GM1 Gangliosidosis Treatment Market Size, Share, Industry, Forecast and Outlook (2024-2031)

Global GM1 Gangliosidosis Treatment Market is segmented By Type (GM1 Gangliosidosis Type 1 (Infantile), GM1 Gangliosidosis Type 2 (Juvenile), GM1 Gangliosidosis Type 3 (Adult)), By Treatment (Enzyme Replacement Therapy, Gene Therapy, Stem Cell Transplants, Substrate Reduction Therapy, Pharmacological Chaperones, Miglustat, N-Butyl-Deoxynojirimycin (NB-DNJ), Anticonvulsant Drugs), By End User (Hospitals, Ambulatory Surgical Centers, Others), and By Region (North America, South America, Europe, Asia Pacific, Middle East, and Africa) – Share, Size, Outlook, and Opportunity Analysis, 2023-2030

GM1 Gangliosidosis Treatment Market Overview

Global GM1 gangliosidosis treatment market is expected to reach at a high CAGR during the forecast period 2023-2030.

The global GM1 gangliosidosis treatment market has witnessed significant growth and transformations over the years, with various factors influencing its dynamics. Rising lysosomal storage disorders, research and development and government investments in pharmaceuticals and biotechnology are among the key drivers impacting GM1 gangliosidosis treatment globally. After the challenging year of 2020, the GM1 gangliosidosis treatment industry showed decent resilience and adaptability.

Government investments in lysosomal storage disorders, particularly in developing economies, will continue to drive utilization of gene therapy and boost the global GM1 gangliosidosis treatment market. The global GM1 gangliosidosis treatment industry is placing increasing emphasis on better management of GM 1 gangliosidosis. The respective initiatives includes the use of other treatment, such as enzyme replacement therapies for treatment of gangliosidosis.

The GM1 gangliosidosis type 1 (infantile), accounts for over one third of the market share. Similarly, the North America dominates the GM1 gangliosidosis treatment market, capturing the largest market share of over 1/3rd. The region’s rapid investments in healthcare and rising major players in the GM1 gangliosidosis treatment market globally, makes it the largest market share holder. United States has the largest pool of manufacturers as well as consumers.

GM1 Gangliosidosis Treatment Market Scope

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GM1 Gangliosidosis Treatment Market Dynamics

Growing Research and Development by Researchers

Developed economies have been witnessing rapid growth in betterment of their healthcare sectors, driven by high income levels, investments and infrastructure development. Several countries have experienced substantial demand for GM1 gangliosidosis treatment, reflecting their expanding healthcare industries. Rising research and development for the same will be a crucial factor driving the growth of the market. 

On May 30, 2023, in patient plasma, urine, and cerebral fluid (CSF), two pentasaccharide biomarkers, H3N2b and H3N2a, were raised more than 18-fold. In the cat model, only H3N2b was discernible, and it was inversely linked with -galactosidase activity. Reduction of H3N2b was seen in the central nervous system, plasma, urine, and CSF samples that were collected from the cat model as well as in the urine, plasma, and CSF samples from a patient after intravenous (IV) AAV9 gene therapy treatment. 

The restoration of the normal range of neuropathology in the cat model and the enhancement of clinical results in the patient were appropriately mirrored by the reduction of H3N2b. These findings show that H3N2b is a valuable pharmacodynamic biomarker for GM1 gangliosidosis gene therapy efficacy assessment. The discovery that H3N2b is a frequent biomarker in the cat model and for people with GM1 gangliosidosis raises the possibility that this pentasaccharide can help in the translation of novel therapies from the cat model to humans.

Rising Number of Clinical Outcomes with Positive Outcomes

Rising clinical trials with positive outcomes for treatment of GM1 gangliosidosis will drive the growth of the market in coming period of time. For instance, on December 14, 2022, new interim security, biomarker, and clinical development data from cohorts 1-3 in the Imagine-1 clinical research have been released by Passage Bio, Inc., a trial-stage genetic medicines firm that specializes in developing transformative treatments for illnesses of the central nervous system (CNS). Imagine-1 is a Phase 1/2, international, open-label, dose-escalation research of the intra-cisternal magna (ICM) injection with the AAVhu68 gene therapy PBGM01 in four cohorts of young patients with late or early infantile GM1 Gangliosidosis (GM1). 

