Gene And Cell Therapies Targeting CNS Disorders Market By Technology (Viral Vector-Based Delivery, Non-Viral Vector-Based Delivery, CRISPR/Cas9, Stem Cell-Based Therapy), By CNS Disorder Targeted (Alzheimer’s Disease, Parkinson’s Disease, Multiple Sclerosis, Amyotrophic Lateral Sclerosis (ALS), Huntington’s Disease), By End-User (Hospitals, Research Institutes, Biopharmaceutical Companies), and By Region; Global Insights & Forecast (2024 – 2030)

As per Intent Market Research, the Gene And Cell Therapies Targeting CNS Disorders Market was valued at USD 7.5 billion in 2023 and will surpass USD 12.0 billion by 2030; growing at a CAGR of 7.0% during 2024 - 2030.

The gene and cell therapies targeting central nervous system (CNS) disorders market is witnessing significant growth due to the increasing prevalence of neurological conditions and the potential of advanced therapies to address unmet medical needs. CNS disorders, such as Alzheimer's disease, Parkinson's disease, and amyotrophic lateral sclerosis (ALS), represent major healthcare challenges globally, with millions of people affected. Traditional treatments often provide only symptomatic relief, and there is a growing need for therapies that can target the underlying causes of these disorders. Gene and cell therapies offer a promising approach, aiming to repair or replace damaged neurons, alter disease-causing genes, or provide new cellular therapies to slow or halt disease progression.

The market for gene and cell therapies targeting CNS disorders is evolving rapidly, with numerous biotech companies, pharmaceutical firms, and academic institutions involved in cutting-edge research. Advances in gene editing technologies like CRISPR/Cas9, viral and non-viral vector-based delivery systems, and stem cell-based therapies have opened new possibilities for treating CNS disorders at the molecular level. These therapies are not only focused on improving patient outcomes but also on improving the quality of life for individuals with neurodegenerative diseases. As research progresses, the market is expected to expand significantly, driven by clinical advancements and regulatory approvals for novel therapies.

Viral Vector-Based Delivery is the Most Widely Used Technology

Viral vector-based delivery systems are the most widely used and established technology in the gene and cell therapies targeting CNS disorders market. This technology involves the use of modified viruses to deliver therapeutic genes into a patient's cells, effectively addressing genetic causes of neurological diseases. Viral vectors, such as adenoviruses, lentiviruses, and adeno-associated viruses (AAV), are engineered to carry genetic material that can correct or replace defective genes, making them an ideal option for treating disorders like Alzheimer's disease, Parkinson's disease, and ALS.

The popularity of viral vector-based delivery systems is attributed to their high efficiency in gene transfer, their ability to target specific tissues, and their proven track record in clinical applications. Despite some challenges related to immune responses and safety concerns, viral vectors remain the go-to method for gene delivery due to their versatility and effectiveness in treating a wide range of CNS disorders. As advancements in viral vector engineering and manufacturing processes continue, this technology is expected to maintain its dominance in the market.

Gene and Cell Therapies Targeting CNS Disorders Market Size

CRISPR/Cas9 is the Fastest-Growing Technology in the Market

CRISPR/Cas9 technology is the fastest-growing segment in the gene and cell therapies targeting CNS disorders market, driven by its precision and ability to edit genes with remarkable accuracy. CRISPR/Cas9 is a gene-editing tool that allows for targeted modification of specific DNA sequences, providing a promising solution for treating genetic disorders at their root cause. This technology has shown great potential in correcting mutations linked to neurological diseases like Huntington's disease, which is caused by a specific genetic mutation.

The rapid growth of CRISPR/Cas9 in the CNS disorder space can be attributed to its ability to offer a more precise and less invasive alternative to traditional gene therapy approaches. In preclinical and early-stage clinical trials, CRISPR/Cas9 has shown promise in correcting genetic mutations associated with various CNS disorders, providing hope for potential treatments that can slow or even reverse disease progression. As CRISPR technology continues to evolve and overcome challenges related to delivery mechanisms and off-target effects, its use in gene and cell therapies for CNS disorders is expected to expand rapidly in the coming years.

Alzheimer's Disease Is the Largest Targeted CNS Disorder

Alzheimer's disease is the largest targeted CNS disorder in the gene and cell therapies market, driven by the growing aging population and the increasing burden of dementia globally. Alzheimer's is a progressive neurodegenerative disease characterized by the accumulation of amyloid plaques and tau tangles in the brain, leading to memory loss, cognitive decline, and eventual loss of independence. With no cure currently available, there is an urgent need for therapies that can modify the disease process rather than just manage its symptoms.

