Pompe Disease – Drugs In Development, 2024

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Pompe disease is an inherited disorder caused by defect in a gene called GAA. The GAA gene is responsible for the production of the GAA enzyme (acid alpha-glucosidase). This enzyme is needed to break down glycogen, a form of sugar stored in muscle cells. When too much glycogen builds up in the muscle cells, the cells become damaged and the muscles cannot function properly. Symptoms include respiratory failure, muscle pain, proximal muscle weakness, respiratory tract infections, headache, and difficulty chewing or jaw muscle fatigue.

The Pompe Disease drugs in development market research report provide comprehensive information on the therapeutics under development for Pompe Disease, 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 and product description of the therapeutics, and the latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Pompe Disease and features dormant and discontinued products.

Note:

* This is an “on-demand” report and will be delivered within 2 business days (excluding weekends and holidays) of the purchase.

* Certain sections in the report may be removed or altered based on the availability and relevance of data.

Quick View – Pompe Disease
Key Targets
· Lysosomal Alpha Glucosidase; Glycogen Starch Synthase; B Cell Antigen Receptor Complex Associated Protein Beta Chain; and Low Affinity Immunoglobulin Gamma Fc Region Receptor II-b
Key Mechanisms of Action
· Lysosomal Alpha Glucosidase Replacement; Lysosomal Alpha Glucosidase Activator; Glycogen Starch Synthase Inhibitor; Low Affinity Immunoglobulin Gamma Fc Region Receptor II-b Agonist; and B Cell Antigen Receptor Complex Associated Protein Beta Chain Antagonist
Key Routes of Administration
· Intravenous; Oral; Parenteral; Subcutaneous; and Intramuscular
Key Molecule Types
· Gene Therapy; Recombinant Enzyme; Small Molecule; Gene-Modified Cell Therapy; Fusion Protein; Antisense Oligonucleotide; Antisense RNAi Oligonucleotide; Monoclonal Antibody; and Enzyme
Major Companies
· Sanofi; M6P Therapeutics; Amicus Therapeutics Inc; Beijing GeneCradle Technology Co Ltd; BioStrategies LC; AVROBIO Inc; Avidity Biosciences Inc; Asklepios BioPharmaceutical Inc; Astellas Pharma Inc; and Astellas Gene Therapies

Scope

  • Therapeutics in Development: Covering 34 molecules, with 31 developed by companies and the rest by universities/institutes.
  • Analysis Parameters: Offering insights by stage of development, drug target, MoA, RoA, and molecule type for a comprehensive overview.
  • Pharmacological Insights: Understand the descriptive pharmacological action of therapeutics.
  • Development History: Access the complete research and development history.
  • Latest News and Press Releases: Stay updated with the latest developments through comprehensive news coverage.

Reasons to Buy

  • Holistic insights: Understand the broad spectrum of Pompe Disease therapeutics, aiding strategic decision-making with insights into stages, targets, MoA, RoA and molecule types.
  • Pipeline Exploration: Explore the detailed Pompe Disease pipeline, offering nuanced analysis of drug targets, mechanisms of action and routes of administration for varied decision-making.
  • Comprehensive R&D: Access thorough R&D histories, providing a comprehensive understanding of Pompe Disease treatments for adaptable decision-making.
  • Save valuable hours: Identify key players steering innovation in therapeutics, enabling strategic partnerships.

Key Players

Amicus Therapeutics Inc
Aro Biotherapeutics Co
Asklepios BioPharmaceutical Inc
Astellas Gene Therapies
Astellas Pharma Inc
Avidity Biosciences Inc
AVROBIO Inc
Beijing GeneCradle Technology Co Ltd
BioStrategies LC
CANbridge Life Sciences Ltd
Denali Therapeutics Inc
Eleva GmbH
Entrada Therapeutics Inc
Equaly SA
Erasmus MC
Icagen Inc
ImmunityBio Inc
Intellia Therapeutics Inc
JCR Pharmaceuticals Co Ltd
Lapix Therapeutics Inc
M6P Therapeutics
NanoMedSyn SAS
Oxyrane Belgium NV
Provention Bio Inc
Sanofi
Sarepta Therapeutics Inc
Solid Biosciences Inc
Spark Therapeutics Inc
Takeda Pharmaceutical Co Ltd
University of Florida
University of Massachusetts

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Overview

Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Drugs Under Development by Companies

Drugs under Development by Universities/Institutes

Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Companies Involved in Therapeutic Development

Drug Profiles

Dormant Projects

Discontinued Drugs

Product Development Milestones

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

Table

List of Tables

Number of Drugs under Development, 2024

Number of Drugs under Development by Companies, 2024

Number of Drugs under Development by Universities/Institutes, 2024

Drugs under Development by Companies, 2024

Drugs under Development by Universities/Institutes, 2024

Number of Drugs by Stage and Target, 2024

Number of Drugs by Stage and Mechanism of Action, 2024

Number of Drugs by Stage and Route of Administration, 2024

Number of Drugs by Stage and Molecule Type, 2024

Dormant Projects, 2024

Discontinued Drugs, 2024

Figures

List of Figures

Number of Drugs under Development, 2024

Number of Drugs under Development by Companies, 2024

Number of Drugs under Development by Universities/Institutes, 2024

Number of Drugs by Top 10 Targets, 2024

Number of Drugs by Stage and Top 10 Targets, 2024

Number of Drugs by Top 10 Mechanism of Actions, 2024

Number of Drugs by Stage and Top 10 Mechanism of Actions, 2024

Number of Drugs by Top 10 Routes of Administration, 2024

Number of Drugs by Stage and Top 10 Routes of Administration, 2024

Number of Drugs by Top 10 Molecule Types, 2024

Number of Drugs by Stage and Top 10 Molecule Types, 2024

Frequently asked questions

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