Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Drugs in Development, 2021
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Drugs in Development, 2021
SUMMARY
According to the recently published report 'Lysosomal Alpha Glucosidase - Drugs in Development, 2021'; Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) pipeline Target constitutes close to 19 molecules. Out of which approximately 16 molecules are developed by companies and remaining by the universities/institutes.
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Lysosomal alpha-glucosidase is an enzyme encoded by the GAA gene. It is essential for the degradation of glygogen to glucose in lysosomes. Defects in this gene lead to glycogen storage disease II or Pompe disease.
The report 'Lysosomal Alpha Glucosidase - Drugs in Development, 2021' outlays comprehensive information on the Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) 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 Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics development with respective active and dormant or discontinued projects. Currently, The molecules developed by companies in Pre-Registration, Phase III, Phase II, Preclinical and Discovery stages are 1, 1, 3, 10 and 1 respectively. Similarly, the universities portfolio in Phase II, Phase I and Preclinical stages comprises 1, 1 and 1 molecules, respectively. Report covers products from therapy areas Metabolic Disorders which include indications Pompe Disease.
Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
SCOPE
SUMMARY
According to the recently published report 'Lysosomal Alpha Glucosidase - Drugs in Development, 2021'; Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) pipeline Target constitutes close to 19 molecules. Out of which approximately 16 molecules are developed by companies and remaining by the universities/institutes.
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Lysosomal alpha-glucosidase is an enzyme encoded by the GAA gene. It is essential for the degradation of glygogen to glucose in lysosomes. Defects in this gene lead to glycogen storage disease II or Pompe disease.
The report 'Lysosomal Alpha Glucosidase - Drugs in Development, 2021' outlays comprehensive information on the Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) 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 Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics development with respective active and dormant or discontinued projects. Currently, The molecules developed by companies in Pre-Registration, Phase III, Phase II, Preclinical and Discovery stages are 1, 1, 3, 10 and 1 respectively. Similarly, the universities portfolio in Phase II, Phase I and Preclinical stages comprises 1, 1 and 1 molecules, respectively. Report covers products from therapy areas Metabolic Disorders which include indications Pompe Disease.
Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
SCOPE
- The report provides a snapshot of the global therapeutic landscape for Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)
- The report reviews Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics under development by companies and universities/research institutes based on information derived from company and industry-specific sources
- The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages
- The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities
- The report reviews key players involved in Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics and enlists all their major and minor projects
- The report assesses Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics based on mechanism of action (MoA), route of administration (RoA) and molecule type
- The report summarizes all the dormant and discontinued pipeline projects
- The report reviews latest news and deals related to Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics
- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
- Identify and understand the targeted therapy areas and indications for Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20)Identify the use of drugs for target identification and drug repurposing
- Identify potential new clients or partners in the target demographic
- Develop strategic initiatives by understanding the focus areas of leading companies
- Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics
- Devise corrective measures for pipeline projects by understanding Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) development landscape
- 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
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
Denali Therapeutics Inc
eleva GmbH
Etubics Corp
Genzyme Corp
JCR Pharmaceuticals Co Ltd
M6P Therapeutics
NanoMedSyn 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
Recombinant 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 additional clinical data for investigational avalglucosidase alfa in Pompe disease
Feb 01, 2021: Spark Therapeutics announces first participant dosed in phase 1/2 study of investigational 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 investigational 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 investigational 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
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
Denali Therapeutics Inc
eleva GmbH
Etubics Corp
Genzyme Corp
JCR Pharmaceuticals Co Ltd
M6P Therapeutics
NanoMedSyn 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
Recombinant 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 additional clinical data for investigational avalglucosidase alfa in Pompe disease
Feb 01, 2021: Spark Therapeutics announces first participant dosed in phase 1/2 study of investigational 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 investigational 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 investigational 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 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
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
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
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
