Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) Development by Therapy Areas and Indications, Stages, MoA, RoA, Molecule Type and Key Players, 2022 Update
Description
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) Development by Therapy Areas and Indications, Stages, MoA, RoA, Molecule Type and Key Players, 2022 Update
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 glycogen 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 20 molecules. Out of which approximately 17 molecules are developed by companies and remaining by the universities/institutes. The molecules developed by companies in Pre-Registration, Phase II, Preclinical and Discovery stages are 2, 3, 10 and 2 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 - Drugs In Development, 2022, 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 glycogen 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 20 molecules. Out of which approximately 17 molecules are developed by companies and remaining by the universities/institutes. The molecules developed by companies in Pre-Registration, Phase II, Preclinical and Discovery stages are 2, 3, 10 and 2 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 - Drugs In Development, 2022, 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
Companies Mentioned
Companies Mentioned
Amicus Therapeutics Inc
Asklepios BioPharmaceutical Inc
Astellas Gene Therapies
AVROBIO Inc
Denali Therapeutics Inc
Equaly SA
Genzyme Corp
ImmunityBio Inc
JCR Pharmaceuticals Co Ltd
M6P Therapeutics
NanoMedSyn SAS
Oxyrane Belgium NV
Pharming Group NV
Spark Therapeutics Inc
Companies Mentioned
Amicus Therapeutics Inc
Asklepios BioPharmaceutical Inc
Astellas Gene Therapies
AVROBIO Inc
Denali Therapeutics Inc
Equaly SA
Genzyme Corp
ImmunityBio Inc
JCR Pharmaceuticals Co Ltd
M6P Therapeutics
NanoMedSyn SAS
Oxyrane Belgium NV
Pharming Group NV
Spark Therapeutics Inc
Table of Contents
66 Pages
- 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
- Astellas Gene Therapies
- AVROBIO Inc
- Denali Therapeutics Inc
- Equaly SA
- Genzyme Corp
- ImmunityBio Inc
- 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
- History of Events
- ACTUS-101 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- AT-845 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- avalglucosidase alfa - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- AVRRD-03 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Fusion Protein to Replace GAA for Pompe Disease - Drug Profile
- Product Description
- Mechanism Of Action
- Gene Therapy 1 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Gene Therapy 2 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Gene Therapy to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile
- Product Description
- Mechanism Of Action
- Gene Therapy to Activate GAA for Pompe Disease - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Gene Therapy to Activate GAA for Pompe Disease - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- JR-162 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- M-021 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- M-023 - Drug Profile
- Product Description
- Mechanism Of Action
- OXY-2810 - Drug Profile
- Product Description
- Mechanism Of Action
- PGN-004 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Recombinant Alpha Glucosidase Replacement for Pompe Disease - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Recombinant Enzyme to Replace Lysosomal Alpha-Glucosidase for Pompe Disease - Drug Profile
- Product Description
- Mechanism Of Action
- RPV-002 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- SPK-3006 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- 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
- Feb 11, 2022: M6P Therapeutics presents promising preclinical data in Pompe disease at the 18th Annual WORLDSymposium 2022
- Feb 08, 2022: Nexviazyme (avalglucosidase alfa) shows sustained improvements in respiratory function and mobility in patients with Pompe disease
- Feb 07, 2022: Astellas announces positive safety data from the FORTIS study of AT845 in adults with late-onset Pompe disease
- Dec 03, 2021: European Medicines Agency validates Amicus Therapeutics marketing authorization applications for AT-GAA for the treatment of Pompe disease
- Nov 29, 2021: Sanofi’s Pompe Disease drug Nexviazyme now available in Japan
- Nov 18, 2021: The Lancet Neurology publishes pivotal Phase 3 PROPEL study results of AT-GAA in late-onset Pompe disease
- Nov 15, 2021: Nexviazyme (avalglucosidase alfa for injection) is now approved in Canada for patients with late-onset Pompe disease (acid a-glucosidase deficiency)
- Nov 12, 2021: Sanofi announces results of CHMP re-examination of the New Active Substance status for avalglucosidase alfa, a potential new standard of care for the treatment of Pompe disease
- Sep 29, 2021: U.S. FDA accepts filings for Amicus’ AT-GAA for the treatment of Pompe disease
- Sep 27, 2021: Orsini Specialty Pharmacy selected By Sanofi as a limited distribution partner for Nexviazyme
- Sep 20, 2021: Amicus Therapeutics announces presentations at the 26th International Annual Congress of the World Muscle Society
- Sep 17, 2021: CHMP announces re-examinations of recommendations for Nexviadyme
- Aug 09, 2021: FDA approves Sanofi’s Nexviazyme for Pompe disease treatment
- Aug 06, 2021: FDA approves Nexviazyme (avalglucosidase alfa-ngpt), an important new treatment option for late-onset Pompe disease
- Jul 27, 2021: Sanofi provides update on avalglucosidase alfa EU submission for patients with Pompe Disease
- 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, 2022
- Number of Products under Development by Therapy Areas, 2022
- Number of Products under Development by Indication, 2022
- Number of Products under Development by Companies, 2022
- Products under Development by Companies, 2022
- Products under Development by Companies, 2022 (Contd..1)
- Number of Products under Investigation by Universities/Institutes, 2022
- Products under Investigation by Universities/Institutes, 2022
- Number of Products by Stage and Mechanism of Actions, 2022
- Number of Products by Stage and Route of Administration, 2022
- Number of Products by Stage and Molecule Type, 2022
- Pipeline by Amicus Therapeutics Inc, 2022
- Pipeline by Asklepios BioPharmaceutical Inc, 2022
- Pipeline by Astellas Gene Therapies, 2022
- Pipeline by AVROBIO Inc, 2022
- Pipeline by Denali Therapeutics Inc, 2022
- Pipeline by Equaly SA, 2022
- Pipeline by Genzyme Corp, 2022
- Pipeline by ImmunityBio Inc, 2022
- Pipeline by JCR Pharmaceuticals Co Ltd, 2022
- Pipeline by M6P Therapeutics, 2022
- Pipeline by NanoMedSyn SAS, 2022
- Pipeline by Oxyrane Belgium NV, 2022
- Pipeline by Pharming Group NV, 2022
- Pipeline by Spark Therapeutics Inc, 2022
- Dormant Projects, 2022
- Discontinued Products, 2022
- List of Figures
- Number of Products under Development by Stage of Development, 2022
- Number of Products by Mechanism of Actions, 2022
- Number of Products by Stage and Mechanism of Actions, 2022
- Number of Products by Routes of Administration, 2022
- Number of Products by Stage and Routes of Administration, 2022
- Number of Products by Molecule Types, 2022
- Number of Products by Stage and Molecule Types, 2022
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