Overview


According to FutureWise analysis, the GM1 gangliosidosis treatment market in 2025 is US$0.13 billion, and is expected to reach US$0.86 billion by 2033 at a CAGR of 26.64%

The GM1 gangliosidosis treatment market is experiencing remarkable growth, driven by significant advancements in gene therapy and enzyme replacement therapy specifically targeting this rare lysosomal storage disorder. Recent breakthroughs in these therapeutic approaches demonstrate the potential to transform patient outcomes, offering hope to individuals affected by this devastating condition. A key factor driving this market's growth is heightened advocacy from patient organizations. These groups are not only raising awareness but also pushing for improved newborn screening programs. Timely and accurate screenings enable earlier diagnosis and intervention, which are crucial to improving the effectiveness of treatments and ultimately improving patients' quality of life. Moreover, regulatory measures such as orphan drug designations and accelerated approval processes are providing valuable incentives for pharmaceutical companies. These measures encourage investment in the development of innovative therapies tailored to this ultra-rare genetic disorder, making it more feasible for companies to explore new treatment options. In addition to these regulatory incentives, collaborative research initiatives are playing an essential role in advancing the field. Partnerships among academic institutions, biotechnology firms, and rare disease foundations are fostering an environment of innovation. These collaborations not only facilitate the sharing of resources and expertise but also promote faster enrollment in clinical trials. As a result, multiple therapeutic candidates are progressing through the development pipeline at an accelerated pace, bringing us closer to potential treatments.

FutureWise Market Research has instantiated a report that provides an intricate analysis of GM1 Gangliosidosis Treatment Market trends that shall affect the overall market growth. Furthermore, it includes detailed information on the graph of profitability, SWOT analysis, market share and regional proliferation of this business. Moreover, the report offers insights on the current stature of prominent market players in the competitive landscape analysis of this market.

According to the research study conducted by FutureWise research analysts, the GM1 Gangliosidosis Treatment Market is anticipated to attain substantial growth by the end of the forecast period. The report explains that this business is predicted to register a noteworthy growth rate over the forecast period. This report provides crucial information pertaining to the total valuation that is presently held by this industry and it also lists the segmentation of the market along with the growth opportunities present across this business vertical.

  • Axovant Gene Therapies Ltd
  • Passage Bio
  • Lysogene
  • Sio Gene Therapies Inc.
  • Orchard Therapeutics plc
  • Regenxbio Inc.
  • Amicus Therapeutics, Inc.
  • BioMarin Pharmaceutical Inc.
  • Sangamo Therapeutics, Inc.
  • Ultragenyx Pharmaceutical Inc.
  • Spark Therapeutics, Inc.
  • Denali Therapeutics Inc.
  • Voyager Therapeutics, Inc.
  • Bluebird Bio, Inc.
  • Homology Medicines, Inc.
  • Prevail Therapeutics Inc.
  • Aspa Therapeutics, Inc.
  • Genethon
  • JCR Pharmaceuticals Co., Ltd.
  • uniQure N.V.

(Note: The list of the major players will be updated with the latest market scenario and trends)

By Product Type

  • LYS-GM101
  • PBGM01
  • AXO-AAV-GM1

By Disease Type

  • Type 1 GM1 Gangliosidosis
  • Type 2 GM1 Gangliosidosis
  • Type 3 GM1 Gangliosidosis

By Region

  • North America
  • Europe
  • Asia-Pacific
  • Latin America
  • Middle East and Africa

  • Tier 1 players-established companies in the market with a major market share
  • Tier 2 players
  • Emerging players which are growing rapidly
  • New Entrants

  • Growth prospects
  • SWOT analysis
  • Key trends
  • Key data-points affecting market growth

  • To provide with an exhaustive analysis on the GM1 Gangliosidosis Treatment Market By Product Type, By Disease Type and By Region
  • To cater comprehensive information on factors impacting market growth (drivers, restraints, opportunities, and industry-specific restraints)
  • To evaluate and forecast micro-markets and the overall market
  • To predict the market size, in key regions—North America, Europe, Asia Pacific, Latin America and Middle East and Africa
  • To record and evaluate competitive landscape mapping-product launches, technological advancements, mergers and expansions

  • We have a flexible delivery model and you can suggest changes in the scope/table of content as per your requirement
  • The customization Mobility Care offered are free of charge with purchase of any license of the report
  • You can directly share your requirements/changes to the current table of content to: sales@futurewiseresearch.com

