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Key Trend Reshaping the GM1 Gangliosidosis Market in 2025: Strategic Partnership To Advance Gene Therapies For Rare Diseases
GM1 Gangliosidosis Market
The increasing use of gene therapy is set to drive the growth of the GM1 gangliosidosis market. Gene therapy is a medical approach that involves altering or replacing genes within an individual's cells to treat or prevent diseases. The increasing adoption of gene therapy is driven by advancements in genetic research, better treatment outcomes for previously untreatable conditions, and greater regulatory support for innovative therapies. Gene therapy for GM1 gangliosidosis involves introducing a functional copy of the defective GLB1 gene into patient cells to restore ß-galactosidase enzyme activity and reduce the buildup of toxic substrates. For instance, in April 2024, the American Society of Gene & Cell Therapy, a US-based non-profit organization, reported that the number of phase III gene therapy clinical trials increased by 10% in the fourth quarter, marking the first rise since the third quarter of 2022. Consequently, the increasing adoption of gene therapy is driving the growth of the GM1 gangliosidosis market.
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What is the projected compound annual growth rate (CAGR) of the gm1 gangliosidosis market from 2025 to 2034, and what factors influence it?
The GM1 gangliosidosis market has expanded rapidly, growing from $0.51 billion in 2024 to $0.57 billion in 2025 at a CAGR of 11.4%. Growth in this segment is driven by an increasing number of GM1 gangliosidosis patients, heightened focus on rare diseases, greater awareness, patient-centric research initiatives, and an increased emphasis on early intervention.
The GM1 gangliosidosis market is projected to expand rapidly, reaching $0.87 billion by 2029 at a CAGR of 11.1%. The expected growth is attributed to rising cases of GM1 gangliosidosis, increasing occurrences of lysosomal storage disorders, a surge in clinical trials, a greater focus on gene therapy and gene editing, an increase in personalized and precision medicine, and wider adoption of genetic screening programs. Key trends include breakthroughs in gene-editing technologies, integration of digital tools in healthcare, advancements in diagnostic methods, the development of advanced formulations, and improvements in genetic therapies.
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How are the latest trends influencing the growth of the gm1 gangliosidosis market?
Major companies in the GM1 gangliosidosis market are focusing on strategic partnerships to advance gene therapy development. These partnerships combine resources and expertise to accelerate research and clinical trials. In October 2024, GEMMA Biotherapeutics Inc., a US-based biotechnology company, partnered with the Oswaldo Cruz Foundation (Fiocruz) in Brazil to develop gene therapies for rare diseases, including GM1 gangliosidosis. This collaboration, with up to $100 million in funding, aims to bring advanced therapies to public healthcare systems in Brazil, democratizing access to treatments that were previously limited to wealthier populations.
What are the major segments of the gm1 gangliosidosis market and their role in driving growth?
The GM1 gangliosidosis market covered in this report is segmented -
1) By Disease Type: Type 1 GM1 Gangliosidosis, Type 2 GMT Gangliosidosis, Type 3 GMT Gangliosidosis
2) By Treatment: Anticonvulsants, Bone Marrow Transplantation, Cord-Blood Hematopoietic Stem-Cell Transplantation, Enzyme Replacement, Gene Therapy
3) By End-User: Research Institutes, Hospitals, Other End-Users
Subsegments:
1) By Type 1 GM1 Gangliosidosis: Classic Infantile Onset, Atypical Infantile Onset
2) By Type 2 GM1 Gangliosidosis: Classic Juvenile Onset, Atypical Juvenile Onset
3) By Type 3 GM1 Gangliosidosis: Classic Adult Onset, Atypical Adult Onset
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Which regions are key players in the growth of the gm1 gangliosidosis market?
North America was the largest region in the GM1 gangliosidosis market in 2024. The regions covered in the GM1 gangliosidosis market report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East, Africa.
Who are the key firms paving the way for growth in the gm1 gangliosidosis market?
Major companies operating in the GM1 gangliosidosis market are Pfizer Inc., Sanofi S.A., Novartis AG, Takeda Pharmaceutical Company Limited, Sarepta Therapeutics Inc., CRISPR Therapeutics AG, REGENXBIO Inc., Passage Bio Inc., Orchard Therapeutics plc, AVROBIO Inc., uniQure N.V., GEMMA Biotherapeutics Inc., Lysogene S.A., AZAFAROS B.V., BioStrategies LC, Sio Gene Therapies Inc., Gain Therapeutics Inc., Dorphan S.A., bluebird bio Inc., Axovant Gene Therapies Ltd., SphinCS GmbH.
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What Is Covered In The GM1 Gangliosidosis Global Market Report?
•Market Size Forecast: Examine the gm1 gangliosidosis market size across key regions, countries, product categories, and applications.
•Segmentation Insights: Identify and classify subsegments within the gm1 gangliosidosis market for a structured understanding.
•Key Players Overview: Analyze major players in the gm1 gangliosidosis market, including their market value, share, and competitive positioning.
•Growth Trends Exploration: Assess individual growth patterns and future opportunities in the gm1 gangliosidosis market.
•Segment Contributions: Evaluate how different segments drive overall growth in the gm1 gangliosidosis market.
•Growth Factors: Highlight key drivers and opportunities influencing the expansion of the gm1 gangliosidosis market.
•Industry Challenges: Identify potential risks and obstacles affecting the gm1 gangliosidosis market.
•Competitive Landscape: Review strategic developments in the gm1 gangliosidosis market, including expansions, agreements, and new product launches.
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GM1 gangliosidosis is a rare, inherited lysosomal storage disorder caused by mutations in the GLB1 gene, leading to a deficiency of the β-galactosidase enzyme. This results in the accumulation of GM1 gangliosides in cells, primarily affecting the central nervous system. The disease is classified into infantile, juvenile, and adult-onset forms, with infantile GM1 being the most severe and often fatal in early childhood.
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