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Phase-Xs | Biologics & Biosimilars | Coherent Market Insights

Impact Analysis of Covid-19

The complete version of the Report will include the impact of the COVID-19, and anticipated change on the future outlook of the industry, by taking into the account the political, economic, social, and technological parameters.

GM1 Gangliosidosis is an inherited central nervous system disorder that destroys nerve cells in the spinal cord and brain. GM1 gangliosidosis is classified mainly into three types depending upon the signs and symptoms of the disorder. Type 1 GM1 gangliosidosis includes severe signs and symptoms such as mental regression and development of an exaggerated startle reaction to loud noises. Type 1 GM1 gangliosidosis usually occurs in newborns under 6 months of age. Type 2 GM1 gangliosidosis is also known as the late infantile and juvenile form. Children suffering from this usually exhibit symptoms such as mental regression till the age of 18. In type 3 GM1 gangliosidosis (also known as the adult or chronic form), the symptoms are usually seen in adults but exhibit a very low disease spectrum.

The global GM1 gangliosidosis treatment market size is estimated to be valued at US$ 142.1 million in 2025 and is expected to exhibit a CAGR of 35.6 % during the forecast period (2025-2030).

Figure 1. Global GM1 Gangliosidosis Treatment Market Value (US$ Mn), 2025 - 2030

GM1 Gangliosidosis Treatment  | Coherent Market Insights

GM1 gangliosidosis is a rare central nervous system disorder and can develop throughout the lifetime in diagnosed individuals. For instance, in 2019, according to World Health Organization WHO data, around 2,000 patients suffer from GM1 ganglioside across the world. According to the same data source, incidence GM1 gangliosidosis accounts for around 0.5-1.0/100,000 births across the globe.

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GM1 Gangliosidosis Treatment Market Report Coverage

Report Coverage Details
Base Year: 2019 Market Size in 2025: US$ 142.1 Mn
Historical Data for: 2016 to 2019 Forecast Period: 2020 to 2027
Forecast Period 2025 to 2030 CAGR: 36.5% 2030 Value Projection: US$ 651.7 Mn
Geographies covered:
  • North America: U.S. and Canada
  • Europe: Germany, U.K., Spain, France, Italy, Russia, and Rest of Europe
  • Rest of World: Latin America, Asia Pacific, Middle East and Africa.
Segments covered:
  • By Product Type: LYS-GM101, PBGM01, AXO-AAV-GM1.
  • By Disease Type: Type 1 GM1 Gangliosidosis, Type 2 GM1 Gangliosidosis, Type 3 GM1 Gangliosidosis.
Companies covered:

Axovant Gene Therapies Ltd, Passage Bio and Lysogene.

Growth Drivers:
  • Key players are focusing on research and development of treatment of GM1 gangliosidosis
  • Key players are focusing on inorganic strategies such as collaborations for development of gene therapy treatment for GM1 gangliosidosis
Restraints & Challenges:
  • Low awareness about GM1 gangliosidosis in emerging countries

Global GM1 Gangliosidosis Treatment Market – Impact of Coronavirus (COVID-19)

Following the outbreak of COVID-19 in December 2019, the disease has spread to over 100 countries across the globe and the World Health Organization has declared it a public health emergency. According to the World Health Organization’s report, the manifestation of the coronavirus (COVID-19) has resulted in more than 52.7 million infected individuals worldwide as of November 13, 2020.

COVID-19 has affected each and every market across the world and has also impacted the global GM1 gangliosidosis treatment market in the following ways; by directly affecting production and demand, by creating disruptions in distribution channels, and through its financial impact on firms and financial markets. Due to the lockdown, several countries such as China, India, Saudi Arabia, UAE, Egypt, and others are facing problems with regards to the transportation of drugs from one place to another.

Furthermore, players operating in the global GM1 gangliosidosis market are facing major challenges on various fronts due to the COVID-19 pandemic. The major challenges include supply of raw materials for manufacturing drug formulations due to irregularities in transportation facility. Moreover, distributors of products are experiencing irregular demand for products from the retailers due to increasing patient population suffering from COVID-19 and other life threatening disorders.

Figure 2. Global GM1 Gangliosidosis Treatment Market Share (%), by Region, 2025

GM1 Gangliosidosis Treatment  | Coherent Market Insights

Among region, the North America GM1 gangliosidosis market is expected to witness significant growth during the forecast period, owing to high disease population in the U.S. For instance, in 2019, according to National Center for Biotechnology Information (NCBI) data, the GM1 gangliosidosis accounts for around 500 patients, which is expected to drive growth of the U.S market.

