Tay Sachs Disease Pipeline Analysis Report 2025
Description
Tay-Sachs disease is a rare genetic central nervous system disorder. Studies reveal that approximately 1 in every 320,000 babies born is affected by Tay-Sachs disease in the United States. Consequently, there has been a significant emphasis on developing effective Tay Sachs disease therapeutics as major pharma companies are exploring the possibility of developing innovative treatments such as enzyme replacement therapies and gene therapies among others.
Report Coverage
The Tay Sachs Disease Pipeline Report by Expert Market Research gives comprehensive insights into Tay Sachs disease drugs currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for Tay Sachs disease. The Tay Sachs disease pipeline insight includes the analysis of over 100 pipeline drugs and 50+ companies . It will include an analysis based on efficacy and safety measure outcomes published for the trials including their adverse effects on patients suffering from Tay Sachs disease.
The detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration and ongoing Tay Sachs pipeline development activities are covered in the report.
Tay Sachs Disease Pipeline Outlook
Tay-Sachs disease is a rare neurodegenerative disorder in which an enzyme deficiency called hexosaminidase A results in excessive accumulation of lipids in the brain and nerve cells. This abnormal accumulation of lipids leads to progressive dysfunction of the central nervous system. The most common form of Tay-Sachs disease is infantile form which is present around 6 months of age and gradually progresses to loss of skills and seizure by the age of 2 and early death by the age of 5. There is also another form called the juvenile category of the disease which begins about the age of 5 years. The adult forms of Tay-Sachs disease begin in the late teens and beyond. All forms of disease are inherited in an autosomal recessive manner. The symptoms of the disease are based on the stage and type of disorder including muscle weakness, difficulty turning over, involuntary muscle twitching, seizures, vision and hearing loss, cherry-red spot-on eyes, and respiratory infections among others.
There is no cure for the disease and no treatment is currently proven to slow the progression of the disease. However, several treatments help in managing symptoms and preventing complications. The supporting treatment includes Tay Sachs disease therapeutic drugs, respiratory care, nutrition and hydration, physical therapy, speech, and language among others. Several prescription medications are available to reduce the symptoms of disease and prevent complications including anti-seizure medications and antibiotics for infection among others.
Companies are developing innovative Tay Sachs disease drug candidates. For instance, Sio Gene Therapies company is developing gene therapies for neurodegenerative diseases. The company announced that the first patient has been dosed with AXO-AAV-GM2 in a Phase I/II trial which is evaluating the efficacy of gene therapy for the treatment of GM2 gangliosidosis that causes Tay-Sachs diseases. Several Tay Sachs disease emerging drugs in the pipeline are being developed by major pharma companies which has impacted the drug pipeline for Tay Sachs disease.
Tay Sachs Disease – Drug Pipeline Therapeutic Assessment
This section of the Tay Sachs disease report covers the analysis of Tay Sachs Disease drugs based on various segmentations such as:
By Phase
EMR’s pipeline assessment report covers 50+ drug analyses based on phase.
EMR’s Tay Sachs disease therapeutic assessment covers 50+ drug analyses based on drug classes:
EMR’s Tay Sachs disease clinical assessment covers 50+ drug analyses based on the route of administration.
The Tay Sachs disease report assessment covers phase I, phase II, phase III, phase IV, and early phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR analysis , phase II covers a major share of the total clinical trials for Tay Sachs disease.
Tay Sachs Disease – Pipeline Assessment Segmentation, By Drug Class
The drug class categories covered under Tay Sachs disease pipeline analysis include recombinant fusion proteins, small molecules, monoclonal antibodies, peptides, polymers, and gene therapy. Gene therapy is a promising treatment for Tay-Sachs disease as it is caused by mutations in a single gene. The purpose of gene therapy is to deliver working copies of the HEXA and HEXB genes into cells. The report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for Tay Sachs Disease.
Tay Sachs Disease Clinical Trials Assessment – Competitive Dynamics
The EMR Tay Sachs disease report insights cover the profile of key companies involved in clinical trials and their drugs under development. Below is the list of a few players involved in Tay Sachs disease clinical trials:
This section covers the detailed analysis of each drug under multiple phases including phase I, phase II, phase III, phase IV, and emerging drugs for Tay Sachs disease. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of Tay Sachs disease drug candidates.