Gene and cell therapies targeting Alzheimer's disease aim to address the underlying biological mechanisms of the disease, such as abnormal protein aggregation, neuronal damage, and neuroinflammation. Therapies such as gene delivery of proteins that break down amyloid plaques or stem cell therapies to replace damaged neurons hold great promise. As the market continues to focus on Alzheimer's, it is expected to remain the dominant target for gene and cell therapies, accounting for the largest share of the market due to the high unmet medical need and significant patient population.

Hospitals Are the Leading End-User Segment

Hospitals are the largest end-user segment in the gene and cell therapies targeting CNS disorders market. Hospitals serve as the primary treatment centers for patients with neurological disorders, offering advanced medical care, diagnostics, and therapeutic interventions. As gene and cell therapies for CNS disorders move toward commercialization, hospitals are becoming key players in the delivery of these cutting-edge treatments, which often require specialized facilities, skilled medical personnel, and rigorous monitoring.

With the increasing availability of gene and cell therapies for CNS disorders, hospitals are expected to play an even more significant role in providing these therapies as part of clinical trials and eventually as standard treatment options. Moreover, the growing focus on personalized medicine and the need for specialized care in treating complex neurological disorders make hospitals the ideal environment for delivering gene and cell therapies. As more therapies gain regulatory approval, hospitals will continue to be at the forefront of implementing these innovations in patient care.

North America Leads the Gene and Cell Therapies Market

North America is the largest region in the gene and cell therapies targeting CNS disorders market, driven by advanced healthcare infrastructure, significant research and development activities, and high healthcare expenditure. The United States, in particular, is home to many of the leading biotechnology and pharmaceutical companies focused on CNS disorders, and it benefits from a well-established regulatory framework that supports the development and approval of gene therapies. Additionally, the high prevalence of neurological diseases such as Alzheimer's and Parkinson's disease in North America has prompted increased investment in gene and cell therapy research.

The region's strong medical infrastructure, coupled with increasing awareness and demand for innovative treatments, positions North America as the leader in the global gene and cell therapies market for CNS disorders. As clinical trials progress and more therapies receive regulatory approval, North America will continue to be a key market, driving growth in both research and patient access to novel treatments.

Gene and Cell Therapies Targeting CNS Disorders Market Size by Region 2030

Competitive Landscape and Leading Companies

The gene and cell therapies targeting CNS disorders market is highly competitive, with several leading players involved in the development and commercialization of advanced therapies. Prominent companies in the market include Biogen, Novartis, CRISPR Therapeutics, Editas Medicine, and Sangamo Therapeutics. These companies are at the forefront of gene and cell therapy research, with numerous clinical trials underway to develop effective treatments for neurological diseases.

The competitive landscape is marked by significant investment in research and development, with companies focusing on overcoming challenges related to delivery mechanisms, safety, and efficacy. Partnerships and collaborations between biotech companies, academic institutions, and pharmaceutical firms are common as stakeholders work together to bring innovative therapies to market. As gene and cell therapies continue to show promise in treating CNS disorders, the competition is expected to intensify, with companies striving to establish themselves as leaders in this rapidly evolving market.

List of Leading Companies:

  • Novartis AG
  • Biogen Inc.
  • Gilead Sciences, Inc.
  • Vertex Pharmaceuticals
  • Editas Medicine, Inc.
  • Sangamo Therapeutics, Inc.
  • Amgen Inc.
  • Sanofi Genzyme
  • Alnylam Pharmaceuticals, Inc.
  • Regeneron Pharmaceuticals, Inc.
  • Astellas Pharma Inc.
  • Pfizer Inc.
  • Horizon Therapeutics
  • Bluebird Bio
  • CRISPR Therapeutics

Recent Developments:

  • In December 2024, Biogen Inc. announced the successful clinical trial results of a gene therapy targeting ALS, showing improved patient outcomes.
  • In November 2024, Vertex Pharmaceuticals expanded its gene therapy pipeline, focusing on Parkinson’s disease with a new collaboration with a leading research institute.
  • In October 2024, Novartis AG received FDA approval for its gene therapy targeting spinal cord injury, marking a breakthrough in regenerative treatments.
  • In September 2024, Sangamo Therapeutics, Inc. partnered with CRISPR Therapeutics to advance their joint clinical studies on gene-editing technologies for Huntington’s disease.
  • In August 2024, Gilead Sciences, Inc. launched a Phase 2 clinical trial for its novel gene therapy targeting Alzheimer’s disease, showing promising early-stage results.

