disease progression, improve neurological outcomes, and provide durable benefits beyond conventional treatments.
• Our secondary research indicates that in the US, CNS disorders affect a substantial population, with stroke affecting around 1,537 per 100,000 patients, Alzheimer disease with nearly 470 per 100,000 patients, and multiple sclerosis with 97.6 per 100,000 patients. This distribution highlights the diverse burden of CNS disorders and the significant impact of these conditions within the neurological disease landscape.
• The CNS disorder treatment landscape is advancing with gene therapies such as ZOLGENSMA (onasemnogene abeparvovec-xioi) from Novartis, SKYSONA (elivaldogene autotemcel) from bluebird bio, and KEBILIDI/UPSTAZA (eladocagene exuparvovec / eladocagene exuparvovec-tneq) from PTC Therapeutics, which target the underlying genetic causes of neurological dysfunction and aim to deliver durable, disease-modifying benefits beyond conventional treatment options.
• The CNS disorder treatment landscape is further evolving with emerging gene therapies such as AAV2-GDNF/AB 1005 from AskBio, AAV-GAD from MeiraGTx, and LX1001 from Lexeo Therapeutics, which are designed to address the root causes of neurodegeneration and aim to provide durable, disease-modifying benefits beyond conventional treatment approaches.

DelveInsight’s comprehensive report titled “Gene Therapy in CNS Disorder — Market Insights, Epidemiology, and Market Forecast – 2034” offers a detailed analysis of gene therapy in CNS disorder. The report presents historical and projected epidemiological data covering type-specific diagnosed prevalent cases of CNS disorder, total diagnosed prevalent cases of CNS disorder and total treated cases of CNS disorder. In addition to epidemiology, the market report encompasses various aspects related to the patient population. These aspects include the diagnosis process, prescription patterns, physician perspectives, market accessibility, treatment options, and prospective developments in the market across seven major markets: the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan, spanning from 2020 to 2034.

The report analyzes the existing treatment practices and unmet medical requirements Gene Therapy in CNS disorder. It evaluates the market potential and identifies potential business prospects for enhancing therapies or interventions. This valuable information enables stakeholders to make well-informed decisions regarding product development and strategic planning for the market.

Gene Therapy in CNS Disorder Overview

Gene therapies in CNS disorders represent a transformative approach targeting the genetic defects that drive neurodegeneration and functional decline. These therapies are designed to address conditions such as Spinal Muscular Atrophy (SMA), cerebral adrenoleukodystrophy, Aromatic L-amino Acid Decarboxylase (AADC) deficiency, and others by correcting or compensating for faulty genes within the central nervous system. Clinical manifestations range from early motor delays, seizures, or developmental regression to advanced complications including progressive paralysis, cognitive impairment, or premature death. While each disorder arises from distinct genetic mechanisms, common pathways such as impaired neuronal signaling, loss of myelin, and widespread neuronal death drive disease progression. Gene therapies aim to intervene at the molecular level, offering the potential for durable, disease-modifying effects. Early genetic diagnosis and timely intervention are critical, as untreated or progressive cases may result in irreversible neurological damage and a profound impact on quality of life.

Gene Therapy in CNS Disorder Diagnosis and Treatment Overview 

Gene therapies in CNS disorders are evaluated through a comprehensive clinical assessment, supported by detailed patient history, neurological examination, and genetic testing. While no single test can determine eligibility for all patients, molecular diagnostics such as next-generation sequencing are essential for identifying disease-causing mutations and guiding therapeutic targeting. Neuroimaging modalities, including MRI, PET, and diffusion tensor imaging, provide critical insights into brain structure, connectivity, and functional decline, while electrophysiological tests and cognitive assessments help quantify disease severity. Ultimately, precise genetic characterization remains the cornerstone for determining suitability and optimizing outcomes with gene therapy in CNS disorders such as SMA, cerebral adrenoleukodystrophy, and AADC deficiency.

Gene therapy in CNS disorders is tailored to the specific mutation, disease subtype, and clinical severity, recognizing that no single approach can address all patients. These therapies aim to correct or compensate for faulty genes driving neurodegeneration and impaired neural signaling, with the goal of preserving or restoring neurological function and preventing disease progression. Personalized treatment strategies are essential, developed through collaboration between patients and clinicians and guided by genetic profiling, neuroimaging, and functional assessments. Investigational approaches include adeno-associated virus vectors to deliver functional genes, ex vivo lentiviral gene-modified stem cells to restore protein function, and gene editing to repair pathogenic mutations. Applied individually or in combination, these strategies hold promise to preserve neuronal health, limit complications, and deliver durable, disease-modifying benefits far beyond conventional management.

