Sandhoff Disease (Jatzkewitz-Pilz Syndrome) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
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Sandhoff disease is an inherited lipid storage disorder that progressively destroys nerve cells (neurons) in the brain and spinal cord. It is caused by a deficiency of the enzyme beta-hexosaminidase. Symptoms include progressive nervous system deterioration, problems initiating and controlling muscles and movement, increased startle reaction to sound, early blindness, seizures, spasticity, and myoclonus. Treatment includes anticonvulsants to control seizures.
The Sandhoff Disease – Drugs in Development research report provides a comprehensive overview on the therapeutics under development for Sandhoff Disease, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Sandhoff Disease and features dormant and discontinued projects.
Key Targets in the Sandhoff Disease Pipeline Drugs Market
The key targets in the Sandhoff Disease pipeline drugs market are Ceramide Glucosyltransferase, Peroxisome Proliferator Activated Receptor Alpha, Protein O GlcNAcase, Beta-Hexosaminidase Subunit Alpha, Retinoic Acid Receptor RXR Alpha.
Sandhoff Disease Drugs Market, by Targets
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Key MoA in the Sandhoff Disease Pipeline Drugs Market
The key mechanisms of action in the Sandhoff Disease pipeline drugs market are Ceramide Glucosyltransferase Inhibitor, Peroxisome Proliferator Activated Receptor Alpha Agonist, Protein O GlcNAcase Inhibitor, Beta-Hexosaminidase Subunit Alpha Inhibitor, Retinoic Acid Receptor RXR Alpha Inhibitor.
Sandhoff Disease Pipeline Drugs Market, by MoA
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Sandhoff Disease Pipeline Drugs Market Segmentation by RoA
The key routes of administration in the Sandhoff Disease pipeline drugs market are Intrathecal, Oral, and Intracisternal.
Sandhoff Disease Pipeline Drugs Market Analysis, by RoA
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Key Molecule Types in the Sandhoff Disease Pipeline Drugs Market
The key molecule types in the Sandhoff Disease pipeline drugs market are Small Molecule, Gene Therapy, and Cell Therapy.
Sandhoff Disease Pipeline Drugs Market, by Molecule Type
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Major Companies in the Sandhoff Disease Pipeline Drugs Market
The major companies in the Sandhoff Disease pipeline drugs market are Azafaros BV, Genzyme Corp, IntraBio Ltd, Orphi Therapeutics Inc, and Polaryx Therapeutics Inc, Sio Gene Therapies Inc among others.
Sandhoff Disease Pipeline Drugs Market, by Major Companies
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Sandhoff Disease Pipeline Drugs Market Overview
Key Targets | Ceramide Glucosyltransferase, Peroxisome Proliferator Activated Receptor Alpha, Protein O GlcNAcase, Beta-Hexosaminidase Subunit Alpha, Retinoic Acid Receptor RXR Alpha |
Key Mechanisms of action | Ceramide Glucosyltransferase Inhibitor, Peroxisome Proliferator Activated Receptor Alpha Agonist, Protein O GlcNAcase Inhibitor, Beta-Hexosaminidase Subunit Alpha Inhibitor, Retinoic Acid Receptor RXR Alpha Inhibitor |
Key Routes of Administration | Intrathecal, Oral, and Intracisternal |
Key molecule types | Small Molecule, Gene Therapy, and Cell Therapy
|
Major companies | Azafaros BV, Genzyme Corp, IntraBio Ltd, Orphi Therapeutics Inc, and Polaryx Therapeutics Inc, Sio Gene Therapies Inc |
Scope
- The pipeline guide provides a snapshot of the global therapeutic landscape of Sandhoff Disease
- The pipeline guide reviews pipeline therapeutics for Sandhoff Disease by companies and universities/research institutes based on information derived from company and industry-specific sources.
- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
- The pipeline guide reviews key companies involved in Sandhoff Disease therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Sandhoff Disease therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA), and molecule type.
- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
- The pipeline guide reviews the latest news related to pipeline therapeutics for Sandhoff Disease
Reasons to Buy
- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with a potentially strong product portfolio and create effective counter-strategies to gain a competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Sandhoff Disease
- Classify potential new clients or partners in the target demographic.
- Develop tactical initiatives by understanding the focus areas of leading companies.
- Plan mergers and acquisitions meritoriously by identifying key players and their most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Sandhoff Disease pipeline depth and focus of Indication therapeutics.
- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.
- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from the pipeline.
Genzyme Corp
IntraBio Ltd
Orphi Therapeutics Inc
Polaryx Therapeutics Inc
Sio Gene Therapies Inc
Taysha Gene Therapies Inc
Table of Contents
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Frequently asked questions
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What are the key targets in the Sandhoff Disease pipeline drugs market?
The key targets in the Sandhoff Disease pipeline drugs market are Ceramide Glucosyltransferase, Peroxisome Proliferator Activated Receptor Alpha, Protein O GlcNAcase, Beta-Hexosaminidase Subunit Alpha, Retinoic Acid Receptor RXR Alpha
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What are the key mechanisms of action in the Sandhoff Disease pipeline drugs market?
The key mechanisms of action in the Sandhoff Disease pipeline drugs market are Ceramide Glucosyltransferase Inhibitor, Peroxisome Proliferator Activated Receptor Alpha Agonist, Protein O GlcNAcase Inhibitor, Beta-Hexosaminidase Subunit Alpha Inhibitor, Retinoic Acid Receptor RXR Alpha Inhibitor.
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What are the key routes of administration in the Sandhoff Disease pipeline drugs market?
The key routes of administration in the Sandhoff Disease pipeline drugs market are Intrathecal, Oral, and Intracisternal.
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What are the key molecule types in the Sandhoff Disease pipeline drugs market?
The key molecule types in the Sandhoff Disease pipeline drugs market are Small Molecule, Gene Therapy, and Cell Therapy
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What are the major companies in the Sandhoff Disease pipeline drugs market?
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