Pompe Disease Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

Pompe disease is an inherited disorder caused by a defect in a gene called GAA. The GAA gene is responsible for the production of the GAA enzyme (acid alpha-glucosidase). The Pompe Disease pipeline drugs market research report outlays comprehensive information on the Pompe Disease targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA), and molecule type, that are being developed by companies/universities. It also reviews key players involved in Pompe Disease targeted therapeutics development with respective active and dormant or discontinued projects.

Pompe Disease Pipeline Drugs Market Segmentation by Targets

Some of the primary targets in the Pompe Disease pipeline drugs market are Lysosomal Alpha Glucosidase, Glycogen Starch Synthase, Glutamate Ionotropic Receptor AMPA Type Subunit, B Cell Antigen Receptor Complex Associated Protein Beta Chain, and Low Affinity Immunoglobulin Gamma Fc Region Receptor II-b. Lysosomal Alpha Glucosidase accounted for the largest target in the Pompe Disease pipeline drugs market.

Pompe Disease Pipeline Drugs Market Analysis, by Targets

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Pompe Disease Pipeline Drugs Market Segmentation by Mechanisms of Action

The primary mechanisms of action in the Pompe Disease pipeline drugs market are Lysosomal Alpha Glucosidase Replacement, Lysosomal Alpha Glucosidase Activator, Glycogen Starch Synthase Inhibitor, Glutamate Ionotropic Receptor AMPA Type Subunit Agonist, B Cell Antigen Receptor Complex Associated Protein Beta Chain Antagonist, and Low Affinity Immunoglobulin Gamma Fc Region Receptor II-b Agonist. Lysosomal Alpha Glucosidase Replacement has the maximum number of products in the Pompe Disease pipeline drugs market.

Pompe Disease Pipeline Drugs Market Analysis, by Mechanisms of Action

To know more about the MoA in the Pompe Disease pipeline drugs market, download a free report sample

Pompe Disease Pipeline Drugs Market Segmentation by Routes of Administration

The key routes of administration in the Pompe Disease pipeline drugs market are Intravenous, Oral, Intramuscular, and Subcutaneous. Intravenous has the majority number of products in the Pompe Disease pipeline drugs market.

Pompe Disease Pipeline Drugs Market Analysis, by Routes of Administration

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Pompe Disease Pipeline Drugs Market Segmentation by Molecule Type

The key molecule types in the Pompe Disease pipeline drugs market are Gene Therapy, Recombinant Enzyme, Small Molecule, Fusion Protein, Antisense Oligonucleotide, Gene-Modified Cell Therapy, Monoclonal Antibody, Monoclonal Antibody Conjugated, and Oligonucleotide.

Pompe Disease Pipeline Drugs Market Analysis, by Molecule Type

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Competitive Landscape

Some of the leading companies in the Pompe Disease pipeline drugs market are Amicus Therapeutics Inc, Asklepios BioPharmaceutical Inc, M6P Therapeutics, RespireRx Pharmaceuticals Inc, Sanofi, AavantiBio Inc, Aro Biotherapeutics Co, Avidity Biosciences Inc, AVROBIO Inc, and Beijing GeneCradle Technology Co Ltd. Amicus Therapeutics Inc accounted for the largest Pompe Disease pipeline drugs market share in 2022.

Pompe Disease Pipeline Drugs Market Analysis, by Leading Companies

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Pompe Disease Pipeline Drugs Market Report Overview

Scope

• The pipeline guide provides a snapshot of the global therapeutic landscape of Pompe Disease (Metabolic Disorders).
• The pipeline guide reviews pipeline therapeutics for Pompe Disease (Metabolic Disorders) 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 Pompe Disease (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
• The pipeline guide evaluates Pompe Disease (Metabolic Disorders) 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 Pompe Disease (Metabolic Disorders)

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 Pompe Disease (Metabolic Disorders).
• 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 Pompe Disease (Metabolic Disorders) 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 understanding from the know-how what drove them from the pipeline.