CNS Disorder Epidemiology

The epidemiology section of the Gene Therapy in CNS disorder market report offers information on the patient populations, including historical and projected trends for each of the seven major markets. Examining key opinion leader views from physicians or clinical experts can assist in identifying the reasons behind historical and projected trends. The diagnosed patient pool, their trends, and the underlying assumptions are all included in this section of the report.

This section also presents the total diagnosed prevalence rate of CNS disorder, supported by relevant tables and graphs to provide a clear and concise understanding of the data. Additionally, the report discloses the assumptions made during the analysis, ensuring data interpretation and presentation transparency. This epidemiological data is valuable for understanding the disease burden and its impact on the patient population across various regions.

Key Findings

• In the US, CNS disorders such as SMA and AADC deficiency represent important rare neurological conditions. SMA affects approximately 9,000 to 9,500 individuals, while AADC deficiency is estimated to affect fewer than 50 individuals. This highlights the rarity and considerable clinical impact of these disorders within the CNS disease spectrum.
• In France, Parkinson’s disease affects about 200,000 people, while in Germany its prevalence is estimated at 511.4 per 100,000, underscoring its substantial public health impact across both countries. The higher frequency reflects its progressive neurodegenerative nature and strong association with aging, making it a major contributor to the overall CNS disorder burden in European populations.
• In the UK, CNS disorders present a significant burden, with Parkinson’s disease affecting about 153,000 people, multiple sclerosis impacting nearly 150,000 individuals, and stroke affecting around 1 million people. This high frequency highlights their progressive nature and long-term complications, making them major contributors to the overall CNS disorder burden.
• In Japan, multiple sclerosis affects about 14.2 per 100,000 people, reflecting its relatively lower prevalence compared to Western countries but underscoring its chronic course and neurological complications, which contribute significantly to the overall CNS disorder burden.

Gene Therapy in CNS Disorder Market Outlook

The gene therapy in CNS disorder market is further expected to increase by the major drivers, such as the rising prevalent population, technological advancements, and upcoming therapies in the forecast period (2025–2034).

Gene therapy in CNS disorders is centered on addressing the underlying genetic defects that drive progressive neurodegeneration and neurological dysfunction, with the goal of restoring neuronal function and slowing or halting disease progression. Current investigational strategies include adeno-associated virus–mediated delivery of functional genes to motor neurons and glial cells, ex vivo gene-modified hematopoietic stem cell transplantation to enable cross-correction within the brain, and gene-editing platforms designed to directly repair pathogenic mutations in neuronal populations. These approaches are being explored across a spectrum of rare and severe CNS conditions such as SMA, cerebral adrenoleukodystrophy, metachromatic leukodystrophy, AADC deficiency, and others disorders offering the potential for durable and transformative benefit. A growing pipeline of candidates continues to expand the therapeutic frontier, highlighting the promise of precision genetic medicine to address diseases previously limited to palliative or supportive care. Together, these innovations represent a paradigm shift in CNS therapeutics, moving from symptomatic management toward interventions that directly target the genetic root of neurological disease.

With ongoing research and continued dedication, the future holds promise for even more effective treatments and, ultimately, a potential cure for this challenging condition. According to DelveInsight, the gene therapy in CNS disorder market in the 7MM is expected to change significantly during the forecast period (2025–2034).

Gene Therapy in CNS Disorder Drug Chapters 

Marketed Drugs

ZOLGENSMA (onasemnogene abeparvovec-xioi): Novartis

ZOLGENSMA (onasemnogene abeparvovec-xioi), developed by Novartis Gene Therapies, is a US Food and Drug Administration (FDA) and European Medicines Agency (EMA) approved gene therapy in SMA caused by bi-allelic mutations in the SMN1 gene. Delivered via an adeno-associated virus vector, it provides a functional copy of the SMN1 gene to motor neurons, aiming to restore SMN protein expression, improve neuromuscular function, and offer durable, disease-modifying benefits beyond conventional treatment approaches.

• In December 2024, Novartis reported positive topline results from the Phase III STEER study evaluating investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in treatment-naïve patients with SMA Type 2, aged 2 to under 18 years, who can sit but have never walked independently.
• In August 2019, Novartis reported its continued commitment to ZOLGENSMA (onasemnogene abeparvovec-xioi) for the treatment of children under 2 years of age with SMA, reinforcing its role as a groundbreaking gene therapy in this devastating genetic disorder.
• In May 2019, the FDA approved ZOLGENSMA (onasemnogene abeparvovec-xioi), an innovative gene therapy in pediatric patients with SMA, marking the first gene therapy approved to treat children under two years with the most severe form of SMA, a leading genetic cause of infant mortality.