Table of Contents


  • 1.   Market Introduction
    •   1. Objectives of the Study
        2. Market Definition
        3. Market Scope
         3.1. Years Considered for the Study
         3.2. Market Covered
        4. Currency
        5. Limitations
        6. Stakeholders
  • 2.   Research Methodology
    •   1. Research Data
         1.1. Secondary Data
          1.1.1. Key Data from Secondary Sources
         1.2. Primary Data
          1.2.1. Key Data from Primary Sources
        2. Market Size Estimation
        3. Market Breakdown and Data Triangulation
        4. Assumptions for the Study
  • 3.   Executive Summary
    •   1. Market Outlook
        2. Segment Outlook
        3. Competitive Insights
  • 4.   Global GM1 Gangliosidosis Treatment Market Variables, Trends and Scope
    •          1. Market Lineage Outlook
        2. Penetration and Growth Prospect Mapping
        3. Industry Value Chain Analysis
        4. Cost Analysis Breakdown
        5. Technology Overview
        6. Regulatory Framework
         6.1. Reimbursement Framework
         6.2. Standards and Compliances

  • 5.   Market Overview
    •   1. Market Dynamics
         1.1. Market Driver Analysis
          1.1.1. Increasing focus of GM1 Gangliosidosis Treatment Companies on Brand Protection
          1.1.2. Untapped Opportunities in Emerging Regions
         1.2. Market Restraint Analysis
          1.2.1. High Cost Associated with Implementation of Predictive Analysis
         1.3. Industry Challenges
          1.3.1. Presence of Ambiguous Regulatory Framework
  • 6.   Global GM1 Gangliosidosis Treatment Market Analysis Tools
    •   1. Industry Analysis - Porter’s
         1.1. Supplier Power
         1.2. Buyer Power
         1.3. Substitution Threat
         1.4. Threat from New Entrants
         1.5. Competitive Rivalry
        2. Pestel Analysis
         2.1. Political Landscape
         2.2. Environmental Landscape
         2.3. Social Landscape
         2.4. Technology Landscape
         2.5. Legal Landscape
        3. Major Deals And Strategic Alliances Analysis
         3.1. Joint Ventures
         3.2. Mergers and Acquisitions
         3.3. Licensing and Partnership
         3.4. Technology Collaborations
         3.5. Strategic Divestments
        4. Market Entry Strategies
        5. Case Studies
  • 7.   Global GM1 Gangliosidosis Treatment Market, By Product Type Historical Analysis and Forecast 2025-2033 (USD Million)
    •   1. LYS-GM101
        2. PBGM01
        3. AXO-AAV-GM1

  • 8.   Global GM1 Gangliosidosis Treatment Market, By Disease Type Historical Analysis and Forecast 2025-2033 (USD Million)
    •   1. Type 1 GM1 Gangliosidosis
        2. Type 2 GM1 Gangliosidosis
        3. Type 3 GM1 Gangliosidosis

  • 9.   North America GM1 Gangliosidosis Treatment Market Analysis 2018-2024 and Forecast 2025-2033 (USD Million)
    •   1. Introduction
        2. Historical Market Size (USD Million) Analysis By Country, 2018-2024
         2.1. U.S.A
         2.2. Canada
         2.3. Mexico
        3. Market Size (USD Million) Forecast for North America 2025-2033

  • 10.   Latin America GM1 Gangliosidosis Treatment Market Analysis 2018-2024 and Forecast 2025-2033 (USD Million)
    •   1. Introduction
        2. Historical Market Size (USD Million) Analysis By Country, 2018-2024
         2.1. Brazil
         2.2. Venezuela
         2.3. Argentina
         2.4. Rest of Latin America
        3. Market Size (USD Million) Forecast for Latin America 2025-2033

  • 11.   Europe GM1 Gangliosidosis Treatment Market Analysis 2018-2024 and Forecast 2025-2033 (USD Million)
    •   1. Introduction
        2. Historical Market Size (USD Million) Analysis By Country, 2018-2024
         2.1. Germany
         2.2. U.K
         2.3. France
         2.4. Italy
         2.5. Spain
         2.6. Russia
         2.7. Poland
         2.8. Rest of Europe
        3. Market Size (USD Million) Forecast for Europe 2025-2033

  • 12.   Asia Pacific GM1 Gangliosidosis Treatment Market Analysis 2018-2024 and Forecast 2025-2033 (USD Million)
    •   1. Introduction
        2. Historical Market Size (USD Million) Analysis By Country, 2018-2024
         2.1. Japan
         2.2. China
         2.3. India
         2.4. Australia and New Zealand
         2.5. ASEAN
         2.6. Rest of Asia Pacific
        3. Market Size (USD Million) Forecast for Asia Pacific 2025-2033

  • 13.   Middle East and Africa Market Analysis 2018-2024 and Forecast 2025-2033 (USD Million)
    •   1. Introduction
        2. Historical Market Size (USD Million) Analysis By Country, 2018-2024
         2.1. Saudi Arabia
         2.2. UAE
         2.3. South Africa
         2.4. Egypt
        3. Market Size (USD Million) Forecast for MEA 2025-2033

