Introduction
Pompe Disease - Overview
Pompe Disease - Therapeutics Development
Pompe Disease - Therapeutics Assessment
Pompe Disease - Companies Involved in Therapeutics Development
Pompe Disease - Drug Profiles
Pompe Disease - Dormant Projects
Pompe Disease - Discontinued Products
Pompe Disease - Product Development Milestones
Appendix

List of Tables
Number of Products under Development for Pompe 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
Pompe Disease - Pipeline by AavantiBio Inc, 2021
Pompe Disease - Pipeline by Amicus Therapeutics Inc, 2021
Pompe Disease - Pipeline by Asklepios BioPharmaceutical Inc, 2021
Pompe Disease - Pipeline by Astellas Gene Therapies, 2021
Pompe Disease - Pipeline by Avidity Biosciences Inc, 2021
Pompe Disease - Pipeline by AVROBIO Inc, 2021
Pompe Disease - Pipeline by CANbridge Life Sciences Ltd, 2021
Pompe Disease - Pipeline by Denali Therapeutics Inc, 2021
Pompe Disease - Pipeline by eleva GmbH, 2021
Pompe Disease - Pipeline by EpiVax Inc, 2021
Pompe Disease - Pipeline by Etubics Corp, 2021
Pompe Disease - Pipeline by Genzyme Corp, 2021
Pompe Disease - Pipeline by Icagen Inc, 2021
Pompe Disease - Pipeline by JCR Pharmaceuticals Co Ltd, 2021
Pompe Disease - Pipeline by M6P Therapeutics, 2021
Pompe Disease - Pipeline by Maze Therapeutics Inc, 2021
Pompe Disease - Pipeline by NanoMedSyn SAS, 2021
Pompe Disease - Pipeline by Oxyrane Belgium NV, 2021
Pompe Disease - Pipeline by Pharming Group NV, 2021
Pompe Disease - Pipeline by Provention Bio Inc, 2021
Pompe Disease - Pipeline by RespireRx Pharmaceuticals Inc, 2021
Pompe Disease - Pipeline by Sarepta Therapeutics Inc, 2021
Pompe Disease - Pipeline by Spark Therapeutics Inc, 2021
Pompe Disease - Pipeline by Takeda Pharmaceutical Co Ltd, 2021
Pompe Disease - Dormant Projects, 2021
Pompe Disease - Discontinued Products, 2021

List of Figures
Number of Products under Development for Pompe 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
• AavantiBio Inc
• Amicus Therapeutics Inc
• Asklepios BioPharmaceutical Inc
• Astellas Gene Therapies
• Avidity Biosciences Inc
• AVROBIO Inc
• CANbridge Life Sciences Ltd
• Denali Therapeutics Inc
• eleva GmbH
• EpiVax Inc
• Etubics Corp
• Genzyme Corp
• Icagen Inc
• JCR Pharmaceuticals Co Ltd
• M6P Therapeutics
• Maze Therapeutics Inc
• NanoMedSyn SAS
• Oxyrane Belgium NV
• Pharming Group NV
• Provention Bio Inc
• RespireRx Pharmaceuticals Inc
• Sarepta Therapeutics Inc
• Spark Therapeutics Inc
• Takeda Pharmaceutical Co Ltd