openPR Logo
Press release

Sandhoff Disease Pipeline: 5+ Biotech Firms Driving Rare Disease Innovation Through Gene Therapy and Enzyme Replacement | DelveInsight

04-30-2025 04:05 PM CET | Health & Medicine

Press release from: DelveInsight

Sandhoff Disease Pipeline

Sandhoff Disease Pipeline

Sandhoff Disease, a rare and fatal lysosomal storage disorder, is seeing progress through the efforts of over 5 companies, including IntraBio, Sanofi, Polaryx Therapeutics, and Azafaros. The Sandhoff pipeline primarily focuses on gene therapy, substrate reduction, and enzyme replacement therapies aimed at correcting the underlying enzyme deficiency. Despite its ultra-rare prevalence, advancements in AAV-based vectors and CNS-targeted delivery systems are driving a new era of hope for patients and caregivers dealing with this devastating disorder.

DelveInsight's "Sandhoff Disease Pipeline Insight, 2025" comprehensively analyzes the current clinical landscape and growth prospects in the Sandhoff disease market. The report covers disease insights, treatment guidelines, and a detailed pipeline assessment from preclinical to marketed stages. It includes drug mechanisms, clinical studies, regulatory progress, and key developments such as collaborations, mergers, funding, and designations.

For emerging Sandhoff disease drugs, the Sandhoff disease pipeline analysis report provides a 360° view of the therapeutic landscape by development stage, product type, route of administration, molecule type, and mechanism of action (MOA). The pipeline research covers business opportunities, challenges, future partnerships, strong competitors, and growth strategies.

Explore the full pipeline analysis for Sandhoff disease and uncover key opportunities @ https://www.delveinsight.com/report-store/sandhoff-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Key Takeaways from the Sandhoff Disease Pipeline Report
• DelveInsight's Sandhoff disease pipeline analysis depicts a strong landscape with 5+ active players developing 5+ pipeline drugs for Sandhoff Disease treatment.
• The leading Sandhoff disease companies include IntraBio, Sanofi, Polaryx Therapeutics, Azafaros, and others are evaluating their lead assets to improve the Sandhoff disease treatment landscape.
• Key Sandhoff disease pipeline therapies in various stages of development include IB1001, Venglustat, PLX 300, PLX-200, AZ 3102, and others.
• ​In November 2024, McMaster University researchers published a study in Human Molecular Genetics identifying 4-phenylbutyric acid (4-PBA), an FDA-approved drug, as a potential treatment for Sandhoff and Tay-Sachs diseases. The study found that 4-PBA alleviated endoplasmic reticulum stress in spinal cord neurons of a mouse model, leading to improved motor function and extended lifespan. This repurposed drug could offer a new avenue for managing these rare genetic disorders, pending further clinical trials to determine optimal human dosing. ​
• ​In March 2021, UMass Chan Medical School used a novel neurosurgical technique to deliver gene therapy for Tay-Sachs and Sandhoff diseases. The procedure involved convection-enhanced delivery (CED) of AXO-AAV-GM2 directly into the brain and spinal cord, overcoming the blood-brain barrier. This approach, tailored for infants, marked a significant advancement in treating these neurodegenerative disorders.
• ​In November 2020, Axovant Gene Therapies announced that the FDA lifted its clinical hold and cleared the IND application to begin a registrational study of AXO-AAV-GM2 gene therapy for treating Tay-Sachs and Sandhoff diseases.

Request a sample and discover the recent breakthroughs happening in the Sandhoff disease pipeline landscape @ https://www.delveinsight.com/report-store/sandhoff-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Sandhoff Disease Overview
Sandhoff disease is a rare and severe lysosomal storage disorder caused by harmful mutations in the HEXB gene, which result in a deficiency of the enzymes beta-hexosaminidase A and B. These enzymes are crucial for breaking down certain lipids within lysosomes, the recycling centers of cells. When they are deficient, lipids accumulate abnormally in nerve cells, leading to progressive damage of the brain and spinal cord (central nervous system). This neurodegeneration impairs both cognitive and motor functions. Sandhoff disease closely resembles Tay-Sachs disease in its symptoms and underlying mechanisms. It is extremely rare, affecting approximately one in a million individuals worldwide.