SKYSONA (elivaldogene autotemcel): Bluebird bio

SKYSONA (elivaldogene autotemcel), developed by bluebird bio, is an FDA-approved Gene Therapy in cerebral adrenoleukodystrophy (CALD). Using an ex vivo lentiviral vector approach to insert functional copies of the ABCD1 gene into a patient’s hematopoietic stem cells, it is designed to restore ALDP protein expression, slow neurologic disease progression, and deliver durable, disease-modifying benefits beyond conventional management options.

• In August 2025, the FDA updated SKYSONA’s label to limit its use only to patients who do not have a suitable stem cell donor or alternative therapy options.
• In September 2022, bluebird bio received FDA accelerated approval for SKYSONA (elivaldogene autotemcel), the first approved therapy shown to slow progression of neurologic dysfunction in boys with early, active CALD, and a devastating and fatal neurodegenerative disease.
• In July 2021, bluebird bio received European Commission approval for SKYSONA (elivaldogene autotemcel, Lenti-D) for patients under 18 years with early CALD without a matched sibling donor, making it the first gene therapy approved in the EU for early CALD.

KEBILIDI / UPSTAZA (eladocagene exuparvovec / eladocagene exuparvovec-tneq): PTC Therapeutics

KEBILIDI / UPSTAZA (eladocagene exuparvovec / eladocagene exuparvovec-tneq), developed by PTC Therapeutics, is an approved Gene Therapy in AADC deficiency. Using an adeno-associated virus vector to deliver the DDC gene directly to the putamen, it is designed to restore dopamine production, improve motor and developmental function, and provide durable, disease-modifying benefits beyond conventional management options.

• In November 2024, the US FDA approved the first gene therapy in the treatment of AADC deficiency, marking a landmark advancement for this rare neurological disorder.
• In July 2022, the European Commission granted marketing authorization for Upstaza (eladocagene exuparvovec), developed by PTC Therapeutics, as the first disease-modifying treatment for patients with AADC deficiency.

Emerging Drugs

AAV2-GDNF/ AB 1005: AskBio

AAV2-GDNF (AB-1005) developed by AskBio, is an investigational gene therapy in Parkinson’s disease. Using an adeno-associated virus serotype 2 vector to deliver the glial cell line-derived neurotrophic factor (GDNF) gene directly to targeted brain regions, it is designed to protect and restore dopaminergic neurons, enhance motor function, and provide durable, disease-modifying benefits beyond conventional management options.

• In September 2025, AskBio reported that the first European participants were randomized in its Phase II trial of AB-1005, an investigational Gene Therapy in Parkinson’s disease, marking a key milestone in advancing clinical evaluation of this CNS-directed therapy.
• In September 2025, AskBio reported the completion of enrollment in its Phase I clinical trial of AB-1005, an investigational gene therapy in multiple system atrophy–parkinsonian type (MSA-P), marking a key milestone in advancing its CNS pipeline.
• In January 2025, AskBio reported that the first participants were randomized in its Phase II trial of AB-1005, an investigational gene therapy in Parkinson’s disease, highlighting progress in the clinical development of this CNS-targeted therapy.

AAV-GAD: MeiraGTx/Hologen AI

AAV-GAD is an investigational gene therapy being studied for the treatment of Parkinson’s disease. Delivered using an adeno-associated virus vector, it is designed to increase GABA production in the subthalamic nucleus, aiming to restore neurotransmitter balance and reduce motor dysfunction. This targeted approach directly addresses disease pathophysiology rather than only managing symptoms, with the potential to improve motor control, enhance quality of life, and provide durable, disease-modifying benefits in patients with this debilitating CNS disorder.

• In May 2025, MeiraGTx was granted FDA Regenerative Medicine Advanced Therapy (RMAT) designation for AAV-GAD in the treatment of Parkinson’s disease, underscoring its potential as a transformative, disease-modifying gene therapy.
• In March 2025, MeiraGTx entered into a strategic collaboration with Hologen AI to accelerate Phase III development of AAV-GAD for Parkinson’s disease and advance industrialization of its proprietary manufacturing process, strengthening its path toward late-stage clinical and commercial readiness.
• In October 2024, MeiraGTx reported positive results from its randomized, sham-controlled clinical bridging study of AAV-GAD for the treatment of Parkinson’s disease, highlighting the therapy’s potential to restore neurotransmitter balance and deliver durable, disease-modifying benefits.