In the rare and deadly lysosomal storage disease GM1, mutations in the GLB1 gene cause the enzyme beta-galactosidase (-Gal) to function at very low levels. Six treated individuals from the initial three groups are represented in the interim results. Each cohort had three patients: cohort 1 (late infantile, lower dose), cohort 2 (late infantile, heavy dose), and cohort 3 (early infantile, low dose). By the middle of 2023, results for Cohort 4 (early infantile, large dosage) participants should be available.

Challenges Associated with Gene Therapy  

For monogenic illnesses like GM1, gene therapy is a possible treatment option. It is difficult to achieve worldwide viral genome distribution or the diffusion of a functioning enzyme across the CNS in hereditary illnesses with neuropathology. The BBB not only prevents medications from being delivered to the brain intravenously, but techniques to get around the BBB through intrathecal or intracranial injections are invasive. 

Despite these difficulties, gene therapy has the power to stop the spread of a disease with just one treatment. Only a few of the indications and symptoms of GM1 can currently be treated medically, and even those do not stop the disease from progressing. As a result, some people might think about trying experimental gene therapy.

Limitations Related to the Therapies   

SRT in GM1 is dependent on strong GSL biosynthesis inhibitors. SRT may have a smaller effect on clinical disease if ganglioside production is inhibited without a way to degrade previously accumulated substrate. Therefore, it is believed that some residual -gal activity is necessary, limiting the use of SRT to individuals who are not infantile. 

Glucosylceramide Synthase, that catalyzes the first committed step of GSL biosynthesis, is inhibited by the N-alkylated imino-sugar derivatives N-butyl-deoxygalactonojirimycin (NB-DGJ) and N-butyl-deoxynojirimycin (NB-DNJ), which has been FDA-approved for therapy of Gaucher disease. 

GM1 Gangliosidosis Treatment Market Segment Analysis

The global GM1 gangliosidosis treatment market is segmented based on type, treatment, end user and region.

High Advantages of Treatment via Gene Therapy 

To possibly treat the infantile, late infantile, or juvenile forms of GM1, gene therapy is being investigated. A functioning clone of the GLB1 gene will be introduced into cells using gene therapy for GM1 utilizing a viral vector. Cells are instructed to produce the ß-gal enzyme by the GLB1 gene. Scientists have figured out a safe way to harness viruses' propensity for entering cells as a vehicle for delivering functional genes. Only curative (intended) genes are supplied because the viral genes have been eliminated. 

The objective is to reestablish the way lysosomes work, which is to eliminate and stop the accumulation of hazardous chemicals. There are various ways to provide GM1 gene therapy. One method involves administering an intra-cisterna magna or intracisternal injection near the base of the skull. The fluid-filled region around the brain or spinal cord is where the gene therapy is delivered.  A different strategy is to administer the gene therapy intravenously, or into a vein. Then, the bloodstream spreads the gene therapy throughout the body. Clinical studies may vary in their design in a number of ways, including how gene therapy is administered. 

GM1 Gangliosidosis Treatment Market Geographical Share

North America Accounted for Largest Market Share in 2022, Owing to the Strong Presence of Major Players and Increasing Healthcare Infrastructure Investment

Due to the rising need for GM1 gangliosidosis treatment in healthcare, manufacturers in North America have chances of increasing their operations. There are many producers and suppliers in North America and owing to the quick economic growth of the region, industrial production has expanded, driving the demand for GM1 gangliosidosis treatment. 

Increasing expenditure on healthcare and rising research studies, advancement of technologies for different treatments of gangliosidosis, and increase in pharmaceutical or biotech business establishment across the region are also contributing to the growth of GM1 gangliosidosis treatment market share of this region. 

The key healthcare organizations' and businesses' joint research initiatives are also expected to contribute to the rising demand, as will new product development, which constantly looks to improve present options. The market in this area is growing as people become more aware of various novel treatments such as gene therapy or enzyme replacement therapy. The aforementioned elements further attest to North America's hegemonic position in the world.