Report Scope:

Report Features

Description

Market Size (2023)

USD 7.5 billion

Forecasted Value (2030)

USD 12.0 billion

CAGR (2024 – 2030)

7.0%

Base Year for Estimation

2023

Historic Year

2022

Forecast Period

2024 – 2030

Report Coverage

Market Forecast, Market Dynamics, Competitive Landscape, Recent Developments

Segments Covered

Gene And Cell Therapies Targeting CNS Disorders Market By Technology (Viral Vector-Based Delivery, Non-Viral Vector-Based Delivery, CRISPR/Cas9, Stem Cell-Based Therapy), By CNS Disorder Targeted (Alzheimer’s Disease, Parkinson’s Disease, Multiple Sclerosis, Amyotrophic Lateral Sclerosis (ALS), Huntington’s Disease), By End-User (Hospitals, Research Institutes, Biopharmaceutical Companies)

Regional Analysis

North America (US, Canada, Mexico), Europe (Germany, France, UK, Italy, Spain, and Rest of Europe), Asia-Pacific (China, Japan, South Korea, Australia, India, and Rest of Asia-Pacific), Latin America (Brazil, Argentina, and Rest of Latin America), Middle East & Africa (Saudi Arabia, UAE, Rest of Middle East & Africa)

Major Companies

Novartis AG, Biogen Inc., Gilead Sciences, Inc., Vertex Pharmaceuticals, Editas Medicine, Inc., Sangamo Therapeutics, Inc., Amgen Inc., Sanofi Genzyme, Alnylam Pharmaceuticals, Inc., Regeneron Pharmaceuticals, Inc., Astellas Pharma Inc., Pfizer Inc., Horizon Therapeutics, Bluebird Bio, CRISPR Therapeutics

Customization Scope

Customization for segments, region/country-level will be provided. Moreover, additional customization can be done based on the requirements

1. Introduction

   1.1. Market Definition

   1.2. Scope of the Study

   1.3. Research Assumptions

   1.4. Study Limitations

2. Research Methodology

   2.1. Research Approach

      2.1.1. Top-Down Method

      2.1.2. Bottom-Up Method

      2.1.3. Factor Impact Analysis

  2.2. Insights & Data Collection Process

      2.2.1. Secondary Research

      2.2.2. Primary Research

   2.3. Data Mining Process

      2.3.1. Data Analysis

      2.3.2. Data Validation and Revalidation

      2.3.3. Data Triangulation

3. Executive Summary

   3.1. Major Markets & Segments

   3.2. Highest Growing Regions and Respective Countries

   3.3. Impact of Growth Drivers & Inhibitors

   3.4. Regulatory Overview by Country

4. Gene And Cell Therapies Targeting CNS Disorders Market, by Technology (Market Size & Forecast: USD Million, 2022 – 2030)

   4.1. Viral Vector-Based Delivery

   4.2. Non-Viral Vector-Based Delivery

   4.3. CRISPR/Cas9

   4.4. Stem Cell-Based Therapy

   4.5. Others

5. Gene And Cell Therapies Targeting CNS Disorders Market, by CNS Disorder Targeted (Market Size & Forecast: USD Million, 2022 – 2030)

   5.1. Alzheimer’s Disease

   5.2. Parkinson’s Disease

   5.3. Multiple Sclerosis

   5.4. Amyotrophic Lateral Sclerosis (ALS)

   5.5. Huntington’s Disease

   5.6. Others

6. Gene And Cell Therapies Targeting CNS Disorders Market, by End-User (Market Size & Forecast: USD Million, 2022 – 2030)

