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

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    Report

  • 79 Pages
  • November 2021
  • Region: Global
  • Global Markets Direct
  • ID: 5509471
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The publisher'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


The publisher'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 the publisher’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.

Table of Contents

Introduction
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
  • 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
  • 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

Companies Mentioned

A selection of companies mentioned in this report includes:

  • 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