Globoid Cell Leukodystrophy (Krabbe Disease) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

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.

The Globoid Cell Leukodystrophy (Krabbe Disease) pipeline market research report provides comprehensive information on the therapeutics under development for Globoid Cell Leukodystrophy (Krabbe Disease) (Central Nervous System), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report also provides an overview of key players involved in therapeutic development for Globoid Cell Leukodystrophy (Krabbe Disease) and features dormant and discontinued projects.

Globoid Cell Leukodystrophy (Krabbe Disease) Pipeline Products Market Segmentation by Targets

The key targets in the Globoid Cell Leukodystrophy (Krabbe Disease) pipeline products market are Galactocerebrosidase, Peroxisome Proliferator Activated Receptor Alpha, Acid Ceramidase, and Retinoic Acid Receptor RXR Alpha. Galactocerebrosidase is the leading target in the pipeline.

Globoid Cell Leukodystrophy (Krabbe Disease) Pipeline Products Market Analysis by Targets

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Globoid Cell Leukodystrophy (Krabbe Disease) Pipeline Products Market Segmentation by Mechanisms of Action

The key mechanisms of action in the Globoid Cell Leukodystrophy (Krabbe Disease) pipeline products market are Galactocerebrosidase Activator, Galactocerebrosidase Replacement, Peroxisome Proliferator Activated Receptor Alpha Agonist, Acid Ceramidase Inhibitor, and Retinoic Acid Receptor RXR Alpha Agonist. Galactocerebrosidase Activator is the leading MoA in the market.

Globoid Cell Leukodystrophy (Krabbe Disease) Pipeline Products Market Analysis by Mechanisms of Action

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Globoid Cell Leukodystrophy (Krabbe Disease) Pipeline Products Market Segmentation by Routes of Administration

The key routes of administration in the Globoid Cell Leukodystrophy (Krabbe Disease) pipeline products market are oral, intravenous, and intrathecal. Oral is the most preferred route in terms of administration.

Globoid Cell Leukodystrophy (Krabbe Disease) Pipeline Products Market Analysis by Routes of Administration

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Globoid Cell Leukodystrophy (Krabbe Disease) Pipeline Products Market Segmentation by Molecule Types

The key molecule types in the Globoid Cell Leukodystrophy (Krabbe Disease) pipeline products market are small molecule, gene therapy, cell therapy, gene-modified cell therapy, and recombinant enzyme. Small molecule is the leading one in the pipeline.

Globoid Cell Leukodystrophy (Krabbe Disease) Pipeline Products Market Analysis by Molecule Types

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Globoid Cell Leukodystrophy (Krabbe Disease) Pipeline Products Market - Competitive Landscape

The leading companies in the Globoid Cell Leukodystrophy (Krabbe Disease) pipeline products market are Forge Biologics Inc, M6P Therapeutics, Polaryx Therapeutics Inc, Apteeus SAS, Bial – Portela & Ca SA, BioXcel Corp, Gain Therapeutics Inc, Neurogene Inc, and Vascumab LLC.

Globoid Cell Leukodystrophy (Krabbe Disease) Pipeline Products Market Analysis by Companies

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Globoid Cell Leukodystrophy (Krabbe Disease) Pipeline Products Market Report Overview

Scope

• The pipeline guide provides a snapshot of the global therapeutic landscape of Globoid Cell Leukodystrophy (Krabbe Disease) (Central Nervous System).
• The pipeline guide reviews pipeline therapeutics for Globoid Cell Leukodystrophy (Krabbe Disease) (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 (Krabbe Disease) (Central Nervous System) therapeutics and enlists all their major and minor projects.
• The pipeline guide evaluates Globoid Cell Leukodystrophy (Krabbe Disease) (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 (Krabbe Disease) (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 (Krabbe Disease) (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 (Krabbe Disease) (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.