Lysosomal Alpha Glucosidase - Pipeline Review, H1 2020
Lysosomal Alpha Glucosidase - Pipeline Review, H1 2020
SUMMARY
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.
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) pipeline Target constitutes close to 17 molecules. Out of which approximately 14 molecules are developed by companies and remaining by the universities/institutes. The molecules developed by companies in Phase III, Phase II, IND/CTA Filed and Preclinical stages are 2, 2, 1 and 9 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.
The latest report Lysosomal Alpha Glucosidase - Pipeline Review, H1 2020, 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. 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.
The report is built using data and information sourced from proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources.
Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
SCOPE
SUMMARY
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.
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) pipeline Target constitutes close to 17 molecules. Out of which approximately 14 molecules are developed by companies and remaining by the universities/institutes. The molecules developed by companies in Phase III, Phase II, IND/CTA Filed and Preclinical stages are 2, 2, 1 and 9 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.
The latest report Lysosomal Alpha Glucosidase - Pipeline Review, H1 2020, 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. 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.
The report is built using data and information sourced from proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources.
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
Actus Therapeutics Inc
Amicus Therapeutics Inc
Audentes Therapeutics Inc
AVROBIO Inc
Etubics Corp
Genzyme Corp
Greenovation Biotech GmbH
JCR Pharmaceuticals Co Ltd
NanoMedSyn SAS
Oxyrane Belgium NV
Pharming Group NV
Spark Therapeutics Inc
Valerion Therapeutics LLC
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Drug Profiles
(alglucosidase 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
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
MOSS-GAA - 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
SPK-3006 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
VAL-1221 - 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
Appendix
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
Actus Therapeutics Inc
Amicus Therapeutics Inc
Audentes Therapeutics Inc
AVROBIO Inc
Etubics Corp
Genzyme Corp
Greenovation Biotech GmbH
JCR Pharmaceuticals Co Ltd
NanoMedSyn SAS
Oxyrane Belgium NV
Pharming Group NV
Spark Therapeutics Inc
Valerion Therapeutics LLC
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Drug Profiles
(alglucosidase 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
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
MOSS-GAA - 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
SPK-3006 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
VAL-1221 - 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
Appendix
LIST OF TABLES
Number of Products under Development by Stage of Development, H1 2020
Number of Products under Development by Therapy Areas, H1 2020
Number of Products under Development by Indication, H1 2020
Number of Products under Development by Companies, H1 2020
Products under Development by Companies, H1 2020
Number of Products under Investigation by Universities/Institutes, H1 2020
Products under Investigation by Universities/Institutes, H1 2020
Number of Products by Stage and Mechanism of Actions, H1 2020
Number of Products by Stage and Route of Administration, H1 2020
Number of Products by Stage and Molecule Type, H1 2020
Pipeline by Actus Therapeutics Inc, H1 2020
Pipeline by Amicus Therapeutics Inc, H1 2020
Pipeline by Audentes Therapeutics Inc, H1 2020
Pipeline by AVROBIO Inc, H1 2020
Pipeline by Etubics Corp, H1 2020
Pipeline by Genzyme Corp, H1 2020
Pipeline by Greenovation Biotech GmbH, H1 2020
Pipeline by JCR Pharmaceuticals Co Ltd, H1 2020
Pipeline by NanoMedSyn SAS, H1 2020
Pipeline by Oxyrane Belgium NV, H1 2020
Pipeline by Pharming Group NV, H1 2020
Pipeline by Spark Therapeutics Inc, H1 2020
Pipeline by Valerion Therapeutics LLC, H1 2020
Dormant Projects, H1 2020
Discontinued Products, H1 2020
Number of Products under Development by Stage of Development, H1 2020
Number of Products under Development by Therapy Areas, H1 2020
Number of Products under Development by Indication, H1 2020
Number of Products under Development by Companies, H1 2020
Products under Development by Companies, H1 2020
Number of Products under Investigation by Universities/Institutes, H1 2020
Products under Investigation by Universities/Institutes, H1 2020
Number of Products by Stage and Mechanism of Actions, H1 2020
Number of Products by Stage and Route of Administration, H1 2020
Number of Products by Stage and Molecule Type, H1 2020
Pipeline by Actus Therapeutics Inc, H1 2020
Pipeline by Amicus Therapeutics Inc, H1 2020
Pipeline by Audentes Therapeutics Inc, H1 2020
Pipeline by AVROBIO Inc, H1 2020
Pipeline by Etubics Corp, H1 2020
Pipeline by Genzyme Corp, H1 2020
Pipeline by Greenovation Biotech GmbH, H1 2020
Pipeline by JCR Pharmaceuticals Co Ltd, H1 2020
Pipeline by NanoMedSyn SAS, H1 2020
Pipeline by Oxyrane Belgium NV, H1 2020
Pipeline by Pharming Group NV, H1 2020
Pipeline by Spark Therapeutics Inc, H1 2020
Pipeline by Valerion Therapeutics LLC, H1 2020
Dormant Projects, H1 2020
Discontinued Products, H1 2020
LIST OF FIGURES
Number of Products under Development by Stage of Development, H1 2020
Number of Products by Mechanism of Actions, H1 2020
Number of Products by Stage and Mechanism of Actions, H1 2020
Number of Products by Routes of Administration, H1 2020
Number of Products by Stage and Routes of Administration, H1 2020
Number of Products by Molecule Types, H1 2020
Number of Products by Stage and Molecule Types, H1 2020
COMPANIES MENTIONED
Actus Therapeutics Inc
Amicus Therapeutics Inc
Audentes Therapeutics Inc
AVROBIO Inc
Etubics Corp
Genzyme Corp
Greenovation Biotech GmbH
JCR Pharmaceuticals Co Ltd
NanoMedSyn SAS
Oxyrane Belgium NV
Pharming Group NV
Spark Therapeutics Inc
Valerion Therapeutics LLC
Number of Products under Development by Stage of Development, H1 2020
Number of Products by Mechanism of Actions, H1 2020
Number of Products by Stage and Mechanism of Actions, H1 2020
Number of Products by Routes of Administration, H1 2020
Number of Products by Stage and Routes of Administration, H1 2020
Number of Products by Molecule Types, H1 2020
Number of Products by Stage and Molecule Types, H1 2020
COMPANIES MENTIONED
Actus Therapeutics Inc
Amicus Therapeutics Inc
Audentes Therapeutics Inc
AVROBIO Inc
Etubics Corp
Genzyme Corp
Greenovation Biotech GmbH
JCR Pharmaceuticals Co Ltd
NanoMedSyn SAS
Oxyrane Belgium NV
Pharming Group NV
Spark Therapeutics Inc
Valerion Therapeutics LLC
