DelveInsight’s, “Sandhoff Disease Pipeline Insight 2025” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Sandhoff Disease pipeline landscape. It covers the Sandhoff Disease pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Sandhoff Disease pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive Sandhoff Disease pipeline products in this space.

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Key Takeaways from the Sandhoff Disease Pipeline Report

• DelveInsight’s Sandhoff Disease Pipeline report depicts a robust space with 5+ active players working to develop 5+ pipeline therapies for Sandhoff Disease treatment.
• The leading Sandhoff Disease Companies such as IntraBio, Sanofi, Polaryx Therapeutics, Azafaros and others.
• Promising Sandhoff Disease Pipeline Therapies such as IB1001, Zavesca (Miglustat), TSHA-101, Miglustat and others.

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Sandhoff Disease Emerging Drugs Profile

• IB1001: IntraBio

IB1001, N-acetyl-L-leucine, is an orally administered modified amino acid. In vivo studies have identified N-acetyl-L-leucine to be the active isomer of N-acetyl-DL-leucine and can restore neuronal function and protect against/delay disease progression in multiple neurological circuits of the brain. The mechanism of N-acetyl-L-leucine is known to be multi-modal, including altered glucose and antioxidant metabolism, reduced lysosomal storage, and the reduction of neuroinflammation in the cerebellum, leading to the attenuation of cell death. The company is conducting a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease).

• Venglustat: Sanofi

Venglustat is a novel, oral investigational therapy that has the potential to slow the progression of certain diseases by inhibiting abnormal glycosphingolipid (GSL) accumulation. GSLs are cellular building blocks whose abnormal accumulation is implicated in several rare diseases, responsible for both cell dysfunction and disease progression.Currently, the drug is in Phase III stage of Clinical trial evaluation for the treatment of Sandhoff Disease.

The Sandhoff Disease Pipeline Report Provides Insights into

• The report provides detailed insights about companies that are developing therapies for the treatment of Sandhoff Disease with aggregate therapies developed by each company for the same.
• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Sandhoff Disease Treatment.
• Sandhoff Disease Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Sandhoff Disease Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Sandhoff Disease market

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Sandhoff Disease Companies

IntraBio, Sanofi, Polaryx Therapeutics, Azafaros and others.

Sandhoff disease Pipeline Report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Parenteral
• Intravenous
• Subcutaneous
• Topical

Sandhoff Disease Products have been categorized under various Molecule types such as

• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

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Scope of the Sandhoff Disease Pipeline Report

• Coverage- Global
• Sandhoff Disease Companies- IntraBio, Sanofi, Polaryx Therapeutics, Azafaros and others.
• Sandhoff Disease Pipeline Therapies- IB1001, Zavesca (Miglustat), TSHA-101, Miglustat and others.
• Sandhoff Disease Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Sandhoff Disease Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

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Table of Contents

1. Introduction
2. Executive Summary
3. Sandhoff disease: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Sandhoff disease – DelveInsight’s Analytical Perspective
7. Late Stage Products (Phase III)
8. Venglustat: Sanofi
9. Drug profiles in the detailed report…..
10. Mid-Stage Products (Phase II)
11. IB1001: IntraBio
12. Drug profiles in the detailed report…..
13. Early Stage Products (Phase I)
14. Drug Name: Company Name
15. Drug profiles in the detailed report…..
16. Preclinical and Discovery Stage Products
17. Drug Name: Company Name
18. Drug profiles in the detailed report…..
19. Inactive Products
20. Sandhoff disease Key Companies
21. Sandhoff disease Key Products
22. Sandhoff disease- Unmet Needs
23. Sandhoff disease- Market Drivers and Barriers
24. Sandhoff disease- Future Perspectives and Conclusion
25. Sandhoff disease Analyst Views
26. Sandhoff disease Key Companies
27. Appendix

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