Key Players

Major players operating in the global GM1 Gangliosidosis Treatment market are Axovant Gene Therapies Ltd. and Passage Bio and Lysogene.

GM1 gangliosidosis is a progressive neurological genetic disorder mainly caused by the absence of a vital enzyme known as beta-galactosidase (GLB1). Without GLB1, a fatty substance called GM1 gangliosidosis accumulates in cells, and nerve cells of the brain. GM1 gangliosidosis is one of the 50 genetically inherited disorders known as lysosomal storage diseases. Currently, there is no treatment or cure for treatment of GM1 gangliosidosis.

Market Dynamics

Key players are focusing on strategic agreements in order to expand their presence in the market. For instance, in May 2017, Lysogene entered a strategic manufacturing agreement with Brammer Bio, a viral gene and cell therapy manufacturer. The agreement is expected to help Brammer Bio manufacture LYS-GM101, an AAVrh10- based gene therapy for the treatment of GM1 gangliosidosis.

Key features of the study:

  • This report studies the current scenario as well as the future potential of the global GM1 gangliosidosis market. The market for GM1 gangliosidosis is segmented on the basis of product type, disease type, and region.
  • This report provides in-depth analysis of the global GM1 gangliosidosis market, and provides market size (US$ Mn) and compound annual growth rate (CAGR) (%) for the forecast period (2025–2030)
  • It elucidates potential revenue opportunity across different segments and explains attractive investment proposition matrix for this market. It provides valuable insights about market drivers, restraints, opportunities, country outlook, and competitive strategies adopted by the leading players.
  • It profiles leading players in the global GM1 gangliosidosis market based on the following parameters – company overview, financial performance, product portfolio, geographical presence, key developments, and future plans
  • Insights from this report would allow marketers and management authorities of companies to make informed decisions with respect to future market expansion and marketing tactics
  • The global GM1 gangliosidosis market report caters to various stakeholders in this industry, including investors, product developers and manufacturers, distributors, healthcare organizations, research institutes, new entrants, and financial analysts
  • Stakeholders would greatly benefit in decision making through various strategy matrices used in analyzing the global GM1 gangliosidosis market

Detailed Segmentation:

  • Global GM1 Gangliosidosis Treatment Market, By Product Type :
    • LYS-GM101
    • PBGM01
    • AXO-AAV-GM1
  • Global GM1 Gangliosidosis Treatment Market, By Disease Type :
    • Type 1 GM1 Gangliosidosis
    • Type 2 GM1 Gangliosidosis
    • Type 3 GM1 Gangliosidosis
  • Global GM1 Gangliosidosis Treatment Market, By Region:
    • North America
      • 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 Country:
        • U.S.
        • Canada
    • Europe
      • 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 Country:
        • Germany
        • U.K.
        • France
        • Italy
        • Spain
        • Russia
        • Rest of Europe
    • Rest of World
      • 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:
        • Latin America
        •  Asia Pacific
        •  Middle East
        • Africa.
  • Company Profiles.
    • Axovant Gene Therapies Ltd. *
      • Company Overview
      • Material Portfolio
      • Financial Performance
      • Key Highlights
      • Market Strategies
    • Passage Bio
    • Lyosgene

 “*” marked represents similar segmentation in other categories in the respective section.