Biological: AXO-AAV-GM2
The trial is designed to evaluate the safety and dose-escalation of a bilateral thalamic and intracisternal/intrathecal infusion of AXO-AAV-GM2 in pediatric participants with Tay-Sachs disease. The trial is sponsored by Terence Flotte, and it is currently under phase I.
Drug: Venglustat GZ402671
It is in a phase III study, sponsored by Genzyme, a Sanofi Company. The study is being conducted to assess the efficacy and pharmacodynamics of daily oral dosing of Tay Sachs disease drug candidate venglustat when administered over a 104-week period.
Drug: IB1001
IntraBio Inc. is developing this drug, which is currently under phase II. The study is being conducted to assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Tay-Sachs disease.
Reasons To Buy This Report
The Tay Sachs Disease Drug Pipeline Analysis Report provides a strategic overview of the latest and future landscape of treatments for Tay Sachs disease. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into Tay Sachs disease collaborations, market trends, regulatory environments, and potential growth opportunities within Tay Sachs disease pipeline insights.
Key Questions Answered in the Tay Sachs Disease – Pipeline Assessment Report
Tay-Sachs Disease Market
Hereditary Angioedema Therapeutic Market
Hereditary Orotic Aciduria Treatment Market
Report Coverage
The Tay Sachs Disease Pipeline Report by Expert Market Research gives comprehensive insights into Tay Sachs disease drugs currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for Tay Sachs disease. The Tay Sachs disease pipeline insight includes the analysis of over 100 pipeline drugs and 50+ companies . It will include an analysis based on efficacy and safety measure outcomes published for the trials including their adverse effects on patients suffering from Tay Sachs disease.
The detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration and ongoing Tay Sachs pipeline development activities are covered in the report.
Tay Sachs Disease Pipeline Outlook
Tay-Sachs disease is a rare neurodegenerative disorder in which an enzyme deficiency called hexosaminidase A results in excessive accumulation of lipids in the brain and nerve cells. This abnormal accumulation of lipids leads to progressive dysfunction of the central nervous system. The most common form of Tay-Sachs disease is infantile form which is present around 6 months of age and gradually progresses to loss of skills and seizure by the age of 2 and early death by the age of 5. There is also another form called the juvenile category of the disease which begins about the age of 5 years. The adult forms of Tay-Sachs disease begin in the late teens and beyond. All forms of disease are inherited in an autosomal recessive manner. The symptoms of the disease are based on the stage and type of disorder including muscle weakness, difficulty turning over, involuntary muscle twitching, seizures, vision and hearing loss, cherry-red spot-on eyes, and respiratory infections among others.
There is no cure for the disease and no treatment is currently proven to slow the progression of the disease. However, several treatments help in managing symptoms and preventing complications. The supporting treatment includes Tay Sachs disease therapeutic drugs, respiratory care, nutrition and hydration, physical therapy, speech, and language among others. Several prescription medications are available to reduce the symptoms of disease and prevent complications including anti-seizure medications and antibiotics for infection among others.
Companies are developing innovative Tay Sachs disease drug candidates. For instance, Sio Gene Therapies company is developing gene therapies for neurodegenerative diseases. The company announced that the first patient has been dosed with AXO-AAV-GM2 in a Phase I/II trial which is evaluating the efficacy of gene therapy for the treatment of GM2 gangliosidosis that causes Tay-Sachs diseases. Several Tay Sachs disease emerging drugs in the pipeline are being developed by major pharma companies which has impacted the drug pipeline for Tay Sachs disease.
Tay Sachs Disease – Drug Pipeline Therapeutic Assessment
This section of the Tay Sachs disease report covers the analysis of Tay Sachs Disease drugs based on various segmentations such as:
By Phase
EMR’s pipeline assessment report covers 50+ drug analyses based on phase.
