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
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
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
TGTX-101 - 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 03, 2021: Sio Gene Therapies announces first patient dosed in clinical trial of AXO-AAV-GM2 in patients with Tay-Sachs and Sandhoff Disease (GM2 Gangliosidosis)
Nov 09, 2020: Axovant Gene Therapies announces FDA clearance of IND for AXO-AAV-GM2 gene therapy in Tay-Sachs and Sandhoff Diseases
Oct 13, 2020: Axovant Gene Therapies receives Rare Pediatric Disease Desigtion for AXO-AAV-GM2 for Tay-Sachs and Sandhoff disease
Mar 25, 2020: IntraBio Fast Track Desigtion for GM2 Gangliosidosis
Jan 13, 2020: Axovant provides update on gene therapy pipeline program AXO-AAV-GM2
Feb 21, 2019: Axovant to provide update on drug candidate AXO-AAV-GM2 at its first R&D Day March 2019
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

List of Figures
Number of Products under Development for Sandhoff Disease (Jatzkewitz-Pilz Syndrome), 2021
Number of Products under Development by Companies, 2021
Number of Products by Targets, 2021
Number of Products by Stage and Targets, 2021
Number of Products by Mechanism of Actions, 2021
Number of Products by Stage and Mechanism of Actions, 2021
Number of Products by Routes of Administration, 2021
Number of Products by Stage and Top 10 Routes of Administration, 2021
Number of Products by Molecule Types, 2021
Number of Products by Stage and Molecule Types, 2021

List of Tables

List of Tables
Number of Products under Development for Sandhoff Disease (Jatzkewitz-Pilz Syndrome), 2021
Number of Products under Development by Companies, 2021
Number of Products under Development by Universities/Institutes, 2021
Products under Development by Companies, 2021
Products under Development by Universities/Institutes, 2021
Number of Products by Stage and Target, 2021
Number of Products by Stage and Mechanism of Action, 2021
Number of Products by Stage and Route of Administration, 2021
Number of Products by Stage and Molecule Type, 2021
Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Pipeline by Genzyme Corp, 2021
Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Pipeline by IntraBio Ltd, 2021
Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Pipeline by Orphi Therapeutics Inc, 2021
Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Pipeline by Polaryx Therapeutics Inc, 2021
Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Pipeline by Sio Gene Therapies Inc, 2021
Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Pipeline by Taysha Gene Therapies Inc, 2021
Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Dormant Projects, 2021