Table of Content


List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Overview
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Therapeutics Development
Products under Development by Stage of Development
Products under Development by Therapy Area
Products under Development by Indication
Products under Development by Companies
Products under Development by Universities/Institutes
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Therapeutics Assessment
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Companies Involved in Therapeutics Development
Amicus Therapeutics Inc
Asklepios BioPharmaceutical Inc
Audentes Therapeutics Inc
AVROBIO Inc
Deli Therapeutics Inc
eleva GmbH
Etubics Corp
Genzyme Corp
JCR Pharmaceuticals Co Ltd
M6P Therapeutics
noMedSyn SAS
Oxyrane Belgium NV
Pharming Group NV
Spark Therapeutics Inc
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Drug Profiles
(cipaglucosidase alfa + miglustat) - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ACTUS-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AT-845 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
avalglucosidase alfa - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AVRRD-03 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Fusion Protein to Replace GAA for Pompe Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy 1 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy 2 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate GAA for Pompe Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate GAA for Pompe Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
JR-162 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
M-021 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
M-023 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
OXY-2810 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PGN-004 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombint Alpha Glucosidase Replacement for Pompe Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
RPV-002 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SPK-3006 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Dormant Products
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Discontinued Products
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Product Development Milestones
Featured News & Press Releases
Mar 15, 2021: Amicus Therapeutics announces presentation and posters at the 2021 MDA Clinical & Scientific Conference
Feb 11, 2021: Amicus’ AT-GAA shows clinically meaningful & significant improvements in both musculoskeletal and respiratory measures in late-onset Pompe disease compared to standard of care in pivotal phase 3 PROPEL study
Feb 02, 2021: Data presented at WORLDSymposium reinforces robust rare disease pipeline and highlights additiol clinical data for investigatiol avalglucosidase alfa in Pompe disease
Feb 01, 2021: Spark Therapeutics announces first participant dosed in phase 1/2 study of investigatiol gene therapy for late-onset pompe disease
Feb 01, 2021: AVROBIO announces clinical data presentation on AVRRD-03 at 17th Annual WORLDSymposium 2021
Jan 20, 2021: Sanofi announced that it has submitted a new drug application in Japan for avalglucosidase alfa, an investigatiol enzyme replacement therapy
Dec 01, 2020: Amicus Therapeutics initiates rolling Biologic License Application to the U.S. Food and Drug Administration for AT-GAA in late-onset Pompe disease
Nov 18, 2020: FDA grants priority review for avalglucosidase alfa, a potential new therapy for Pompe disease
Oct 02, 2020: EMA accepts regulatory submission for avalglucosidase alfa, a potentially new standard of care enzyme replacement therapy for Pompe disease
Jun 16, 2020: Sanofi’s investigatiol enzyme replacement therapy shows clinically meaningful improvement in critical manifestations of late-onset Pompe disease
Jun 08, 2020: Sanofi to present Phase 3 results of avalglucosidase alfa in patients with late-onset Pompe disease
May 14, 2020: AVROBIO presents new preclinical data on lentiviral gene therapy program for Pompe Disease at ASGCT 2020
May 05, 2020: Amicus Therapeutics announces presentations on its pompe disease gene therapy at the American Society of Gene & Cell Therapy 23rd Annual Meeting
May 05, 2020: AVROBIO to present preclinical data at upcoming American Society of Gene & Cell Therapy (ASGCT) annual meeting, May 12-15, 2020
Feb 07, 2020: Amicus Therapeutics announces presentation on its Pompe Disease gene therapy at 16th Annual WORLDSymposium 2020
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer



List of Figures


Number of Products under Development by Stage of Development, 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


List of Tables


Number of Products under Development by Stage of Development, 2021
Number of Products under Development by Therapy Areas, 2021
Number of Products under Development by Indication, 2021
Number of Products under Development by Companies, 2021
Products under Development by Companies, 2021
Products under Development by Companies, 2021 (Contd..1)
Number of Products under Investigation by Universities/Institutes, 2021
Products under Investigation by Universities/Institutes, 2021
Number of Products by Stage and Mechanism of Actions, 2021
Number of Products by Stage and Route of Administration, 2021
Number of Products by Stage and Molecule Type, 2021
Pipeline by Amicus Therapeutics Inc, 2021
Pipeline by Asklepios BioPharmaceutical Inc, 2021
Pipeline by Audentes Therapeutics Inc, 2021
Pipeline by AVROBIO Inc, 2021
Pipeline by Denali Therapeutics Inc, 2021
Pipeline by eleva GmbH, 2021
Pipeline by Etubics Corp, 2021
Pipeline by Genzyme Corp, 2021
Pipeline by JCR Pharmaceuticals Co Ltd, 2021
Pipeline by M6P Therapeutics, 2021
Pipeline by NanoMedSyn SAS, 2021
Pipeline by Oxyrane Belgium NV, 2021
Pipeline by Pharming Group NV, 2021
Pipeline by Spark Therapeutics Inc, 2021
Dormant Projects, 2021
Discontinued Products, 2021