- Late-Stage Products (Phase 3 and Phase 4)
- Mid-Stage Products (Phase 2)
- Early-Stage Products (Phase I)
- Preclinical and Discovery Stage Products
EMR’s Tay Sachs disease therapeutic assessment covers 50+ drug analyses based on drug classes:
- Recombinant Fusion Proteins
- Small Molecule
- Monoclonal Antibody
- Peptide
- Polymer
- Gene Therapy
EMR’s Tay Sachs disease clinical assessment covers 50+ drug analyses based on the route of administration.
- Oral
- Parenteral
- Others
The Tay Sachs disease report assessment covers phase I, phase II, phase III, phase IV, and early phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR analysis , phase II covers a major share of the total clinical trials for Tay Sachs disease.
Tay Sachs Disease – Pipeline Assessment Segmentation, By Drug Class
The drug class categories covered under Tay Sachs disease pipeline analysis include recombinant fusion proteins, small molecules, monoclonal antibodies, peptides, polymers, and gene therapy. Gene therapy is a promising treatment for Tay-Sachs disease as it is caused by mutations in a single gene. The purpose of gene therapy is to deliver working copies of the HEXA and HEXB genes into cells. The report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for Tay Sachs Disease.
Tay Sachs Disease Clinical Trials Assessment – Competitive Dynamics
The EMR Tay Sachs disease report insights cover the profile of key companies involved in clinical trials and their drugs under development. Below is the list of a few players involved in Tay Sachs disease clinical trials:
- Terence Flotte
- Genzyme, a Sanofi Company
- IntraBio Inc.
- Azafaros A.G.
- Natera, Inc.
- Exsar Corporation
- Azafaros A.G.
- Idorsia Pharmaceuticals Ltd.
- Talaris Therapeutics Inc.
- Aldagen
- Others
This section covers the detailed analysis of each drug under multiple phases including phase I, phase II, phase III, phase IV, and emerging drugs for Tay Sachs disease. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of Tay Sachs disease drug candidates.
Biological: AXO-AAV-GM2
The trial is designed to evaluate the safety and dose-escalation of a bilateral thalamic and intracisternal/intrathecal infusion of AXO-AAV-GM2 in pediatric participants with Tay-Sachs disease. The trial is sponsored by Terence Flotte, and it is currently under phase I.
Drug: Venglustat GZ402671
It is in a phase III study, sponsored by Genzyme, a Sanofi Company. The study is being conducted to assess the efficacy and pharmacodynamics of daily oral dosing of Tay Sachs disease drug candidate venglustat when administered over a 104-week period.
Drug: IB1001
IntraBio Inc. is developing this drug, which is currently under phase II. The study is being conducted to assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Tay-Sachs disease.
Reasons To Buy This Report
The Tay Sachs Disease Drug Pipeline Analysis Report provides a strategic overview of the latest and future landscape of treatments for Tay Sachs disease. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into Tay Sachs disease collaborations, market trends, regulatory environments, and potential growth opportunities within Tay Sachs disease pipeline insights.
Key Questions Answered in the Tay Sachs Disease – Pipeline Assessment Report
- What is the current landscape of Tay Sachs disease pipeline drugs?
- Which companies/institutions are developing Tay Sachs disease emerging drugs?
- How many phase II drugs are currently present in Tay Sachs disease pipeline drugs?
- Which company is leading the Tay Sachs disease pipeline development activities?
- What is the current Tay Sachs disease therapeutic assessment?
- What are the opportunities and challenges present in the Tay Sachs disease drug pipeline landscape?
- What is the efficacy and safety profile of Tay Sachs disease pipeline drugs?
- Which companies/institutions are involved in Tay Sachs disease collaborations aimed at providing enhanced therapeutic alternatives for patients?
- What are the geographies covered for clinical trials in Tay Sachs disease?