  • 14.   Market Share Analysis and Competitive Landscape
    •   1. Global Landscape - Key Players, Revenue and Presence
        2. Global Share Analysis - Key Players (Tier 1, Tier 2, Tier 3)
        3. Global Emerging Companies
        4. North America - Market Share Analysis and Key Regional Players
        5. Europe - Market Share Analysis and Key Regional Players
        6. Asia Pacific - Market Share Analysis and Key Regional Players
        7. Global Key Player - Growth Matrix
  • 15.   Company Profiles (Competition Dashboard, Competitors Deep Dive, Products Offered and Financial Layouts)
    •   1. Axovant Gene Therapies Ltd
         1.1. Company Overview
         1.2. Product Portfolio
         1.3. SWOT Analysis
         1.4. Financial Overview
         1.5. Strategic Overview
        2. Passage Bio
         2.1. Company Overview
         2.2. Product Portfolio
         2.3. SWOT Analysis
         2.4. Financial Overview
         2.5. Strategic Overview
        3. Lysogene
         3.1. Company Overview
         3.2. Product Portfolio
         3.3. SWOT Analysis
         3.4. Financial Overview
         3.5. Strategic Overview
        4. Sio Gene Therapies Inc.
         4.1. Company Overview
         4.2. Product Portfolio
         4.3. SWOT Analysis
         4.4. Financial Overview
         4.5. Strategic Overview
        5. Orchard Therapeutics plc
         5.1. Company Overview
         5.2. Product Portfolio
         5.3. SWOT Analysis
         5.4. Financial Overview
         5.5. Strategic Overview
        6. Regenxbio Inc.
         6.1. Company Overview
         6.2. Product Portfolio
         6.3. SWOT Analysis
         6.4. Financial Overview
         6.5. Strategic Overview
        7. Amicus Therapeutics, Inc.
         7.1. Company Overview
         7.2. Product Portfolio
         7.3. SWOT Analysis
         7.4. Financial Overview
         7.5. Strategic Overview
        8. BioMarin Pharmaceutical Inc.
         8.1. Company Overview
         8.2. Product Portfolio
         8.3. SWOT Analysis
         8.4. Financial Overview
         8.5. Strategic Overview
        9. Sangamo Therapeutics, Inc.
         9.1. Company Overview
         9.2. Product Portfolio
         9.3. SWOT Analysis
         9.4. Financial Overview
         9.5. Strategic Overview
        10. Ultragenyx Pharmaceutical Inc.
         10.1. Company Overview
         10.2. Product Portfolio
         10.3. SWOT Analysis
         10.4. Financial Overview
         10.5. Strategic Overview
        11. Spark Therapeutics, Inc.
         11.1. Company Overview
         11.2. Product Portfolio
         11.3. SWOT Analysis
         11.4. Financial Overview
         11.5. Strategic Overview
        12. Denali Therapeutics Inc.
         12.1. Company Overview
         12.2. Product Portfolio
         12.3. SWOT Analysis
         12.4. Financial Overview
         12.5. Strategic Overview
        13. Voyager Therapeutics, Inc.
         13.1. Company Overview
         13.2. Product Portfolio
         13.3. SWOT Analysis
         13.4. Financial Overview
         13.5. Strategic Overview
        14. Bluebird Bio, Inc.
         14.1. Company Overview
         14.2. Product Portfolio
         14.3. SWOT Analysis
         14.4. Financial Overview
         14.5. Strategic Overview
        15. Homology Medicines, Inc.
         15.1. Company Overview
         15.2. Product Portfolio
         15.3. SWOT Analysis
         15.4. Financial Overview
         15.5. Strategic Overview
        16. Prevail Therapeutics Inc.
         16.1. Company Overview
         16.2. Product Portfolio
         16.3. SWOT Analysis
         16.4. Financial Overview
         16.5. Strategic Overview
        17. Aspa Therapeutics, Inc.
         17.1. Company Overview
         17.2. Product Portfolio
         17.3. SWOT Analysis
         17.4. Financial Overview
         17.5. Strategic Overview
        18. Genethon
         18.1. Company Overview
         18.2. Product Portfolio
         18.3. SWOT Analysis
         18.4. Financial Overview
         18.5. Strategic Overview
        19. JCR Pharmaceuticals Co., Ltd.
         19.1. Company Overview
         19.2. Product Portfolio
         19.3. SWOT Analysis
         19.4. Financial Overview
         19.5. Strategic Overview
        20. uniQure N.V.
         20.1. Company Overview
         20.2. Product Portfolio
         20.3. SWOT Analysis
         20.4. Financial Overview
         20.5. Strategic Overview

  • 16.   Pre and Post COVID-19 Impact
    •   1. Positive influence on the healthcare industry
        2. The financial disruption of the manufacturing sector
        3. Impact of COVID-19 on emerging companies
        4. Significant mandates in the healthcare regulations initiated by
                   administrations
        5. The overall economic slowdown of the developing and developed nations

  • 17.   FutureWise SME Key Takeaway Points for Client
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