As Tay-Sachs Disease continues to impact populations worldwide and is associated with comorbidities such as diabetes, cardiovascular disease, and certain cancers, there is an increasing need for safer and more effective treatments. DelveInsight reports that the Tay-Sachs Disease pipeline includes 4+ pharmaceutical and biotech companies actively developing over 5 therapeutic candidates for the condition. These therapies are in various stages of clinical and preclinical development, highlighting significant innovation and dedication to tackling this critical public health challenge.
DelveInsight’s “Tay-Sachs Disease Pipeline Insight 2025” offers a comprehensive strategic analysis of the R&D landscape. The report details clinical trial progress, emerging therapies, mechanisms of action, competitive dynamics, and key company initiatives, serving as an essential resource for researchers, healthcare investors, and decision-makers seeking insights into the evolving Tay-Sachs Disease therapeutics market and its future advancements.
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Key Takeaways from the Tay-Sachs Disease Pipeline Report
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DelveInsight’s Tay-Sachs Disease pipeline report highlights a dynamic landscape with 4+ active companies developing over 5 therapeutic candidates for Tay-Sachs Disease.
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AXO-AAV-GM2, a gene therapy developed by Axovant Gene Therapies, employs adeno-associated viral vectors to deliver functional HEXA and HEXB genes to the central nervous system. In 2020, the U.S. Food and Drug Administration (FDA) approved the Investigational New Drug (IND) application for AXO-AAV-GM2, enabling the start of a registrational study for Tay-Sachs and Sandhoff diseases. The therapy has also received Orphan Drug and Rare Pediatric Disease designations from the FDA.
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Leading Tay-Sachs Disease companies such as Sanofi Genzyme, IntraBio, and others are actively pursuing new treatments to enhance the therapeutic landscape. Promising pipeline candidates include Venglustat, IB1001, and additional therapies in development.
Tay-Sachs Disease Overview:
Tay-Sachs disease is a serious inherited disorder that primarily affects the nervous system and results from a deficiency of the enzyme beta-hexosaminidase A, caused by mutations in the HEXA gene on chromosome 15. This enzyme is essential for breaking down GM2 ganglioside, a lipid, within lysosomes. In its absence, GM2 accumulates in nerve cells, causing progressive neurological damage. Tay-Sachs is inherited in an autosomal recessive pattern, meaning a child must inherit a defective gene from both parents to develop the disease, while carriers with only one mutated gene usually remain symptom-free. Because of its effects on lysosomal function and GM2 buildup, Tay-Sachs is classified as both a lysosomal storage disorder and a GM2-gangliosidosis.
The disease is divided into three main forms based on when symptoms appear: infantile, juvenile, and late-onset. The infantile form is the most severe, typically appearing around six months of age, with symptoms including loss of motor skills, exaggerated startle response, seizures, hearing and vision loss, and a characteristic “cherry-red” spot on the retina. Neurological decline progresses rapidly, and affected children rarely survive beyond early childhood. Juvenile Tay-Sachs emerges in early childhood or adolescence and is characterized by coordination difficulties, speech impairment, and cognitive decline. While its progression is slower than the infantile form, it remains a serious, often fatal condition by early adulthood.
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Tay-Sachs Disease Pipeline Analysis
The Tay-Sachs Disease pipeline insights report 2025, provides insights into:
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Provides comprehensive insights into key companies developing therapies in the Tay-Sachs Disease Market.
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Categorizes Tay-Sachs Disease therapeutic companies by development stage: early, mid, and late-stage.
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Highlights major companies involved in targeted therapy development, including both active and inactive (paused/discontinued) projects.
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Reviews emerging Tay-Sachs Disease drugs under development based on:
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Stage of development
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Tay-Sachs Disease Route of administration
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Target receptor
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Monotherapy vs. combination therapy
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Tay-Sachs Disease Mechanism of action
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Molecular type
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Offers detailed analysis of:
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Company-to-company and company-academia collaborations
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Tay-Sachs Disease Licensing agreements
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Funding and investment activities supporting future Tay-Sachs Disease market advancement.
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Unlock key insights into emerging Tay-Sachs Disease therapies and market strategies here: https://www.delveinsight.com/report-store/tay-sachs-disease-pipeline-insight?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr
Tay-Sachs Disease Emerging Drugs
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Venglustat: Sanofi Genzyme
Venglustat is an investigational oral therapy aimed at slowing disease progression by reducing the accumulation of glycosphingolipids (GSLs). It is still in clinical trials and has not received approval from any regulatory authority. The U.S. FDA has granted Venglustat Fast Track designation for Gaucher disease type 3, Fabry disease, and GM2 gangliosidosis. Furthermore, it has obtained Orphan Drug Designation in both the U.S. and Europe for GM2 gangliosidosis. Currently, Venglustat is in Phase III clinical development for Tay-Sachs disease.
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IB1001: IntraBio
IB1001 is a novel oral therapy developed by IntraBio, a U.S.-based biopharmaceutical company focused on therapies for rare and common neurological disorders. The drug, a modified amino acid (N-acetyl-L-leucine), employs a specialized transport mechanism to reach target tissues, including crossing the blood-brain barrier. This enables it to restore normal lysosomal and mitochondrial function, reduce neuroinflammation, and stabilize neuronal membrane potential and intracellular ion balance via calcium channels. IB1001 is currently being evaluated in Phase II clinical trials for Tay-Sachs disease.
Tay-Sachs Disease Pipeline Therapeutic Assessment
Tay-Sachs Disease Assessment by Product Type
• Mono
• Combination
• Mono/Combination
Tay-Sachs Disease By Stage
• 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
Tay-Sachs Disease Assessment by Route of Administration
• Oral
• Parenteral
• Intravenous
• Subcutaneous
• Topical
Tay-Sachs Disease Assessment by Molecule Type
• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy
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Table of Contents
1. Report Introduction
2. Executive Summary
3. Tay-Sachs Disease Current Treatment Patterns
4. Tay-Sachs Disease – DelveInsight’s Analytical Perspective
5. Therapeutic Assessment
6. Tay-Sachs Disease Late-Stage Products (Phase-III)
7. Tay-Sachs Disease Mid-Stage Products (Phase-II)
8. Early Stage Products (Phase-I)
9. Pre-clinical Products and Discovery Stage Products
10. Inactive Products
11. Dormant Products
12. Tay-Sachs Disease Discontinued Products
13. Tay-Sachs Disease Product Profiles
14. Tay-Sachs Disease Key Companies
15. Tay-Sachs Disease Key Products
16. Dormant and Discontinued Products
17. Tay-Sachs Disease Unmet Needs
18. Tay-Sachs Disease Future Perspectives
19. Tay-Sachs Disease Analyst Review
20. Appendix
21. Report Methodology
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