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

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

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

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