Gene and Cell Therapies Targeting CNS Disorders Market Set for Robust Growth Driven by Innovation and Pipeline Advances by 2036-DeveInsight

DelveInsight's "Gene and Cell Therapies Targeting CNS Disorders Market Insights, Epidemiology, and Market Forecast-2036′′ report offers an in-depth understanding of the Gene and Cell 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, France) the United Kingdom and Japan.

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Some of the key facts of the Gene and Cell Therapies Targeting CNS Disorders Market Report:
• The Gene and Cell Therapies Targeting CNS Disorders market size is anticipated to grow with a significant CAGR during the study period (2022-2036).
• In March 2026, Denali Therapeutics announced the US Food and Drug Administration (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 the treatment of neurologic manifestations of Hunter syndrome.
• In March 2026, uniQure announced that the company received final meeting minutes from the US FDA regarding a Type A meeting held on January 30, 2026, to discuss AMT-130, an investigational gene therapy for Huntington's disease. uniQure intends to continue engaging with the FDA regarding Phase III development considerations and plans to request a Type B meeting in the second quarter of 2026 to further discuss potential study design approaches.
• In December 2025, AskBio announced that Japan's Ministry of Health, Labour and Welfare (MHLW) had granted the Pioneering Regenerative Medical Product designation (SAKIGAKE) for two of AskBio's investigational gene therapy programs: AB-1005, for the treatment of Parkinson's disease.
• In December 2025, Bayer and its wholly owned, independently operated subsidiary BlueRock Therapeutics announced that the investigative cell therapy bemdaneprocel had received Pioneering Regenerative Medical Product designation (SAKIGAKE) from Japan's MHLW.
• In August 2025, the FDA approved new safety labeling changes for SKYSONA due to the increased risk of hematologic malignancy.
• According to DelveInsight analysis, 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 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 total number of diagnosed prevalent cases of Alzheimer's disease in the 7MM was 17,000,000. Among the 7MM countries, the US accounted for the highest number of diagnosed prevalent cases of Alzheimer's disease, contributing around 45%, while the UK reported the lowest number of cases in 2025.
• Key Gene and Cell Therapies Targeting CNS Disorders Companies: In8bio Inc., Brainstorm Cell Therapeutics, Neurocrine Biosciences, Voyager Therapeutics, Atara Biotherapeutics, Neuroplast, UniQure Biopharma, Asklepios Biopharmaceutical, Inc., Brain Neurotherapy Bio, Inc., Myrtelle Inc., Forge Biologics, Inc, Sio Gene Therapies, Lexeo Therapeutics, Partner Therapeutics, Inc., Hoffmann-La Roche, Novartis, Freeline Therapeutics, Passage Bio, Inc., Black Diamond Therapeutics, Inc., and others
• Key Gene and Cell Therapies Targeting CNS Disorders Therapies: DRI cell therapy, NurOwn, VY-AADC02, ATA188, Neuro-Cells, AMT-130, AAV2-GDNF, rAAV-Olig001-ASPA, FBX-101, AAV9-GLB1, LX2006, Sargramostim, Entrectinib, ACTUS-101, Capmatinib, FLT201, PBKR03, BDTX-4933, and others
• The Gene and Cell Therapies Targeting CNS Disorders market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Gene and Cell Therapies Targeting CNS Disorders pipeline products will significantly revolutionize the Gene and Cell Therapies Targeting CNS Disorders market dynamics.

Gene and Cell Therapies Targeting CNS Disorders Overview
Gene and cell therapies targeting CNS (Central Nervous System) disorders represent advanced treatment approaches aimed at addressing diseases that affect the brain and spinal cord. These therapies leverage genetic engineering and cellular techniques to modify or replace malfunctioning cells and genes, aiming to restore normal function or halt disease progression.

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Gene and Cell Therapies Targeting CNS Disorders Epidemiology
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2022 to 2036. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.
Gene and Cell Therapies Targeting CNS Disorders Epidemiology Segmentation:
The Gene and Cell Therapies Targeting CNS Disorders market report proffers epidemiological analysis for the study period 2022-2036 in the 7MM segmented into:
• Total Prevalence of Gene and Cell Therapies Targeting CNS Disorders
• Prevalent Cases of Gene and Cell Therapies Targeting CNS Disorders by severity
• Gender-specific Prevalence of Gene and Cell Therapies Targeting CNS Disorders
• Diagnosed Cases of Episodic and Chronic Gene and Cell Therapies Targeting CNS Disorders

