GM1 Gangliosidosis Global Market Report 2025

The GM1 gangliosidosis market size has grown rapidly in recent years. It will grow from $0.51 billion in 2024 to $0.57 billion in 2025 at a compound annual growth rate (CAGR) of 11.4%. The growth during the historic period was driven by the increasing number of patients with GM1 gangliosidosis, a growing focus on the disorder within the medical community, rising awareness of rare diseases, the increasing popularity of patient-centric research, and a greater emphasis on early intervention.

The GM1 gangliosidosis market size is expected to see rapid growth in the next few years. It will grow to $0.87 billion in 2029 at a compound annual growth rate (CAGR) of 11.1%. The projected growth in the forecast period can be attributed to the rising cases of GM1 gangliosidosis, an increasing prevalence of lysosomal storage disorders, a growing number of clinical trials, advancements in gene therapy and gene editing, the expansion of personalized and precision medicine, and the rising adoption of genetic screening programs. Key trends expected during this period include advancements in gene-editing technologies, the integration of technology in healthcare, improvements in diagnostic techniques, the development of advanced formulations, and progress in genetic therapies.

The growing use of gene therapy is expected to drive the expansion of the GM1 gangliosidosis market. Gene therapy is a medical approach that involves modifying or replacing genes within a patient’s cells to treat or prevent diseases. The rising adoption of gene therapy is fueled by advancements in genetic research, improved treatment outcomes for previously untreatable conditions, and increasing regulatory support for innovative therapies. In the case of GM1 gangliosidosis, gene therapy involves delivering a functional copy of the defective GLB1 gene to patient cells, restoring β-galactosidase enzyme activity, and reducing toxic substrate buildup. For example, in April 2024, the American Society of Gene & Cell Therapy, a US-based non-profit medical and scientific organization, reported a 10% increase in the number of phase III gene therapy clinical trials in the fourth quarter, marking the first rise since the third quarter of 2022. As a result, the expanding adoption of gene therapy is contributing to the growth of the GM1 gangliosidosis market.

Leading companies in the GM1 gangliosidosis market are prioritizing strategic partnerships to maintain their market presence. These collaborations play a key role in advancing gene therapy development by pooling expertise, resources, and technologies to accelerate research, clinical trials, and commercialization. For instance, in October 2024, GEMMA Biotherapeutics Inc., a US-based biotechnology company, collaborated with Oswaldo Cruz Foundation (Fiocruz), a Brazil-based health research institution, to develop gene therapies for rare diseases, including GM1 gangliosidosis. This agreement includes up to $100 million in funding from Fiocruz to support clinical research and manufacturing, with the goal of making these advanced therapies available through Brazil's public healthcare system. The partnership also emphasizes technology transfer and aims to expand access to treatments that were previously limited to wealthier populations.

In August 2024, Passage Bio Inc., a US-based biotech company, entered into a partnership with GEMMA Biotherapeutics to advance pediatric gene therapies. As part of this agreement, Passage Bio is out-licensing three pediatric gene therapies targeting lysosomal storage diseases, including PBGM01 for GM1 gangliosidosis, PBKR03 for Krabbe disease, and PBML04 for metachromatic leukodystrophy. GEMMA Biotherapeutics Inc. is a US-based biotechnology research company specializing in the development and commercialization of genetic medicines for rare pediatric diseases.

Major players 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., and SphinCS GmbH.

North America was the largest region in the GM1 gangliosidosis market in 2024. The regions covered in GM1 gangliosidosis report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa.

The countries covered in the GM1 gangliosidosis market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

GM1 gangliosidosis is a rare, inherited neurodegenerative disorder caused by a deficiency of the β-galactosidase (GLB1) enzyme, leading to the toxic accumulation of GM1 gangliosides in the brain and other tissues. This condition results in progressive neurological decline, developmental delays, muscle weakness, and organ enlargement.

The primary types of GM1 gangliosidosis include Type 1, Type 2, and Type 3 GM1 gangliosidosis. Type 1 GM1 gangliosidosis is the most severe form, diagnosed in infancy, and is marked by rapid neurological degeneration. Affected children experience progressive motor and cognitive decline, often leading to early death. Treatment options for GM1 gangliosidosis include anticonvulsants, bone marrow transplantation, cord-blood hematopoietic stem cell transplantation, enzyme replacement therapy, and gene therapy. These treatments are primarily utilized in research institutes, hospitals, and other medical facilities.

The GM1 gangliosidosis market research report is one of a series of new reports that provides GM1 gangliosidosis market statistics, including GM1 gangliosidosis industry global market size, regional shares, competitors with a GM1 gangliosidosis market share, detailed GM1 gangliosidosis market segments, market trends and opportunities, and any further data you may need to thrive in the GM1 gangliosidosis industry. This GM1 gangliosidosis market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenario of the industry.

The GM1 gangliosidosis market consists of revenues earned by entities by providing services such as gene therapy, diagnostic testing, patient care, and clinical trials. The market value includes the value of related goods sold by the service provider or included within the service offering. The GM1 gangliosidosis market also includes sales of diagnostic tools, research kits, biomarkers, and assistive devices. Values in this market are ‘factory gate’ values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).

The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.

This product will be delivered within 3-5 business days.