Find out more about Sandhoff disease medication @ https://www.delveinsight.com/report-store/sandhoff-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Sandhoff Disease Treatment Analysis: Drug Profile
IB1001: IntraBio
IB1001 (N-acetyl-L-leucine) is an orally administered modified amino acid that has shown potential in restoring neuronal function and slowing disease progression in neurological circuits. Its mechanism includes altering glucose and antioxidant metabolism, reducing lysosomal storage, and decreasing neuroinflammation in the cerebellum. IntraBio is conducting a Phase II study to assess the safety and efficacy of IB1001 for treating GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease).

Venglustat: Sanofi
Venglustat is a novel oral therapy designed to inhibit the accumulation of abnormal glycosphingolipids (GSLs), which contribute to cell dysfunction and disease progression. It is currently undergoing Phase III trials for the treatment of Sandhoff Disease.

Learn more about the novel and emerging Sandhoff disease pipeline therapies @ https://www.delveinsight.com/report-store/sandhoff-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Sandhoff Disease Therapeutics Assessment
By Product Type
• Mono
• Combination
• Mono/Combination.

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

By Route of Administration
• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

By Molecule Type
• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

Scope of the Sandhoff Disease Pipeline Report
• Coverage: Global
• Key Sandhoff Disease Companies: IntraBio, Sanofi, Polaryx Therapeutics, Azafaros, and others.
• Key Sandhoff Disease Pipeline Therapies: IB1001, Venglustat, PLX 300, PLX-200, AZ 3102, and others.

Dive deep into rich insights for drugs used for Sandhoff disease treatment; visit @ https://www.delveinsight.com/report-store/sandhoff-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Table of Contents
1. Introduction
2. Executive Summary
3. Sandhoff Disease Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Sandhoff Disease Pipeline Therapeutics
6. Sandhoff Disease Pipeline: Late-Stage Products (Phase III)
7. Sandhoff Disease Pipeline: Mid-Stage Products (Phase II)
8. Sandhoff Disease Pipeline: Early Stage Products (Phase I)
9. Therapeutic Assessment
10. Inactive Products
11. Company-University Collaborations (Licensing/Partnering) Analysis
12. Key Companies
13. Key Products
14. Unmet Needs
15. Market Drivers and Barriers
16. Future Perspectives and Conclusion
17. Analyst Views
18. Appendix

Contact Us:
Jatin Vimal
jvimal@delveinsight.com
+14699457679
Healthcare Consulting
https://www.delveinsight.com/consulting-services

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. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform, PharmDelve.

This release was published on openPR.

Permanent link to this press release:

Copy
Please set a link in the press area of your homepage to this press release on openPR. openPR disclaims liability for any content contained in this release.

You can edit or delete your press release Sandhoff Disease Pipeline: 5+ Biotech Firms Driving Rare Disease Innovation Through Gene Therapy and Enzyme Replacement | DelveInsight here

News-ID: 3995182 • Views:

