Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline Review, H1 2020

June 2020 | 77 pages | ID: G57E43F6F00EN
Global Markets Direct

Abstracts

Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline Review, H1 2020

SUMMARY

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Globoid Cell Leukodystrophy - Pipeline Review, H1 2020, 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 - Pipeline Review, H1 2020, 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 1, 5 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
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) designation 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 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), 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

List Of Figures

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

COMPANIES MENTIONED

BioXcel Corp
Commence Bio Inc
GT Gain Therapeutics SA
Kyorin Pharmaceutical Co Ltd
Magenta Therapeutics Inc
Passage Bio Inc
Polaryx Therapeutics Inc
Vascumab LLC