Tay-Sachs Disease Market
Hereditary Angioedema Therapeutic Market
Hereditary Orotic Aciduria Treatment Market
Table of Contents
200 Pages
- 1 Preface
- 1.1 Introduction
- 1.2 Objectives of the Study
- 1.3 Research Methodology and Assumptions
- 2 Executive Summary
- 3 Overview of Tay Sachs
- 3.1 Signs and Symptoms
- 3.2 Causes
- 3.3 Risk Factors
- 3.4 Diagnosis
- 3.5 Treatment
- 4 Patient Profile
- 4.1 Patient Profile Overview
- 4.2 Patient Psychology and Emotional Impact Factors
- 4.3 Risk Assessment and Treatment Success Rate
- 5 Tay Sachs: Epidemiology Snapshot
- 5.1 Tay Sachs Incidence by Key Markets
- 5.2 Tay Sachs– Patients Seeking Treatment in Key Markets
- 6 Tay Sachs: Market Dynamics
- 6.1 Market Drivers and Constraints
- 6.2 SWOT Analysis
- 7 Tay Sachs: Key Facts Covered
- 7.1 Top Countries Contributing to Clinical Trials in Asia-Pacific
- 7.2 Top Countries Contributing to Clinical Trials in Europe
- 7.3 Top Countries Contributing to Clinical Trials in North America
- 7.4 Top Countries Contributing to Clinical Trials in Other Regions
- 8 Tay Sachs, Drug Pipeline Assessment
- 8.1 Assessment by Treatment Type
- 8.2 Assessment by Route of Administration
- 8.3 Assessment by Drug Class
- 9 EMR Drug Pipeline Comparative Analysis
- 9.1 List of Tay Sachs Pipeline Drugs
- 9.1.1 By Company
- 9.1.2 By Phase
- 9.1.3 By Indication
- 9.1.4 By Trial Status
- 9.1.5 By Funder Type
- 9.2 EMR Attribute Scoring Analysis of Pipeline Drugs (Top Drugs)
- 10 Tay Sachs Drug Pipeline - Late-Stage Products (Phase III and IV) (Top Drugs)
- 10.1 Comparative Analysis for Late-Stage Drugs
- 10.1.1 Study Type
- 10.1.2 Recruitment Status
- 10.1.3 Company
- 10.1.4 Funder Type
- 10.2 Product Level Analysis*
- 10.2.1 Venglustat GZ402671
- 10.2.1.1 Product Description
- 10.2.1.2 Trial ID
- 10.2.1.3 Sponsor Name
- 10.2.1.4 Study Type
- 10.2.1.5 Drug Class
- 10.2.1.6 Eligibility Criteria
- 10.2.1.7 Study Record Dates
- 10.2.1.7.1 First Submitted
- 10.2.1.7.2 First Posted
- 10.2.1.7.3 Last Update Posted
- 10.2.1.7.4 Last Verified
- 10.2.1.8 Indication
- 10.2.1.9 Study Design
- 10.2.1.10 Recruitment Status
- 10.2.1.11 Enrollment (Estimated)
- 10.2.1.12 Location Countries
- 10.2.1.13 Recent Results
- 10.2.2 Zavesca
- 10.2.3 ALD-101
- 10.2.4 Other Drug
- 11 Tay Sachs Drug Pipeline - Mid-Stage Products (Phase II) (Top Drugs)
- 11.1 Comparative Analysis for Mid-Stage Drugs
- 11.1.1 Study Type
- 11.1.2 Recruitment Status
- 11.1.3 Company
- 11.1.4 Funder Type
- 11.2 Product Level Analysis*
- 11.2.1 IB1001
- 11.2.1.1 Product Description
- 11.2.1.2 Trial ID
- 11.2.1.3 Sponsor Name
- 11.2.1.4 Study Type
- 11.2.1.5 Drug Class
- 11.2.1.6 Eligibility Criteria
- 11.2.1.7 Study Record Dates
- 11.2.1.7.1 First Submitted
- 11.2.1.7.2 First Posted
- 11.2.1.7.3 Last Update Posted
- 11.2.1.7.4 Last Verified
- 11.2.1.8 Indication
- 11.2.1.9 Study Design
- 11.2.1.10 Recruitment Status
- 11.2.1.11 Enrollment (Estimated)
- 11.2.1.12 Location Countries
- 11.2.1.13 Recent Results
- 11.2.2 TSHA-101
- 11.2.3 AZ-3102
- 11.2.4 Other Drugs
