Sandhoff Disease Therapeutics Market
Number of pages: 100 | Report Format: PDF | Published date: May 11, 2023
Historical Years – 2021 | Base Year – 2022 | Forecasted Years – 2023 to 2031
|
Report Attribute |
Details |
|
Market Size Value in 2022 |
US$ 9.8 billion |
|
Revenue Forecast in 2031 |
US$ 15.38 billion |
|
CAGR |
5.75% |
|
Base Year For Estimation |
2022 |
|
Forecast Period |
2023 to 2031 |
|
Historical Year |
2021 |
|
Segments Covered |
Product, Technology, Services, End User, and Region |
|
Regional Scope |
North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa |
According to the deep-dive market assessment study by Growth Plus Reports, the global Sandhoff disease therapeutics market was valued at US$ 9.8 billion in 2022 and is expected to register a revenue CAGR of 5.75% to reach US$ 15.38 billion by 2031.
Sandhoff Disease Therapeutics Market Fundamentals
Sandhoff disease is a rare lysosomal disease caused by mutations in both copies of the HEXB gene, which provides instructions for producing a specific enzyme called beta-hexosaminidase. This enzyme is responsible for breaking down a fatty substance called GM2 ganglioside. As a result of the mutations in the HEXB gene, GM2 ganglioside accumulates in the brain and spinal cord, leading to progressive damage to nerve cells. Symptoms of Sandhoff disease include developmental delays, seizures, muscle weakness, and vision and hearing loss. The disease affects males and females in equal numbers and is commonly seen in Creole population of Argentina and people with Lebanese ancestry.
Advancements in medical research and diagnosis methods are introducing new and improved treatment options for treating Sandhoff diseases. Recently, substrate reduction therapy has been observed as a potential treatment option for Sandhoff disease. Subtract reduction therapy attempts to prevent hazardous chemical buildup by reducing the generation of substrates digested by the defective enzyme.
The research study on the Sandhoff disease therapeutics market overview thoroughly analyzes the market, along with the aspects that either help or hinder the market's growth, such as its drivers, challenges, and opportunities. The research also discusses the list of businesses that operate in this market and their efforts to establish themselves as major players through expansion plans and innovative ideas. It also emphasizes current events that support the market's revenue expansion.
This report is an important tool for the industry stakeholders to gather all the information about the market, customers, and competition. The report also provides the readers with valuable insights on the segmentation, regions, market size & forecast, revenue CAGRs, and other valuable data to help make informed strategic decisions. The study compiles information gathered from industry participants and specialists across the industry value chain. The report also taps into the qualitative and quantitative assessments by industry analysts. Our reports use different methods for estimating the market size and forecast, including secondary research, surveys and interviews, and statistical modeling. With these steps, our reports provide the most accurate market data. These datasets will cumulatively provide an in-depth Sandhoff disease therapeutics market forecast to the industry stakeholders.
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Sandhoff Disease Therapeutics Market Dynamics
The rising prevalence of the disease and its increased awareness is the major factor increasing the Sandhoff disease therapeutics market. According to 2020 estimates by the National Organization of Rare Diseases (NORD), Sandhoff disease affects 1 in 1,000,000 individuals. It affects both men and women in equal proportions. The disease affects various populations, although it is most prevalent in Argentina's Creole community, Metis people in Saskatchewan, Canada, and people with Lebanese heritage.
Additionally, increasing focus on research and development (R&D) significantly shapes the Sandhoff disease therapeutics market. The growing understanding of the disease's underlying mechanisms, along with advancements in biotechnology and genetic engineering, has fueled an upsurge in R&D efforts for the management of the disease. Moreover, researchers and pharmaceutical companies are actively exploring innovations in gene therapy, enzyme replacement therapy (ERT), substrate reduction therapy (SRT), and small molecule therapies to address unmet medical need in Sandhoff disease.
However, the expensive cost of treatment for Sandhoff disease treatment is a significant challenge in the global market. Furthermore, low healthcare standards and less awareness, especially in low-income countries such as the Middle East and Africa, can also impede the growth of the Sandhoff disease therapeutics market.