More Releases from DelveInsight

Sensorineural Hearing Loss Clinical Trials Analysis 2025: Emerging Regenerative Approaches, Gene Therapies, and Neuromodulation Aim to Restore Auditory Function | DelveInsight
Sensorineural Hearing Loss Clinical Trials Analysis 2025: Emerging Regenerative …
DelveInsight's "Sensorineural Hearing Loss Clinical Trials Analysis, 2025" describes a rapidly evolving pipeline targeting both restoration of inner-ear structures and symptomatic improvement. Ongoing programs span hair cell regeneration via small molecules and biologics, AAV- and non-viral gene-replacement or gene-editing strategies for monogenic deafness, cell-based therapies (supporting synaptic reconnection), and cochlear neuromodulation advancements to augment residual hearing. Trials increasingly pair novel delivery methods (e.g., intracochlear, round-window) with precision patient selection based
Cell and Gene Therapy in Parkinson's Disease Clinical Trials Analysis 2025: Disease-Modifying Strategies and Circuit-Level Restorations Aim to Slow Progression and Improve Motor/Non-motor Outcomes | DelveInsight
Cell and Gene Therapy in Parkinson's Disease Clinical Trials Analysis 2025: Dise …
DelveInsight's "Cell and Gene Therapy in Parkinson's Disease - Clinical Trials Analysis, 2025" highlights a diversified development landscape pursuing symptomatic relief and disease modification. Programs include dopamine-restorative cell implants, neuroprotective gene therapies targeting α-synuclein aggregation or neuroinflammation, and circuit-targeted gene delivery to rebalance basal ganglia networks. Trials incorporate advanced delivery (stereotactic infusion), standardized imaging and molecular biomarkers, and combination strategies pairing cell/gene therapy with neuromodulation. Pivotal and late-phase trials focus on
Staphylococcal Infections Clinical Trials Analysis 2025: New Antibacterials, Anti-virulence Agents, and Host-Directed Therapies Aim to Overcome Resistance and Improve Clinical Cure Rates | DelveInsight
Staphylococcal Infections Clinical Trials Analysis 2025: New Antibacterials, Ant …
DelveInsight's "Staphylococcal Infections Clinical Trials Analysis, 2025" outlines a focused effort to tackle antibiotic resistance and severe invasive disease. Active programs include novel small-molecule antibacterials with activity against MRSA, anti-virulence and quorum-sensing inhibitors, monoclonal antibodies targeting toxins and surface proteins, and host-directed immunotherapies to enhance clearance. Trials cover a range of indications from complicated skin and soft tissue infections to bacteremia and prosthetic-joint infections, with increasing attention on combination regimens
Cough in Idiopathic Pulmonary Fibrosis Clinical Trials Analysis, 2025: Targeted Anti-tussive Biologics, Neuromodulators, and Symptom-Focused Therapies Aim to Reduce Cough Burden and Improve Quality of Life | DelveInsight
Cough in Idiopathic Pulmonary Fibrosis Clinical Trials Analysis, 2025: Targeted …
DelveInsight's "Cough in Idiopathic Pulmonary Fibrosis Clinical Trials Analysis, 2025" captures an expanding set of symptomatic and mechanism-based approaches to address an under-treated, QoL-limiting symptom. Development includes neuromodulators that dampen hypersensitive cough reflexes, monoclonal antibodies against inflammatory mediators implicated in fibrotic cough, and novel inhaled agents to modify airway sensory signaling. Trials increasingly integrate digital cough monitoring, patient-reported cough-specific scales, and physiological cough reflex testing for objective and subjective assessment. Registrational

All 5 Releases


More Releases for Sandhoff

Sandhoff Disease Market to Reach USD 1.2 Billion by 2034
Sandhoff disease is a rare, inherited lysosomal storage disorder caused by mutations in the HEXB gene, leading to a deficiency in the beta-hexosaminidase A and B enzymes. This deficiency results in the accumulation of GM2 gangliosides within nerve cells, causing progressive neurodegeneration. Sandhoff disease presents in various forms-infantile, juvenile, and adult-onset-with the infantile type being the most severe and often fatal by early childhood. Download Full PDF Sample Copy of Market
Sandhoff Disease Pipeline Assessment Report 2025 | DelveInsight
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. Discover the latest drugs
Sandhoff Disease Market Size, Share, Trends, Growth 2024-2032
The Sandhoff disease market size was valued at USD 10.18 billion in 2023, driven growing interest in gene therapy as a potential curative approach across the major markets. The market size is anticipated to grow at a CAGR of 5.87% during the forecast period of 2024-2032 and attain a value of USD 17 billion by 2032. Sandhoff Disease:  Sandhoff disease is a rare, inherited genetic disorder characterized by the harmful accumulation of
Sandhoff Disease Treatment Market Analysis: Understanding Trends, Players, and P …
"Sandhoff Disease Treatment Market Is Growing At A Steady Cagr During Forecast Period". With thorough company profiles, recent developments, and other information, the Sandhoff Disease Treatment Market Report provides an in-depth analysis of major trends, motivating reasons, obstacles, segmentation, regulatory policies, and important players. The Sandhoff Disease Treatment Market Report offers a thorough synopsis of the product portfolio, including product development, planning, and positioning of products with emerging technologies, impending opportunities,
Sandhoff Disease Market: Analysis of Epidemiology, Industry Trends, Size, Share, …
IMARC Group has recently released a report titled "Sandhoff Disease Market : Analysis of Epidemiology, Industry Trends, Size, Share, and Future Forecast (2023-2033)" that presents a comprehensive assessment of the sandhoff disease market size . The report provides an extensive overview of the latest market trends, growth prospects, investment opportunities, and industry outlook, as well as an in-depth analysis of the disease landscape, market size, and growth trends. Furthermore, the
Sandhoff Disease Therapeutics Market Key Players Analysis 2021 to 2027 Covid-19 …
Market share, size, participants, growth and industry analysis are some of the prominent factors covered in this Sandhoff Disease Therapeutics market report. This comprehensive report starts with a goal to give information about market forecast, channel features, end-user market, key pricing structure and different geographies. Besides talking about this, it further mentions key regions, key companies along with their profiles and investment options available in the market. Sandhoff Disease Therapeutics