LX1001: Lexeo Therapeutics

LX1001, developed by Lexeo Therapeutics, is an investigational Gene Therapy in the treatment of Alzheimer’s disease associated with APOE4 homozygosity. It uses an adeno-associated virus (AAV) vector to deliver the APOE2 gene directly to the central nervous system, aiming to counteract the pathogenic effects of APOE4 and restore protective lipid metabolism and neuronal function. This targeted, mechanism-driven approach is designed to provide durable, disease-modifying benefits, offering a potential paradigm shift beyond conventional symptomatic therapies.

• In March 2022, LEXEO Therapeutics reported positive initial data from its ongoing Phase I/II clinical trial of AAV-based gene therapy candidate LX1001 in patients with Alzheimer’s disease, underscoring its potential in addressing this neurodegenerative disorder.
• In April 2021, LEXEO Therapeutics reported that the FDA granted Fast Track Designation (FTD) to its AAV-based gene therapy candidate LX1001 for the treatment of Alzheimer’s disease, supporting its expedited development and review.

Note: Detailed marketed/emerging therapies assessment will be provided in the final report.

Gene Therapy in CNS Disorder Market Segmentation

DelveInsight’s “Gene Therapy in CNS Disorder – Market Insights, Epidemiology, and Market Forecast – 2034” report provides a detailed outlook of the current and future gene therapies of CNS disorder market, segmented within countries, by therapies, and by classes. Further, the market of each region is then segmented by each therapy to provide a detailed view of the current and future market share of all therapies.

Gene Therapy in CNS Disorder Market Size by Countries

The Gene Therapy in CNS disorder market size is assessed separately for various countries, including the US, EU4 (Germany, France, Italy, and Spain), the UK, and Japan. In 2024, the United States held a significant share of the overall 7MM (Seven Major Markets) Gene Therapy in CNS disorder market, primarily attributed to the country’s higher prevalence of the condition and the elevated cost of the available treatments. This dominance is projected to persist, especially with the potential early introduction of new products.

Gene Therapy in CNS Disorder Market Size by Therapies

Gene Therapy in CNS Disorder Market Size by Therapies is categorized into current and emerging markets for the study period 2020–2034. 

Note: Detailed market segment assessment will be provided in the final report.

Gene Therapy in CNS Disorder Drugs Uptake

This section focuses on the sales uptake of potential gene therapy in CNS disorder that have recently been launched or are anticipated to be launched in the gene therapy in CNS disorder market between 2025 and 2034. It estimates the market penetration of gene therapy in CNS disorder for a given country, examining their impact within and across classes and segments. It also touches upon the financial and regulatory decisions contributing to the probability of success (PoS) of the drugs in the gene therapy in CNS disorder market.

The emerging  gene therapy in CNS disorder are analyzed based on various attributes such as efficacy and safety in randomized clinical trials, order of entry and other market dynamics, and the unmet need they fulfill in the  gene therapy in CNS disorder market.

Note: Detailed assessment of drug uptake and attribute analysis will be provided in the full report Gene Therapy in CNS disorder.

Gene Therapy in CNS Disorder Market Access and Reimbursement

DelveInsight’s “Gene Therapy in CNS Disorder – Market Insights, Epidemiology, and Market Forecast – 2034” report provides a descriptive overview of the market access and reimbursement scenario of gene therapy in CNS disorder. This section includes a detailed analysis of the country-wise healthcare system for each therapy, enlightening the market access, reimbursement policies, and health technology assessments.

KOL Views

To keep up with current gene therapy in CNS disorder market trends and to fill gaps in secondary findings, we interview KOLs and SMEs’ working in the gene therapy in CNS disorder domain. Their opinion helps understand and validate current and emerging therapies and treatment patterns or gene therapy in CNS disorder market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the gene therapy in CNS disorder unmet needs.

Gene Therapy in CNS Disorder: KOL Insights

DelveInsight’s analysts connected with 20+ KOLs to gather insights; however, interviews were conducted with 10+ KOLs in the 7MM. These KOLs were from organizations, institutes, and hospitals, such as University of Texas MD Anderson Cancer Center, US; University Medical Center Hamburg-Eppendorf, Germany; PSL Research University, France; University of Campania "Luigi Vanvitelli, Italy; Complutense University, Spain; Liverpool John Moores University, UK; and Keio University School of Medicine, Japan; among others.

