Table of Content


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
Axovant Gene Therapies Ltd
Genzyme Corp
IntraBio Ltd
Orphi Therapeutics Inc
Polaryx Therapeutics Inc
Recursion Pharmaceuticals Inc
Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Drug Profiles
acetylleucine - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AXO-AAV-GM2 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
DUOC-01 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
gemfibrozil - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
OT-1001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
OT-1009 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PLX-300 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecule for Sandhoff disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
venglustat malate - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Dormant Projects
Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Product Development Milestones
Featured News & Press Releases
Feb 11, 2019: ntraBio investigatiol 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 desigtion from the FDA
Nov 19, 2018: IntraBio receives Tay-Sachs and Sandhoff European Orphan Drug Desigtion
Oct 01, 2018: Intrabio receives FDA rare pediatric disease desigtion for ib1000 for GM2 Gangliosidoses
Mar 28, 2018: Intrabio receives FDA orphan drug desigtion for ib1000 for GM2 Gangliosidoses
Oct 30, 2017: Intrabio receives EMA orphan medicil drug desigtion for IB1000s for GM2 Gangliosidoses
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer



List of Figures


Number of Products under Development for Sandhoff Disease (Jatzkewitz-Pilz Syndrome), H1 2020
Number of Products under Development by Companies, H1 2020
Number of Products by Targets, H1 2020
Number of Products by Stage and Targets, H1 2020
Number of Products by Mechanism of Actions, H1 2020
Number of Products by Stage and Mechanism of Actions, H1 2020
Number of Products by Routes of Administration, H1 2020
Number of Products by Stage and Routes of Administration, H1 2020
Number of Products by Molecule Types, H1 2020
Number of Products by Stage and Molecule Types, H1 2020


List of Tables


Number of Products under Development for Sandhoff Disease (Jatzkewitz-Pilz Syndrome), H1 2020
Number of Products under Development by Companies, H1 2020
Number of Products under Development by Universities/Institutes, H1 2020
Products under Development by Companies, H1 2020
Products under Development by Universities/Institutes, H1 2020
Number of Products by Stage and Target, H1 2020
Number of Products by Stage and Mechanism of Action, H1 2020
Number of Products by Stage and Route of Administration, H1 2020
Number of Products by Stage and Molecule Type, H1 2020
Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Pipeline by Axovant Gene Therapies Ltd, H1 2020
Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Pipeline by Genzyme Corp, H1 2020
Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Pipeline by IntraBio Ltd, H1 2020
Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Pipeline by Orphi Therapeutics Inc, H1 2020
Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Pipeline by Polaryx Therapeutics Inc, H1 2020
Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Pipeline by Recursion Pharmaceuticals Inc, H1 2020
Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Dormant Projects, H1 2020