Table of Content


List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Globoid Cell Leukodystrophy (Krabbe Disease) - Overview
Globoid Cell Leukodystrophy (Krabbe Disease) - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Globoid Cell Leukodystrophy (Krabbe Disease) - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Globoid Cell Leukodystrophy (Krabbe Disease) - Companies Involved in Therapeutics Development
BioXcel Corp
Commence Bio Inc
GT Gain Therapeutics SA
Kyorin Pharmaceutical Co Ltd
Magenta Therapeutics Inc
Passage Bio Inc
Polaryx Therapeutics Inc
Vascumab LLC
Globoid Cell Leukodystrophy (Krabbe Disease) - Drug Profiles
Drug for Krabbe Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
DUOC-01 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Galactosylceramidase Replacement for Krabbe Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
gemfibrozil - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy for Globoid Cell Leukodystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ibudilast - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
MSC-2 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PBKR-03 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PLX-300 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Activate GALC for Krabbe Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
spanlecortemlocel - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Globoid Cell Leukodystrophy (Krabbe Disease) - Dormant Projects
Globoid Cell Leukodystrophy (Krabbe Disease) - Discontinued Products
Globoid Cell Leukodystrophy (Krabbe Disease) - Product Development Milestones
Featured News & Press Releases
May 12, 2020: Passage Bio announces presentation of data from animal models of Krabbe disease at the American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting
Feb 20, 2020: Magenta Therapeutics announces updated phase 2 data on MGTA-456 cell therapy, demonstrating continued durability in inherited metabolic disorders
Sep 04, 2019: Magenta Therapeutics announces FDA regenerative medicine advanced therapy (RMAT) desigtion granted to MGTA-456 for the treatment of Inherited Metabolic Disorders
May 30, 2019: Passage Bio announces third gene therapy development program in Krabbe Disease and supports million dreams fundraising gala
May 09, 2019: Magenta Therapeutics presents updated phase 2 clinical data on MGTA-456 cell therapy at American Academy of Neurology Annual Meeting
Feb 25, 2019: Magenta Therapeutics Presents Clinical and Preclinical Data on MGTA-456 Cell Therapy in Best Abstracts Sessions at Transplant and Cellular Therapy (TCT) Annual Meeting
Apr 05, 2018: Magenta Therapeutics Announces First Patient Transplanted with MGTA-456 in Phase 2 Study in Inherited Metabolic Disorders
Jan 18, 2016: FDA Grants Rare Pediatric Disease Desigtion to MediciNova’s MN-166 (ibudilast) for the Treatment of Krabbe Disease
Jun 03, 2015: MediciNova Announces FDA Granted Orphan Drug Desigtion to MN-166 (ibudilast) for Krabbe Disease
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer



List of Figures


Number of Products under Development for Globoid Cell Leukodystrophy (Krabbe Disease), H1 2020
Number of Products under Development by Companies, H1 2020
Number of Products under Development by Universities/Institutes, H1 2020
Number of Products by Top 10 Targets, H1 2020
Number of Products by Stage and Top 10 Targets, H1 2020
Number of Products by Top 10 Mechanism of Actions, H1 2020
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2020
Number of Products by Top 10 Routes of Administration, H1 2020
Number of Products by Stage and Top 10 Routes of Administration, H1 2020
Number of Products by Top 10 Molecule Types, H1 2020
Number of Products by Stage and Top 10 Molecule Types, H1 2020


List of Tables


Number of Products under Development for Globoid Cell Leukodystrophy (Krabbe Disease), 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
Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by BioXcel Corp, H1 2020
Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by Commence Bio Inc, H1 2020
Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by GT Gain Therapeutics SA, H1 2020
Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by Kyorin Pharmaceutical Co Ltd, H1 2020
Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by Magenta Therapeutics Inc, H1 2020
Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by Passage Bio Inc, H1 2020
Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by Polaryx Therapeutics Inc, H1 2020
Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by Vascumab LLC, H1 2020
Globoid Cell Leukodystrophy (Krabbe Disease) - Dormant Projects, H1 2020
Globoid Cell Leukodystrophy (Krabbe Disease) - Discontinued Products, H1 2020