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
Bial - Portela & Ca SA
BioXcel Corp
Forge Biologics Inc
Gain Therapeutics Inc
Kyorin Pharmaceutical Co Ltd
M6P Therapeutics
Neurogene 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
FBX-101 - 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
Gene Therapy to Activate GALC for Globoid Cell Leukodystrophy (Krabbe Disease) - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ibudilast - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
M-031 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
M-033 - 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 Molecule to Inhibit Acid Ceramidase for Gaucher’s and Krabbe’s Diseases - 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
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
Sep 20, 2021: Forge Biologics announces regulatory updates from FDA and EMA, accelerating manufacturing and clinical trial momentum
Apr 05, 2021: Passage Bio receives European Commission Orphan Desigtion for PBKR03 for treatment of Krabbe Disease
Mar 08, 2021: Passage Bio annouced that U.S. Food and Drug Administration grants Fast Track Desigtion to its gene therapy candidate PBKR03
Feb 16, 2021: Forge Biologics Receives FDA Fast Track, Orphan Drug, and Rare Pediatric Disease Desigtions for FBX-101 Gene Therapy for Patients with Krabbe Disease
Feb 10, 2021: Polaryx Therapeutics receives both Rare Pediatric Disease and Orphan Drug Desigtions for the treatment of Krabbe Disease with PLX-300
Feb 08, 2021: FDA clears IND application for Passage Bio’s gene therapy candidate PBKR03 for treatment of patients with early infantile Krabbe disease, a rare pediatric disorder with no approved disease-modifying treatment options
Jan 04, 2021: Forge Biologics announces FDA clearance of Investigatiol New Drug application for phase 1/2 clinical trial (RESKUE) of FBX-101 gene therapy for patients with Krabbe disease
Oct 28, 2020: Passage Bio’s PBKR03 receives Orphan Drug and Rare Pediatric Disease Desigtions from FDA for treatment of Krabbe Disease
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
May 30, 2019: Passage Bio announces third gene therapy development program in Krabbe Disease and supports million dreams fundraising gala
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

List of Figures
Number of Products under Development for Globoid Cell Leukodystrophy (Krabbe Disease), 2021
Number of Products under Development by Companies, 2021
Number of Products under Development by Universities/Institutes, 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 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 Globoid Cell Leukodystrophy (Krabbe Disease), 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
Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by Bial - Portela & Ca SA, 2021
Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by BioXcel Corp, 2021
Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by Forge Biologics Inc, 2021
Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by Gain Therapeutics Inc, 2021
Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by Kyorin Pharmaceutical Co Ltd, 2021
Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by M6P Therapeutics, 2021
Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by Neurogene Inc, 2021
Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by Passage Bio Inc, 2021
Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by Polaryx Therapeutics Inc, 2021
Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by Vascumab LLC, 2021
Globoid Cell Leukodystrophy (Krabbe Disease) - Dormant Projects, 2021
Globoid Cell Leukodystrophy (Krabbe Disease) - Discontinued Products, 2021