GM2 Gangliosidosis - Pipeline Insight, 2024

This “GM2 Gangliosidosis - Pipeline Insight, 2024,” report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in GM2 Gangliosidosis pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

GM2 Gangliosidosis Understanding

GM2 Gangliosidosis: Overview

GM2 gangliosidosis is a rare genetic disorder that progressively destroys nerve cells in the brain and spinal cord. The most common form of the disease typically presents in infancy, but various other forms can present in childhood, adolescence, or even adulthood. GM2 gangliosidosis is sometimes called Tay-Sachs disease or HexA deficiency. The disorder is one of about 50 diseases that are classified as lysosomal storage disorders (LSD), where a genetic variation disrupts the normal activity of lysosomes in human cells. GM2 gangliosidosis is most often caused by a mutation in the HEXA or the GM2A gene, which prevents an enzyme called beta-hexosaminidase A, from properly breaking down large molecules inside the body’s cells. This genetic condition is inherited in an autosomal recessive pattern, which means that an affected child has received one defective copy of the gene from each of their parents. The severity of symptoms and the time at which they first present can vary in GM2 gangliosidosis. Children with this disease typically show no signs at the time of birth and develop normally during the first few months of life. There are currently no approved therapies to reverse the effects of GM2 gangliosidosis. Current approaches involve interdisciplinary collaboration to provide targeted management for specific symptoms.

GM2 Gangliosidosis - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the GM2 Gangliosidosis pipeline landscape is provided which includes the disease overview and GM2 Gangliosidosis treatment guidelines. The assessment part of the report embraces, in depth GM2 Gangliosidosis commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, GM2 Gangliosidosis collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

• A better understanding of disease pathogenesis contributing to the development of novel therapeutics for GM2 Gangliosidosis.
• In the coming years, the GM2 Gangliosidosis market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.

The companies and academics that are working to assess challenges and seek opportunities that could influence GM2 Gangliosidosis R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.

• A detailed portfolio of major pharma players who are involved in fueling the GM2 Gangliosidosis treatment market. Several potential therapies for GM2 Gangliosidosis are under investigation. With the expected launch of these emerging therapies, it is expected that there will be a significant impact on the GM2 Gangliosidosis market size in the coming years.
• This in-depth analysis of the pipeline assets (in early-stage, mid-stage and late stage of development for the treatment of GM2 Gangliosidosis) includes therapeutic assessment and comparative analysis. This will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.

GM2 Gangliosidosis Emerging Drugs Chapters

This segment of the GM2 Gangliosidosis report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

GM2 Gangliosidosis Emerging Drugs

Venglustat: Sanofi Venglustat is an oral inhibitor of an enzyme called glucosylceramide synthase (GCS). Enzymes modify specific molecules called substrates. GCS turns its substrate, ceramide, into glucosylceramide (GL-1) during lipid metabolism, a series of biochemical reactions that degrade and generate lipids. GL-1 acts as a substrate to other enzymes and is turned into globosides, a subclass of lipids where Gb3 belongs. When venglustat inhibits GCS, it prevents the synthesis of GL-1, thereby reducing the substrate of the following reactions that lead to the formation of Gb3 and its accumulation in the absence of a-galactosidase A. That’s why venglustat is called a substrate reduction therapy. Currently, it is under Phase III stage of clinical trial evaluation to treat GM2gangliosidoses.

AXO-AAV-GM2: Sio Gene Therapies AXO-AAV-GM2 is an investigational gene therapy for GM2 gangliosidosis (also known as Tay-Sachs and Sandhoff diseases), a set of rare and fatal pediatric neurodegenerative genetic disorders caused by defects in the HEXA (leading to Tay-Sachs disease) or HEXB (leading to Sandhoff disease) genes that encode the two subunits of the ß-hexosaminidase A (HexA) enzyme. These genetic defects lead to progressive neurodegeneration and shortened life expectancy. AXO-AAV-GM2 aims to restore HexA function by introducing a functional copy of the HEXA and HEXB genes via delivery of two co-administered AAVrh8 vectors. AXO-AAV-GM2 has received Orphan Drug Designation, Rare Pediatric Disease Designation and Fast Track Designation from the Food and Drug Administration and is the only gene therapy in clinical development for all pediatric forms of GM2 gangliosidosis. In 2018, Sio licensed exclusive worldwide rights from UMass Chan Medical School for the development and commercialization of gene therapy programs for GM1 gangliosidosis and GM2 gangliosidosis, including Tay-Sachs and Sandhoffdiseases.

GM2 Gangliosidosis: Therapeutic Assessment

This segment of the report provides insights about the different GM2 Gangliosidosis drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in GM2 Gangliosidosis

There are approx. 8+ key companies which are developing the therapies for GM2 Gangliosidosis. The companies which have their GM2 Gangliosidosis drug candidates in the most advanced stage, i.e. phase III include, Sanofi.

Phases

This report covers around 8+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

GM2 Gangliosidosis pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Inhalation
• Inhalation/Intravenous/Oral
• Intranasal
• Intravenous
• Intravenous/ Subcutaneous
• NA
• Oral
• Oral/intranasal/subcutaneous
• Parenteral
• Subcutaneous

Molecule Type

Products have been categorized under various Molecule types such as

• Antibody
• Antisense oligonucleotides
• Immunotherapy
• Monoclonal antibody
• Peptides
• Protein
• Recombinant protein
• Small molecule
• Stem Cell
• Vaccine

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

GM2 Gangliosidosis: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses GM2 Gangliosidosis therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging GM2 Gangliosidosis drugs.

GM2 Gangliosidosis Report Insights

• GM2 Gangliosidosis Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

GM2 Gangliosidosis Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing GM2 Gangliosidosis drugs?
• How many GM2 Gangliosidosis drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of GM2 Gangliosidosis?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the GM2 Gangliosidosis therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for GM2 Gangliosidosis and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Sanofi
• Sio Gene Therapies
• IntraBio Inc.
• Taysha Gene Therapies
• JCR Pharmaceuticals
• Azafaros B.V.
• Allievex Corp.
• Recursion
• Polaryx

Key Products

• Venglustat
• AXO-AAV-GM2
• IB1001
• TSHA-101
• JR-479
• AZ-3102
• AX 451
• PLX-300