Tay-Sachs Disease Pipeline 2025: Groundbreaking Clinical Advancements by 4+ Global Leaders - DelveInsight | Featuring Sanofi Genzyme, IntraBio

With Tay-Sachs Disease reaching epidemic proportions globally and contributing significantly to comorbid conditions such as diabetes, cardiovascular disease, and certain cancers, there is a growing demand for safer, more effective treatment options. According to DelveInsight, the Tay-Sachs Disease pipeline comprises 4+ pharmaceutical and biotech companies actively developing 5+ therapeutic candidates targeting Tay-Sachs Disease. These therapies span various stages of clinical and non-clinical development, underscoring the intense innovation and commitment to addressing one of the most pressing public health challenges of our time.
DelveInsight's "Tay-Sachs Disease Pipeline Insight 2025" report provides a detailed and strategic evaluation of the ongoing R&D landscape. It covers clinical trial progression, emerging therapies, mechanisms of action, competitive positioning, and key company initiatives. The report serves as a crucial resource for stakeholders-including researchers, healthcare investors, and decision-makers, seeking insights into the evolving Tay-Sachs Disease Therapeutics Market and the breakthroughs shaping its future trajectory.

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Key Takeaways from the Tay-Sachs Disease Pipeline Report

DelveInsight's Tay-Sachs Disease pipeline report depicts a robust space with 4+ active players working to develop 5+ pipeline therapies for Tay-Sachs Disease treatment.
Developed by Axovant Gene Therapies, this gene therapy utilizes 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) cleared the Investigational New Drug (IND) application for AXO-AAV-GM2, allowing the initiation of a registrational study for Tay-Sachs and Sandhoff diseases. The therapy has received Orphan Drug and Rare Pediatric Disease Designations from the FDA .
Key Tay-Sachs Disease companies such as Sanofi Genzyme, IntraBio, and others are evaluating new drugs for Tay-Sachs Disease to improve the treatment landscape.
Promising Tay-Sachs Disease pipeline therapies in various stages of development include Venglustat, IB1001, and others.

Tay-Sachs Disease Overview:

Tay-Sachs disease is a severe inherited disorder that primarily impacts the nervous system, caused by a deficiency in the enzyme beta-hexosaminidase A due to mutations in the *HEXA* gene on chromosome 15. This enzyme plays a critical role in breaking down GM2 ganglioside, a type of lipid, within lysosomes. Without adequate levels of beta-hexosaminidase A, GM2 builds up in nerve cells, leading to progressive neurological damage. Tay-Sachs is inherited in an autosomal recessive manner, meaning a child must receive a faulty gene from both parents to develop the disease. Carriers-those with only one mutated gene-typically show no symptoms. Due to its impact on lysosomal function and GM2 accumulation, Tay-Sachs is classified as both a lysosomal storage disorder and a GM2-gangliosidosis.

The disease is categorized into three main types based on the age of symptom onset: infantile, juvenile, and late-onset. The infantile form is the most severe, with symptoms emerging around six months of age. These include loss of motor skills, heightened startle reflex, seizures, hearing and vision loss, and a characteristic "cherry-red" spot on the retina. Neurological deterioration occurs rapidly, and affected children usually do not survive past early childhood. Juvenile Tay-Sachs appears later, during early childhood or adolescence, and is marked by issues with coordination, speech, and cognitive function. Though slower in progression than the infantile form, it remains a serious condition and is often fatal by early adulthood.

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Tay-Sachs Disease Pipeline Analysis
The Tay-Sachs Disease pipeline insights report 2025, provides insights into:
Provides comprehensive insights into key companies developing therapies in the Tay-Sachs Disease Market.

Categorizes Tay-Sachs Disease therapeutic companies by development stage: early, mid, and late-stage.

Highlights major companies involved in targeted therapy development, including both active and inactive (paused/discontinued) projects.

Reviews emerging Tay-Sachs Disease drugs under development based on:

Stage of development

Tay-Sachs Disease Route of administration

Target receptor

Monotherapy vs. combination therapy

Tay-Sachs Disease Mechanism of action

Molecular type

Offers detailed analysis of:

Company-to-company and company-academia collaborations

Tay-Sachs Disease Licensing agreements

Funding and investment activities supporting future Tay-Sachs Disease market advancement.

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=openpr&utm_medium=pressrelease&utm_campaign=kpr

Tay-Sachs Disease Emerging Drugs

Venglustat: Sanofi Genzyme

Venglustat is an experimental oral therapy designed to slow disease progression by targeting and reducing the buildup of glycosphingolipids (GSLs). It is currently in clinical trials and has not yet been approved by any regulatory agency. The U.S. FDA has granted Venglustat Fast Track designation for Gaucher disease type 3, Fabry disease, and GM2 gangliosidosis. Additionally, it has received Orphan Drug Designation in both the U.S. and Europe for treating GM2 gangliosidosis. Venglustat is currently in Phase III clinical development for Tay-Sachs disease.

IB1001: IntraBio
IB1001 is an innovative oral therapy developed by IntraBio, a U.S.-based biopharmaceutical company specializing in treatments for rare and common neurological disorders. The drug is a modified amino acid (N-acetyl-L-leucine) that utilizes a unique transport system to reach target tissues, including crossing the blood-brain barrier. This action helps restore normal lysosomal and mitochondrial function, which in turn reduces neuroinflammation and stabilizes neuronal membrane potential and intracellular ion balance through calcium channels. IB1001 is currently in Phase II clinical trials for the treatment of 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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Contact Info:
Kritika Rehani
Assistant Manager, Marketing & Branding
krehani@delveinsight.com
info@delveinsight.com
+14699457679

About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

This release was published on openPR.