Sandhoff Disease Therapeutics Market Ecosystem
Sandhoff Disease Therapeutics Market, by Therapy
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Sandhoff Disease Therapeutics Market, by Type
Sandhoff Disease Therapeutics Market, by End User
Sandhoff Disease Therapeutics Market by Segments
Gene therapy accounted for the majority of share in the global Sandhoff disease therapeutics market. This is majorly attributed to the favorable results obtained by research on the efficacy of gene therapy in treating Sandhoff disease. Additionally, gene therapy offers restoration of the enzyme, leading to the reduction of substrate accumulation and proper management. Moreover, increased advancements in gene delivery technologies and their long-lasting effects can also contribute to the growth of the Sandhoff disease therapeutics market.
Segmentation of the market refers to the division of the industry into subgroups based on factors such as product, technology, services, end user, etc. Market segmentation data helps businesses to understand the preferences and unique needs of different customer groups and develop targeted sales strategies. This information also helps in identifying potential Sandhoff Disease Therapeutics market growth opportunities.
Sandhoff Disease Therapeutics Market by Region
North America accounted for 41% of the global Sandhoff disease therapeutics market. This is majorly due to the robust healthcare infrastructure, strong research and development capabilities, and regulatory support that contributes to the advancement of therapies for Sandhoff disease. Moreover, North America consists of several prominent biotechnology and pharmaceutical companies actively engaged in developing novel treatments for rare diseases. For instance, December 2018, Sio Gene Therapies, formerly known as Axovent Gene Therapies, licensed AXO-AAV-GM2 gene therapies from the University of Massachusetts School of Medicine aimed at treating Tay-Sachs and Sandhoff diseases. The first patient dosed with AXO-AAV-GM2 gene therapy was reported in 2021.
The Asia Pacific region is expected to show significant developments in the field of Sandhoff disease therapeutics, driving the growth of the Sandhoff disease therapeutics market in the region. The region reports approximately 22% of the global market in 2022. Rising awareness about the disease among healthcare professionals and the general population, coupled with increasing government initiatives to support rare disease treatments, has further fueled the market revenue growth.
Based on the regions, the global Sandhoff disease therapeutics market is segmented into:
Regional demarcation of the industry will provide information on geographic pockets regarding Sandhoff Disease Therapeutics market outlook, size, share, and growth rate. This data helps businesses evaluate the expansion potential into new regional markets, understand the competitive threats across different regions, and develop localized sales and expansion strategies.
Key Components of the Report
Sandhoff Disease Therapeutics Market Competitive Landscape
Some of the major market players in the Sandhoff disease therapeutics market are:
The market competitive landscape is performed by collecting and analyzing information about the key competitors, the present industry trends, and market dynamics. It includes information collection and analysis on factors such as products, pricing, geographic outreach, customer demographics, marketing strategies, and recent developments. Competitive landscape assessment will help businesses identify the current prevalent or potential opportunities and threats keeping in mind the Sandhoff Disease Therapeutics industry trends.
Sandhoff Disease Therapeutics Market Strategic Developments
Key Strengths of Our Report
Target Audience to Benefit from this Report.
Reasons to Buy this Report
Sandhoff disease is a rare genetic disorder that affects the nervous system. It is caused by the absence or deficiency of an enzyme that breaks down certain fats in the body, leading to a buildup of these fats in the brain and other organs.
The global Sandhoff disease therapeutics market was valued at US$ 9.8 billion in 2022.
North America region accounted for 41% of the Sandhoff disease therapeutics market in the forecast years.
The global Sandhoff disease therapeutics market is expected to register a revenue CAGR of 5.75% during the forecast period from 2023 to 2031.
The global Sandhoff disease therapeutics market is expected to register US$ 15.38 billion by 2031.
One of the most prominent Sandhoff disease therapeutics industry trends is the development of gene therapies that aim to replace or repair the faulty genes responsible for the disease. Another trend is the increasing focus on early diagnosis and intervention to improve patient outcomes.
The global Sandhoff disease therapeutics market is expected to grow significantly in the coming years due to innovations in treatment options.
The lack of awareness about the Sandhoff disease among people and healthcare professionals and the high cost of treatment, and the limited availability of effective therapies also pose significant challenges to the growth of the market.
Major strategies adopted by key market players include product innovation, and strategic partnerships.
*Insights on financial performance are subject to the availability of information in the public domain