Table of Contents

  1. Research Objectives and Assumptions
    • Research Objectives
    • Assumptions
    • Abbreviations
  2. Market Purview
    • Report Description
      • Market Definition and Scope
    • Executive Summary
      • Market Snapshot, By Product Type
      • Market Snapshot, By Disease Type
      • Market Snapshot, By Region
      • Coherent Opportunity Map (COM)
  3. Market Dynamics, Regulations, and Trends Analysis
    • Drivers
    • Restraints
    • Market Opportunities
    • Impact Analysis
    • Pipeline Analysis
    • Regulatory Scenario
    • Key Development
    • Epidemiology
    • Primary Research Insights
      • Patient Insights
      • Physician Insights
      • Manufacturer Insights
  4. Global GM1 Glangliosidosis Treatment Market- Impact of COVID-19 Pandemic
    • Economic Impact
    • COVID-19 Epidemiology
    • Impact of COVID-19 on Ongoing Clinical Trials
  5. Global GM1 Glangliosidosis Treatment Market, By Product Type, 2016-2027 (US$ Mn)
    • Introduction
      • Market Share Analysis, 2020 and 2027 (%)
      • Y-o-Y Growth Analysis, 2017–2027
      • Segment Trends
        • LYS-GM101
          • Introduction
          • Market Size and Forecast, and Y-o-Y Growth, 2016–2027, (US$ Mn)
        • PBGM01
          • Introduction
          • Market Size and Forecast, and Y-o-Y Growth, 2016–2027, (US$ Mn)
        • AXO-AAV-GM1
          • Introduction
          • Market Size and Forecast, and Y-o-Y Growth, 2016–2027, (US$ Mn)
  6. Global GM1 Glangliosidosis Treatment Market, By Disease Type, 2016–2027 (US$ Mn)
    • Introduction
      • Market Share Analysis, 2020 and 2027 (%)
      • Y-o-Y Growth Analysis, 2017–2027
      • Segment Trends
        • Type 1 GM1 Gangliosidosis
          • Introduction
          • Market Size and Forecast, and Y-o-Y Growth, 2016–2027, (US$ Mn)
        • Type 2 GM1 Gangliosidosis
          • Introduction
          • Market Size and Forecast, and Y-o-Y Growth, 2016–2027, (US$ Mn)
        • Type 3 GM1 Gangliosidosis
          • Introduction
          • Market Size and Forecast, and Y-o-Y Growth, 2016–2027, (US$ Mn)
  7. Global GM1 Glangliosidosis Treatment Market, By Region, 2016–2027 (US$ Mn)
    • Introduction
      • Market Share Analysis, By Country, 2020 and 2027 (%)
      • Y-o-Y Growth Analysis, For Countries, 2017–2027
    • North America
      • Introduction
      • Market Size and Forecast, By Product Type, 2016–2027 (US$ Mn)
      • Market Size and Forecast, By Disease Type, 2016–2027 (US$ Mn)
      • Market Size and Forecast, By Country, 2016–2027 (US$ Mn)
        • U.S.
        • Canada
    • Europe
      • Introduction
      • Market Size and Forecast, By Product Type, 2016–2027 (US$ Mn)
      • Market Size and Forecast, By Disease Type, 2016–2027 (US$ Mn)
      • Market Size and Forecast, By Country, 2016–2027 (US$ Mn)
        • Brazil
        • Mexico
        • Argentina
        • Rest of Latin America
    • Rest of World
      • Introduction
      • Market Size and Forecast, By Product Type, 2016–2027 (US$ Mn)
      • Market Size and Forecast, By Disease Type, 2016–2027 (US$ Mn)
      • Market Size and Forecast, By Country, 2016–2027 (US$ Mn)
        • Latin America
        • Asia Pacific
        •  Middle East
        • Africa.
  8. Competitive Landscape
    • Company Profiles
      • Axovant Gene Therapies Ltd.
        • Company Overview
        • Clinical Trial Phase Portfolio
        • Financial Overview
        • Key Highlights
        • Market Strategies
      • Passage Bio
        • Company Overview
        • Clinical Trial Phase Portfolio
        • Financial Overview
        • Key Highlights
        • Market Strategies
      • Lysogene
        • Company Overview
        • Clinical Trial Phase Portfolio
        • Financial Overview
        • Key Highlights
        • Market Strategies

*Browse 16 market data tables and 16 figures on “GM1 Glangliosidosis Treatment Market– Global forecast to 2027”.

FAQgrowicon

Frequently Asked Questions

The global GM1 gangliosidosis treatment market size is estimated to be valued at US$ 142.1 million in 2025 and is expected to exhibit a CAGR of 35.6 % between 2025 and 2030.
Key players are focusing on research and development of treatment of GM1 gangliosidosis and key players are focusing on inorganic strategies such as collaborations for development of gene therapy treatment for GM1 gangliosidosis.
LYS-GM101 segment is estimated to hold major market share in the global gangliosidosis treatment market, as LYS-GM101 is has shown greater efficiency in treatment of GM1 Gangliosidosis rare disorder with recombinant adeno- associated virus 9aav, which directly acts on the nerve cells of the patients.
Type-1 GM1 gangliosidosis segment is estimated to hold major market share in the global gangliosidosis treatment market, owing to rising prevalence of type-1 GM1 gangliosidosis in newborns.
North America GM1 gangliosidosis treatment market is estimated to generate largest revenue share in 2020.
Low awareness about GM1 gangliosidosis in emerging countries, which is a key factor hampering growth of the market.
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