- 12 Tay Sachs Drug Pipeline - Early-Stage Products (Phase I) (Top Drugs)
- 12.1 Comparative Analysis for Early-Stage Drugs
- 12.1.1 Study Type
- 12.1.2 Recruitment Status
- 12.1.3 Company
- 12.1.4 Funder Type
- 12.2 Product Level Analysis*
- 12.2.1 Pyrimethamine
- 12.2.1.1 Product Description
- 12.2.1.2 Trial ID
- 12.2.1.3 Sponsor Name
- 12.2.1.4 Study Type
- 12.2.1.5 Drug Class
- 12.2.1.6 Eligibility Criteria
- 12.2.1.7 Study Record Dates
- 12.2.1.7.1 First Submitted
- 12.2.1.7.2 First Posted
- 12.2.1.7.3 Last Update Posted
- 12.2.1.7.4 Last Verified
- 12.2.1.8 Indication
- 12.2.1.9 Study Design
- 12.2.1.10 Recruitment Status
- 12.2.1.11 Enrollment (Estimated)
- 12.2.1.12 Location Countries
- 12.2.2 ALD-601
- 12.2.3 Other Drugs
- 13 Tay Sachs Drug Pipeline - Preclinical and Discovery Stage Products (Top Drugs)
- 13.1 Comparative Analysis for Preclinical and Discovery Stage Drugs
- 13.1.1 Study Type
- 13.1.2 Recruitment Status
- 13.1.3 Company
- 13.1.4 Funder Type
- 13.2 Product Level Analysis*
- 13.2.1 Drug 1
- 13.2.1.1 Product Description
- 13.2.1.2 Trial ID
- 13.2.1.3 Sponsor Name
- 13.2.1.4 Study Type
- 13.2.1.5 Drug Class
- 13.2.1.6 Eligibility Criteria
- 13.2.1.7 Study Record Dates
- 13.2.1.7.1 First Submitted
- 13.2.1.7.2 First Posted
- 13.2.1.7.3 Last Update Posted
- 13.2.1.7.4 Last Verified
- 13.2.1.8 Indication
- 13.2.1.9 Study Design
- 13.2.1.10 Recruitment Status
- 13.2.1.11 Enrollment (Estimated)
- 13.2.1.12 Location Countries
- 13.2.2 Drug 2
- 13.2.3 Other Drugs
- 14 Tay Sachs, Key Drug Pipeline Companies
- 14.1 Genzyme, a Sanofi Company
- 14.1.1 Company Snapshot
- 14.1.2 Pipeline Product Portfolio
- 14.1.3 Financial Analysis
- 14.1.4 Recent News and Developments
- 14.2 Terence Flotte
- 14.2.1 Company Snapshot
- 14.2.2 Pipeline Product Portfolio
- 14.2.3 Financial Analysis
- 14.2.4 Recent News and Developments
- 14.3 Azafaros A.G.
- 14.3.1 Company Snapshot
- 14.3.2 Pipeline Product Portfolio
- 14.3.3 Financial Analysis
- 14.3.4 Recent News and Developments
- 14.4 Natera, Inc.
- 14.4.1 Company Snapshot
- 14.4.2 Pipeline Product Portfolio
- 14.4.3 Financial Analysis
- 14.4.4 Recent News and Developments
- 14.5 Exsar Corporation
- 14.5.1 Company Snapshot
- 14.5.2 Pipeline Product Portfolio
- 14.5.3 Financial Analysis
- 14.5.4 Recent News and Developments
- 14.6 Azafaros A.G.
- 14.6.1 Company Snapshot
- 14.6.2 Pipeline Product Portfolio
- 14.6.3 Financial Analysis
- 14.6.4 Recent News and Developments
- 14.7 Idorsia Pharmaceuticals Ltd.
- 14.7.1 Company Snapshot
- 14.7.2 Pipeline Product Portfolio
- 14.7.3 Financial Analysis
- 14.7.4 Recent News and Developments
- 14.8 Talaris Therapeutics Inc.
- 14.8.1 Company Snapshot
- 14.8.2 Pipeline Product Portfolio
- 14.8.3 Financial Analysis
- 14.8.4 Recent News and Developments
- 14.9 Aldagen
- 14.9.1 Company Snapshot
- 14.9.2 Pipeline Product Portfolio
- 14.9.3 Financial Analysis
- 14.9.4 Recent News and Developments
- 15 Regulatory Framework for Drug Approval, By Region
- 16 Terminated or Suspended Pipeline Products
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