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

Sandhoff Disease Drugs Market, by Targets

For more target insights, download a free report sample

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

Sandhoff Disease Pipeline Drugs Market, by MoA

To get more insights on key MoA, download a free sample report

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

Sandhoff Disease Pipeline Drugs Market Analysis, by RoA

To get more insights on key RoA, download a free sample report

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

Sandhoff Disease Pipeline Drugs Market, by Molecule Type

To get more insights on key molecule types, download a free sample report

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

Sandhoff Disease Pipeline Drugs Market, by Major Companies

For more company insights, download a free sample report

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.

Azafaros BV
Genzyme Corp
IntraBio Ltd
Orphi Therapeutics Inc
Polaryx Therapeutics Inc
Sio Gene Therapies Inc
Taysha Gene Therapies Inc

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Sandhoff Disease (Jatzkewitz-Pilz Syndrome) – Overview

Sandhoff Disease (Jatzkewitz-Pilz Syndrome) – Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Sandhoff Disease (Jatzkewitz-Pilz Syndrome) – Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Sandhoff Disease (Jatzkewitz-Pilz Syndrome) – Companies Involved in Therapeutics Development

Azafaros BV

Genzyme Corp

IntraBio Ltd

Orphi Therapeutics Inc

Polaryx Therapeutics Inc

Sio Gene Therapies Inc

Taysha Gene Therapies Inc

Sandhoff Disease (Jatzkewitz-Pilz Syndrome) – Drug Profiles

acetylleucine – Drug Profile

Product Description

Mechanism Of Action

History of Events

AZ-3102 – Drug Profile

Product Description

Mechanism Of Action

History of Events

cobnabexagene anvuparvovec – Drug Profile

Product Description

Mechanism Of Action

History of Events

DUOC-01 – Drug Profile

Product Description

Mechanism Of Action

gemfibrozil – Drug Profile

Product Description

Mechanism Of Action

History of Events

OT-1001 – Drug Profile

Product Description

Mechanism Of Action

History of Events

OT-1009 – Drug Profile

Product Description

Mechanism Of Action

History of Events

PLX-300 – Drug Profile

Product Description

Mechanism Of Action

History of Events

TGTX-101 – Drug Profile

Product Description

Mechanism Of Action

History of Events

venglustat malate – Drug Profile

Product Description

Mechanism Of Action

History of Events

Sandhoff Disease (Jatzkewitz-Pilz Syndrome) – Dormant Projects

Sandhoff Disease (Jatzkewitz-Pilz Syndrome) – Product Development Milestones

Featured News & Press Releases

Feb 14, 2022: Azafaros presents positive clinical and preclinical data supporting development of lead compound AZ-3102 in lysosomal storage disorders at the 18th Annual WORLDSymposium conference

Jan 18, 2022: Azafaros announces multiple presentations at 18th Annual WORLDSymposium

Aug 30, 2021: Polaryx Therapeutics announces FDA grants Orphan Drug Designation for PLX-200 in GM2 gangliosidoses

Mar 25, 2020: IntraBio Fast Track Designation for GM2 Gangliosidosis

Feb 11, 2019: ntraBio investigational new drug application approved by the FDA for the treatment of tay-sachs and sandhoff disease

Dec 12, 2018: IntraBio receives Tay-Sachs and Sandhoff Rare pediatric disease designation from the FDA

Nov 19, 2018: IntraBio receives Tay-Sachs and Sandhoff European Orphan Drug Designation

Oct 01, 2018: Intrabio receives FDA rare pediatric disease designation for ib1000 for GM2 Gangliosidoses

Mar 28, 2018: Intrabio receives FDA orphan drug designation for ib1000 for GM2 Gangliosidoses

Oct 30, 2017: Intrabio receives EMA orphan medicinal drug designation for IB1000s for GM2 Gangliosidoses

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

Table

List of Tables

Number of Products under Development for Sandhoff Disease (Jatzkewitz-Pilz Syndrome), 2022

Number of Products under Development by Companies, 2022

Number of Products under Development by Universities/Institutes, 2022

Products under Development by Companies, 2022

Products under Development by Universities/Institutes, 2022

Number of Products by Stage and Target, 2022

Number of Products by Stage and Mechanism of Action, 2022

Number of Products by Stage and Route of Administration, 2022

Number of Products by Stage and Molecule Type, 2022

Sandhoff Disease (Jatzkewitz-Pilz Syndrome) – Pipeline by Azafaros BV, 2022

Sandhoff Disease (Jatzkewitz-Pilz Syndrome) – Pipeline by Genzyme Corp, 2022

Sandhoff Disease (Jatzkewitz-Pilz Syndrome) – Pipeline by IntraBio Ltd, 2022

Sandhoff Disease (Jatzkewitz-Pilz Syndrome) – Pipeline by Orphi Therapeutics Inc, 2022

Sandhoff Disease (Jatzkewitz-Pilz Syndrome) – Pipeline by Polaryx Therapeutics Inc, 2022

Sandhoff Disease (Jatzkewitz-Pilz Syndrome) – Pipeline by Sio Gene Therapies Inc, 2022

Sandhoff Disease (Jatzkewitz-Pilz Syndrome) – Pipeline by Taysha Gene Therapies Inc, 2022

Sandhoff Disease (Jatzkewitz-Pilz Syndrome) – Dormant Projects, 2022

Figures

List of Figures

Number of Products under Development for Sandhoff Disease (Jatzkewitz-Pilz Syndrome), 2022

Number of Products under Development by Companies, 2022

Number of Products by Targets, 2022

Number of Products by Stage and Targets, 2022

Number of Products by Mechanism of Actions, 2022

Number of Products by Stage and Mechanism of Actions, 2022

Number of Products by Routes of Administration, 2022

Number of Products by Stage and Routes of Administration, 2022

Number of Products by Molecule Types, 2022

Number of Products by Stage and Molecule Types, 2022

Frequently asked questions

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