   6.1. Hospitals

   6.2. Research Institutes

   6.3. Biopharmaceutical Companies

   6.4. Others

7. Regional Analysis (Market Size & Forecast: USD Million, 2022 – 2030)

   7.1. Regional Overview

   7.2. North America

      7.2.1. Regional Trends & Growth Drivers

      7.2.2. Barriers & Challenges

      7.2.3. Opportunities

      7.2.4. Factor Impact Analysis

      7.2.5. Technology Trends

      7.2.6. North America Gene And Cell Therapies Targeting CNS Disorders Market, by Technology

      7.2.7. North America Gene And Cell Therapies Targeting CNS Disorders Market, by CNS Disorder Targeted

      7.2.8. North America Gene And Cell Therapies Targeting CNS Disorders Market, by End-User

      7.2.9. By Country

         7.2.9.1. US

               7.2.9.1.1. US Gene And Cell Therapies Targeting CNS Disorders Market, by Technology

               7.2.9.1.2. US Gene And Cell Therapies Targeting CNS Disorders Market, by CNS Disorder Targeted

               7.2.9.1.3. US Gene And Cell Therapies Targeting CNS Disorders Market, by End-User

         7.2.9.2. Canada

         7.2.9.3. Mexico

    *Similar segmentation will be provided for each region and country

   7.3. Europe

   7.4. Asia-Pacific

   7.5. Latin America

   7.6. Middle East & Africa

8. Competitive Landscape

   8.1. Overview of the Key Players

   8.2. Competitive Ecosystem

      8.2.1. Level of Fragmentation

      8.2.2. Market Consolidation

      8.2.3. Product Innovation

   8.3. Company Share Analysis

   8.4. Company Benchmarking Matrix

      8.4.1. Strategic Overview

      8.4.2. Product Innovations

   8.5. Start-up Ecosystem

   8.6. Strategic Competitive Insights/ Customer Imperatives

   8.7. ESG Matrix/ Sustainability Matrix

   8.8. Manufacturing Network

      8.8.1. Locations

      8.8.2. Supply Chain and Logistics

      8.8.3. Product Flexibility/Customization

      8.8.4. Digital Transformation and Connectivity

      8.8.5. Environmental and Regulatory Compliance

   8.9. Technology Readiness Level Matrix

   8.10. Technology Maturity Curve

   8.11. Buying Criteria

9. Company Profiles

   9.1. Novartis AG

      9.1.1. Company Overview

      9.1.2. Company Financials

      9.1.3. Product/Service Portfolio

      9.1.4. Recent Developments

      9.1.5. IMR Analysis

    *Similar information will be provided for other companies 

   9.2. Biogen Inc.

   9.3. Gilead Sciences, Inc.

   9.4. Vertex Pharmaceuticals

   9.5. Editas Medicine, Inc.

   9.6. Sangamo Therapeutics, Inc.

   9.7. Amgen Inc.

   9.8. Sanofi Genzyme

   9.9. Alnylam Pharmaceuticals, Inc.

   9.10. Regeneron Pharmaceuticals, Inc.

   9.11. Astellas Pharma Inc.

   9.12. Pfizer Inc.

   9.13. Horizon Therapeutics

   9.14. Bluebird Bio

   9.15. CRISPR Therapeutics

10. Appendix

 

A comprehensive market research approach was employed to gather and analyze data on the Gene and Cell Therapies Targeting CNS Disorders Market . In the process, the analysis was also done to analyze the parent market and relevant adjacencies to measure the impact of them on the Gene and Cell Therapies Targeting CNS Disorders Market . The research methodology encompassed both secondary and primary research techniques, ensuring the accuracy and credibility of the findings.

Research Approach - Gene and Cell Therapies Targeting CNS Disorders Market

Secondary Research

Secondary research involved a thorough review of pertinent industry reports, journals, articles, and publications. Additionally, annual reports, press releases, and investor presentations of industry players were scrutinized to gain insights into their market positioning and strategies.

Primary Research

Primary research involved conducting in-depth interviews with industry experts, stakeholders, and market participants across the E-Waste Management ecosystem. The primary research objectives included:

  • Validating findings and assumptions derived from secondary research
  • Gathering qualitative and quantitative data on market trends, drivers, and challenges
  • Understanding the demand-side dynamics, encompassing end-users, component manufacturers, facility providers, and service providers
  • Assessing the supply-side landscape, including technological advancements and recent developments

Market Size Assessment

A combination of top-down and bottom-up approaches was utilized to analyze the overall size of the Gene and Cell Therapies Targeting CNS Disorders Market . These methods were also employed to assess the size of various subsegments within the market. The market size assessment methodology encompassed the following steps:

  1. Identification of key industry players and relevant revenues through extensive secondary research
  2. Determination of the industry's supply chain and market size, in terms of value, through primary and secondary research processes
  3. Calculation of percentage shares, splits, and breakdowns using secondary sources and verification through primary sources

Bottom Up and Top Down - Gene and Cell Therapies Targeting CNS Disorders Market

Data Triangulation

To ensure the accuracy and reliability of the market size, data triangulation was implemented. This involved cross-referencing data from various sources, including demand and supply side factors, market trends, and expert opinions. Additionally, top-down and bottom-up approaches were employed to validate the market size assessment.

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