Globoid Cell Leukodystrophy (Krabbe Disease) (Central Nervous System) - Drugs in Development, 2021

November 2021 | 79 pages | ID: GDEA1F0A9B4BEN
Global Markets Direct

Abstracts

Globoid Cell Leukodystrophy (Krabbe Disease) (Central Nervous System) - Drugs in Development, 2021

SUMMARY

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Globoid Cell Leukodystrophy - Drugs In Development, 2021, provides an overview of the Globoid Cell Leukodystrophy (Central Nervous System) pipeline landscape.

Krabbe disease also called globoid cell leukodystrophy is a degenerative disorder caused by the deficiency of an enzyme called galactosylceramidase. This enzyme deficiency impairs the growth and maintenance of myelin. Symptoms include seizures, Muscle spasms, Loss of head control, vomiting, fevers, irritability and excessive crying. Risk factors include family history. Treatment focuses on managing symptoms (anticonvulsant, muscle relaxer) and providing supportive care.

REPORT HIGHLIGHTS

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Globoid Cell Leukodystrophy - Drugs In Development, 2021, provides comprehensive information on the therapeutics under development for Globoid Cell Leukodystrophy (Central Nervous System), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Globoid Cell Leukodystrophy (Central Nervous System) pipeline guide also reviews of key players involved in therapeutic development for Globoid Cell Leukodystrophy (Krabbe Disease) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies/Universities/Institutes, the molecules developed by Companies in Phase II, Preclinical and Discovery stages are 2, 7 and 3 respectively. Similarly, the Universities portfolio in Phase I and Preclinical stages comprises 1 and 1 molecules, respectively.

Globoid Cell Leukodystrophy (Central Nervous System) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

SCOPE

• The pipeline guide provides a snapshot of the global therapeutic landscape of Globoid Cell Leukodystrophy (Central Nervous System).
• The pipeline guide reviews pipeline therapeutics for Globoid Cell Leukodystrophy (Central Nervous System) by companies and universities/research institutes based on information derived from company and industry-specific sources.
• The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
• The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
• The pipeline guide reviews key companies involved in Globoid Cell Leukodystrophy (Central Nervous System) therapeutics and enlists all their major and minor projects.
• The pipeline guide evaluates Globoid Cell Leukodystrophy (Central Nervous System) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
• The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
• The pipeline guide reviews latest news related to pipeline therapeutics for Globoid Cell Leukodystrophy (Central Nervous System)

REASONS TO BUY

• Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
• Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
• Find and recognize significant and varied types of therapeutics under development for Globoid Cell Leukodystrophy (Central Nervous System).
• Classify potential new clients or partners in the target demographic.
• Develop tactical initiatives by understanding the focus areas of leading companies.
• Plan mergers and acquisitions meritoriously by identifying key players and it’s most promising pipeline therapeutics.
• Formulate corrective measures for pipeline projects by understanding Globoid Cell Leukodystrophy (Central Nervous System) pipeline depth and focus of Indication therapeutics.
• Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.
• Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

Contents

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 Designation for PBKR03 for treatment of Krabbe Disease
Mar 08, 2021: Passage Bio annouced that U.S. Food and Drug Administration grants Fast Track Designation to its gene therapy candidate PBKR03
Feb 16, 2021: Forge Biologics Receives FDA Fast Track, Orphan Drug, and Rare Pediatric Disease Designations for FBX-101 Gene Therapy for Patients with Krabbe Disease
Feb 10, 2021: Polaryx Therapeutics receives both Rare Pediatric Disease and Orphan Drug Designations 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 Investigational 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 Designations 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 Designation to MediciNova’s MN-166 (ibudilast) for the Treatment of Krabbe Disease
Jun 03, 2015: MediciNova Announces FDA Granted Orphan Drug Designation to MN-166 (ibudilast) for Krabbe Disease
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

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

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