North America continues to be a key player in the global GM1 gangliosidosis treatment market, with United States leading the way. Government initiatives promoting infrastructure development and investment, and a focus on rising lysosomal storage disorders have fueled the demand for GM1 gangliosidosis treatment in the region. United States have been proactive in executing several initiatives or researches, stimulating GM1 gangliosidosis treatment demand.

COVID-19 Impact Analysis

The outbreak of the COVID-19 pandemic in late 2019 created unprecedented challenges for industries worldwide, including the global GM1 gangliosidosis treatment market. As countries grappled with lockdowns, supply chain disruptions and reduced economic activity, the medical device sector, with a significant consumer of GM1 gangliosidosis treatment, was significantly impacted.

Several efforts all throughout the world were impacted by the pandemic's broad lockdowns and limitations that started in early 2020. Major pharmaceutical or medical device industries came to a standstill and shifted their attention towards the management of COVID-19, leading to a slump in demand for GM1 gangliosidosis treatment. As a result, many pharmaceutical plants either suspended or reduced production to align with the reduced demand.

The COVID-19 pandemic severely disrupted global supply chains, impacting the transportation of raw materials to the pharmaceuticals manufacturing plants. Movement limitations and border closures caused production and supply delays. Additionally, some nations experienced a lack of raw resources as a result of supply chain network interruptions.

With the outbreak of the pandemic, many pharmaceutical or biotechnology manufacturing units faced labor shortages as workers fell sick or were unable to travel to work due to lockdown restrictions. To curb the spread of the virus, pharmaceutical plants implemented stringent safety measures, reducing the number of workers allowed on-site at a time. However, these precautions were required to protect employee health and continue crucial activities throughout the pandemic.

Russia-Ukraine War Impact Analysis

The medical device businesses are starting to worry regarding their capacity to continue operating, much like other parts of the world. Russia's invasion of Ukraine has an effect on millions of people not only in this nation in Eastern Europe but also all around the world. Drug and therapies developers will thus experience delays in the development process, a loss of business consistency, and the possibility of non-compliance for therapies/drugs already on the market. 

For many years, clinical trials in several therapeutic fields have benefited significantly from the participation of Ukraine, Russia, and other CIS nations. Clinical trial durations can frequently be accelerated by incorporating more patients into studies when there is availability to skilled investigators, high-quality patient populations, and big patient populations. Trials that are delayed in Russia and Ukraine as well as relocation to other regions of Europe for fresh trials are currently viable choices. These factors will impact the global GM1 gangliosidosis treatment market.

Key Developments

• On August 7, 2023, new intermediate safety, biomarker, and survival results from cohorts 1-4 in the Imagine-1 clinical research were released by Passage Bio, Inc., a developmental-stage genetic medicines business dedicated to discovering breakthrough treatments for illnesses of the central nervous system (CNS). Imagine-1 is a Phase 1/2, international, open-label, dose-escalation research of the intra-cisternal magna (ICM) injection with the AAVhu68 gene therapy PBGM01 in six cohorts of young patients with late or early infantile GM1 Gangliosidosis (GM1). In the rare and fatal lysosomal storage disease GM1, mutations in the GLB1 gene cause the enzyme beta-galactosidase (-Gal) to function at very low levels.
• On November 29, 2022, the independent Data Safety Monitoring Board (DSMB) has finished its review of the data on safety from Stage 1 of the adaptative clinical trial using LYS-GM101, investigational gene therapy, an AAVrh10 vector to transport the human beta-galactosidase (-gal) coding sequence, for the management of GM1 gangliosidosis, and recommended continuation of the study as plenary. This information was released by Lysogene, a stage 3 gene therapy platform business targeting central

GM1 Gangliosidosis Treatment Market Companies

The major global players in the market include Passage Bio, Lysogene, Sio Gene Therapies, Inc., BioStrategies LC, Sanofi, AZAFAROS, Dorphan S.A., Idorsia Pharmaceuticals Ltd, SphinCS GmbH and Viatris Inc.

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• To visualize the global GM1 gangliosidosis treatment market segmentation based on type, treatment, end user and region as well as understand key commercial assets and players.
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The global GM1 gangliosidosis treatment market report would provide approximately 61 tables, 58 figures and 186 Pages.

Target Audience 2023

• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies