Download Free Sample Report

The "Global Gene Therapy in CNS Disorder Market" was valued at US$ 250.00 million in 2024 and is predicted to reach US$ 621.81 million by 2030, exhibiting a Compound Annual Growth Rate (CAGR) of 16.4% during the forecast period (2024-2030).

Gene therapy for central nervous system (CNS) disorders involves the introduction, modification, or replacement of genes within the brain and spinal cord to treat or prevent neurological diseases. CNS disorders such as Parkinson’s disease, Alzheimer’s disease, Huntington’s disease, and various genetic or neurodegenerative conditions have complex pathologies, and gene therapy offers a promising approach by directly targeting the underlying genetic causes of these disorders.

Key components of gene therapy in CNS disorders:

1. Mechanism:

   • Gene therapy typically works by delivering a therapeutic gene to the cells of the CNS using a vector, usually a virus (such as an adeno-associated virus, or AAV). This can either replace a faulty gene, introduce a new gene to help the cells function better, or silence a gene that is causing harmful effects.
   • Delivery methods include:
     • Direct injection into the brain or spinal cord (intracerebral or intrathecal administration).
     • Intravenous (IV) administration where the therapeutic gene is carried through the bloodstream to the CNS.

2. Targets and Strategies:

   • Gene replacement: Used for genetic disorders like spinal muscular atrophy (SMA) where a defective gene is replaced with a functional copy.
   • Gene silencing: Techniques like RNA interference (RNAi) or CRISPR can silence harmful genes, as in Huntington’s disease where abnormal proteins are reduced.
   • Neuroprotection: Introducing genes that code for protective proteins to slow or stop the degeneration of neurons, which is useful in diseases like Parkinson’s.
   • Anti-inflammatory strategies: Gene therapy may also be used to reduce inflammation that contributes to CNS damage, such as in multiple sclerosis (MS).

3. Advantages:

   • Targeting the root cause: Unlike traditional therapies that address symptoms, gene therapy directly modifies the underlying genetic or molecular pathways causing the disease.
   • Long-term effects: Gene therapy can potentially offer long-lasting or permanent treatment effects after a single administration, reducing the need for continuous medication.

4. Challenges:

   • Delivery difficulties: The blood-brain barrier (BBB) makes it difficult for therapeutic agents to reach the CNS, requiring invasive methods or specialized delivery systems.
   • Safety concerns: As with any genetic modification, there are risks related to immune reactions, off-target effects, and long-term consequences.
   • Complexity of CNS diseases: Many CNS disorders are caused by multiple genes or environmental factors, making them more challenging to treat with a single gene therapy.

Applications in CNS disorders:

• Spinal Muscular Atrophy (SMA): Gene therapy, like Zolgensma, replaces the faulty SMN1 gene, allowing for improved motor function and survival in affected infants.
• Parkinson’s Disease: Gene therapy aims to increase the production of dopamine or neuroprotective factors to slow the disease progression.
• Huntington’s Disease: Gene silencing therapies are being developed to reduce the production of harmful mutant proteins.
• Alzheimer’s Disease: Gene therapy approaches are being explored to reduce amyloid-beta plaques or improve neurogenesis.

Gene therapy holds great potential in transforming the treatment landscape for CNS disorders, providing hope for addressing currently untreatable or poorly managed conditions.

The global key manufacturers of Gene Therapy in CNS Disorder include Voyager Therapeutics, Spark Therapeutics, Novartis AG, Bluebird Bio Inc. and Biogen, etc. in 2023, the global top five players have a share approximately % in terms of revenue.
Gene therapyentails the specific transfer of genetic information into affected cells and tissues in order to restore function in diseased areas.

This report aims to provide a comprehensive presentation of the global market for Gene Therapy in CNS Disorder, with both quantitative and qualitative analysis, to help readers develop business/growth strategies, assess the market competitive situation, analyze their position in the current marketplace, and make informed business decisions regarding Gene Therapy in CNS Disorder. This report contains market size and forecasts of Gene Therapy in CNS Disorder in global, including the following market information:

• Global Gene Therapy in CNS Disorder Market Revenue, 2019-2024, 2025-2030, ($ millions)
• Global top five companies in 2023 (%)

We has surveyed the Gene Therapy in CNS Disorder companies, and industry experts on this industry, involving the revenue, demand, product type, recent developments and plans, industry trends, drivers, challenges, obstacles, and potential risks.

Total Market by Segment:
Global Gene Therapy in CNS Disorder Market, by Type, 2019-2024, 2025-2030 ($ millions)
Global Gene Therapy in CNS Disorder Market Segment Percentages, by Type, 2023 (%)

• Viral Gene Therapy
• Non-viral Gene Therapy

Global Gene Therapy in CNS Disorder Market, by Application, 2019-2024, 2025-2030 ($ millions)
Global Gene Therapy in CNS Disorder Market Segment Percentages, by Application, 2023 (%)

• Alzheimer's Disease
• Huntington's Disease
• Parkinson's Disease
• Batten Disease

Global Gene Therapy in CNS Disorder Market, By Region and Country, 2019-2024, 2025-2030 ($ Millions)
Global Gene Therapy in CNS Disorder Market Segment Percentages, By Region and Country, 2023 (%)

• North America (United States, Canada, Mexico)
• Europe (Germany, France, United Kingdom, Italy, Spain, Rest of Europe)
• Asia-Pacific (China, India, Japan, South Korea, Australia, Rest of APAC)
• The Middle East and Africa (Middle East, Africa)
• South and Central America (Brazil, Argentina, Rest of SCA)

Competitor Analysis
The report also provides analysis of leading market participants including:

• Key companies Gene Therapy in CNS Disorder revenues in global market, 2019-2024 (estimated), ($ millions)
• Key companies Gene Therapy in CNS Disorder revenues share in global market, 2023 (%)

Further, the report presents profiles of competitors in the market, key players include:

• Voyager Therapeutics
• Spark Therapeutics
• Novartis AG
• Bluebird Bio Inc.
• Biogen

Outline of Major Chapters:
Chapter 1: Introduces the definition of Gene Therapy in CNS Disorder, market overview.
Chapter 2: Global Gene Therapy in CNS Disorder market size in revenue.
Chapter 3: Detailed analysis of Gene Therapy in CNS Disorder company competitive landscape, revenue and market share, latest development plan, merger, and acquisition information, etc.
Chapter 4: Provides the analysis of various market segments by type, covering the market size and development potential of each market segment, to help readers find the blue ocean market in different market segments.
Chapter 5: Provides the analysis of various market segments by application, covering the market size and development potential of each market segment, to help readers find the blue ocean market in different downstream markets.
Chapter 6: Sales of Gene Therapy in CNS Disorder in regional level and country level. It provides a quantitative analysis of the market size and development potential of each region and its main countries and introduces the market development, future development prospects, market space of each country in the world.
Chapter 7: Provides profiles of key players, introducing the basic situation of the main companies in the market in detail, including product sales, revenue, price, gross margin, product introduction, recent development, etc.
Chapter 8: The main points and conclusions of the report.