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Gene and Cell Therapies Targeting CNS Disorders Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Gene and Cell Therapies Targeting CNS Disorders market or expected to get launched during the study period. The analysis covers Gene and Cell Therapies Targeting CNS Disorders market uptake by drugs, patient uptake by therapies, and sales of each drug.
Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.
The report also covers the Gene and Cell Therapies Targeting CNS Disorders Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Gene and Cell Therapies Targeting CNS Disorders Therapies and Key Companies
• Tividenofusp alfa-eknm (AVLAYAH): Denali Therapeutics
• Atidarsagene autotemcel (LENMELDY/ LIBMELDY): Kyowa Kirin
• Eladocagene exuparvovec-tneq (KEBILIDI/ UPSTAZA): PTC Therapeutics
• Elivaldogene autotemcel (SKYSONA): Genetix Biotherapeutics
• Bemdaneprocel (BRT-DA01): BlueRock Therapeutics (Bayer subsidiary)
• AB-1005: Bayer and AskBio
• DRI cell therapy: In8bio Inc.
• NurOwn: Brainstorm Cell Therapeutics
• VY-AADC02: Neurocrine Biosciences/Voyager Therapeutics
• ATA188: Atara Biotherapeutics
• Neuro-Cells: Neuroplast
• AMT-130: UniQure Biopharma
• AAV2-GDNF: Asklepios Biopharmaceutical, Inc./Brain Neurotherapy Bio, Inc.
• rAAV-Olig001-ASPA: Myrtelle Inc.
• FBX-101: Forge Biologics, Inc
• AAV9-GLB1: Sio Gene Therapies
• LX2006: Lexeo Therapeutics
• Sargramostim: Partner Therapeutics, Inc.
• Entrectinib: Hoffmann-La Roche
• ACTUS-101: Asklepios Biopharmaceutical, Inc.
• Capmatinib: Novartis
• FLT201: Freeline Therapeutics
• PBKR03: Passage Bio, Inc.
• BDTX-4933: Black Diamond Therapeutics, Inc.

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Gene and Cell Therapies Targeting CNS Disorders Market Drivers
• Rising prevalence of neurological and neurodegenerative disorders such as Parkinson's disease, Alzheimer's disease, Huntington's disease, spinal muscular atrophy (SMA), and amyotrophic lateral sclerosis (ALS) is driving demand for advanced therapeutic solutions.
• Growing advancements in gene editing technologies, viral vectors, and stem cell therapies are accelerating innovation in CNS-targeted treatments.
• Increasing investments from biotechnology companies, pharmaceutical firms, and government organizations are supporting research and clinical development activities.
• Strong progress in precision medicine and personalized therapeutics is improving the development of targeted treatments for rare and genetic CNS disorders.

Gene and Cell Therapies Targeting CNS Disorders Market Barriers
• High development and manufacturing costs associated with gene and cell therapies remain a significant market challenge.
• Complexities related to safe and effective delivery across the blood-brain barrier may limit therapeutic success.
• Limited long-term safety and efficacy data for emerging therapies can create regulatory and commercialization hurdles.
• Risk of immune reactions, off-target effects, and neurological complications may impact patient safety and clinical adoption.

Scope of the Gene and Cell Therapies Targeting CNS Disorders Market Report
• Study Period: 2022-2036
• Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
• Key Gene and Cell Therapies Targeting CNS Disorders Companies: In8bio Inc., Brainstorm Cell Therapeutics, Neurocrine Biosciences, Voyager Therapeutics, Atara Biotherapeutics, Neuroplast, UniQure Biopharma, Asklepios Biopharmaceutical, Inc., Brain Neurotherapy Bio, Inc., Myrtelle Inc., Forge Biologics, Inc, Sio Gene Therapies, Lexeo Therapeutics, Partner Therapeutics, Inc., Hoffmann-La Roche, Novartis, Freeline Therapeutics, Passage Bio, Inc., Black Diamond Therapeutics, Inc., and others
• Key Gene and Cell Therapies Targeting CNS Disorders Therapies: DRI cell therapy, NurOwn, VY-AADC02, ATA188, Neuro-Cells, AMT-130, AAV2-GDNF, rAAV-Olig001-ASPA, FBX-101, AAV9-GLB1, LX2006, Sargramostim, Entrectinib, ACTUS-101, Capmatinib, FLT201, PBKR03, BDTX-4933, and others
• Gene and Cell Therapies Targeting CNS Disorders Therapeutic Assessment: Gene and Cell Therapies Targeting CNS Disorders current marketed and Gene and Cell Therapies Targeting CNS Disorders emerging therapies
• Gene and Cell Therapies Targeting CNS Disorders Market Dynamics: Gene and Cell Therapies Targeting CNS Disorders market drivers and Gene and Cell Therapies Targeting CNS Disorders market barriers
• Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies
• Gene and Cell Therapies Targeting CNS Disorders Unmet Needs, KOL's views, Analyst's views, Gene and Cell Therapies Targeting CNS Disorders Market Access and Reimbursement

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