As per KOLs from the US, “Gene therapy is redefining possibilities in CNS disorders, where conventional treatments often provide only symptomatic relief. By targeting the underlying genetic drivers of neurodegeneration, these therapies have the potential to slow or even halt disease progression. This shift from palliative approaches to disease-modifying interventions marks a transformative step for conditions like SMA and CALD, where prognosis has historically been poor.”

As per KOLs from Germany, “CNS disorders such as Parkinson’s disease and multiple sclerosis are complex, chronic, and often debilitating. Gene therapies introduce a paradigm of precision medicine, offering targeted interventions that address specific molecular pathways. The potential of a one-time treatment to produce lasting benefit is particularly promising, as it could reduce the need for lifelong therapy, hospitalizations, and the economic and caregiver burden associated with these conditions.”

As per KOLs from Japan, “The promise of gene therapy in CNS disorders lies in its ability to restore or protect neuronal function through innovative platforms such as AAV-based delivery systems. By directly addressing mechanisms like motor neuron loss or enzyme deficiencies, these therapies move beyond symptom management. If long-term efficacy is proven, they could establish a new standard of care, shifting clinical practice toward durable, curative solutions for patients facing progressive neurological decline.”

Note: Detailed assessment of KOL Views will be provided in the full report Gene Therapy in CNS disorder.

Competitive Intelligence Analysis

We conduct a Competitive and Market Intelligence analysis of the gene therapy in CNS disorder. Market, utilizing various Competitive Intelligence tools such as SWOT analysis and Market entry strategies. The inclusion of these analyses is contingent upon data availability, ensuring a comprehensive and well-informed assessment of the market landscape and competitive dynamics.

Gene Therapy in CNS Disorder Pipeline Development Activities

The report offers an analysis of therapeutic candidates in Phase II and III stages and examines companies involved in developing targeted therapeutics for gene therapy in CNS disorder. It provides valuable insights into the advancements and progress of potential treatments in clinical development for this condition.

Pipeline Development Activities

The report covers information on collaborations, acquisition and merger, licensing, patent details, and other information for emerging gene therapy in CNS disorder.

Gene Therapy in CNS Disorder Report Insights

• CNS Disorder Patient Population
• Therapeutic Approaches
• Gene Therapy in CNS Disorder Pipeline Analysis
• Gene Therapy in CNS Disorder Market Size and Trends
• Gene Therapy in CNS Disorder Market Opportunities
• Impact of Upcoming Therapies

Gene Therapy in CNS Disorder Report Key Strengths

• 10 Years Forecast
• The 7MM Coverage
• CNS Disorder Epidemiology Segmentation
• Key Cross Competition
• Highly Analyzed Gene Therapy in CNS Disorder Market
• Gene Therapy in CNS Disorder Uptake

Gene Therapy in CNS Disorder Report Assessment

• CNS Disorder Current Treatment Practices
• Unmet Needs
• Gene Therapy in CNS Disorder Profiles
• Gene Therapy in CNS Disorder Market Attractiveness

Key Questions

• How common is CNS disorder?
• What are the key findings of CNS disorder epidemiology across the 7MM, and which country will have the highest number of patients during the study period (2020–2034)?
• What are the currently available treatments for CNS disorder?
• What are the disease risk, burden, and unmet needs of CNS disorder?
• At what CAGR is the gene therapy in CNS disorder market and its epidemiology is expected to grow in the 7MM during the forecast period (2025–2034)?
• How would the unmet needs impact the gene therapy in CNS disorder market dynamics and subsequently influence the analysis of the related trends?
• What would be the forecasted patient pool of CNS disorder in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan?
• Among EU4 and the UK, which country will have the highest number of patients during the forecast period (2025–2034)?
• How many companies are currently developing gene therapies in CNS disorder?

Reasons to buy

• The report will help in developing business strategies by understanding the latest trends and changing treatment dynamics driving the gene therapy in CNS disorder market.
• Insights on patient burden/disease prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• To understand the existing market opportunity in varying geographies and the growth potential over the coming years.
• Distribution of historical and current patient share based on real-world prescription data along with reported sales of current treatment in the US, EU4 (Germany, France, Italy, and Spain), the UK, and Japan.
• Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors.
• Detailed analysis and ranking of class-wise potential current and emerging therapies under the attribute analysis section to provide visibility around leading classes.
• Highlights of Market Access and Reimbursement policies of approved therapies, barriers to accessibility of off-label expensive therapies, and patient assistance programs.
• To understand the perspective of Key Opinion Leaders around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.