Lysosomal Alpha Glucosidase - Pipeline Review, H2 2019
Summary
According to the recently published report 'Lysosomal Alpha Glucosidase - Pipeline Review, H2 2019'; 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.
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 - Pipeline Review, H2 2019' 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 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.
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
Reasons to buy- 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
- Oct 02, 2019: Amicus Therapeutics Announces Additional Positive Data in Pompe Disease Phase 1/2 Study of AT-GAA at the 24th International Annual Congress of the World Muscle Society
- Oct 01, 2019: Amicus Therapeutics to present at Upcoming Medical and Investor Conferences in October 2019
- Apr 30, 2019: Amicus Therapeutics presents positive preclinical pompe disease gene therapy data at the American Society of Gene & Cell Therapy (ASGCT) 22nd Annual Meeting
- Apr 15, 2019: Amicus Therapeutics to present preclinical Pompe Disease Gene Therapy Data at the American Society of Gene & Cell Therapy (ASGCT) 22nd Annual Meeting
- Mar 20, 2019: Amicus Therapeutics issued two U.S. patents for pompe enzyme ATB200
- Feb 28, 2019: WUXI Biologics congratulates Amicus on receiving FDA breakthrough therapy designation for AT-GAA
- Feb 25, 2019: U.S. FDA grants Breakthrough Therapy Designation to Amicus’ AT-GAA in late onset pompe disease
- Feb 11, 2019: WuXi Biologics and Amicus sign exclusive manufacturing partnership
- Feb 07, 2019: Sanofi announces additional data on avalglucosidase alfa in Pompe disease
- Feb 07, 2019: Sanofi announces new safety data for investigational avalglucosidase alfa in patients with Pompe disease
- Feb 05, 2019: Amicus Therapeutics Announces Positive data in Pompe Disease Phase 1/2 Study for Up to 24 Months at 15th Annual WORLDSymposium
- Jan 31, 2019: Spark Therapeutics announces presentation of preclinical data in Pompe disease at 15th annual WORLDSymposium
- Jan 30, 2019: Sanofi presents Avalglucosidase Alfa Development Program at 15th Annual WORLDSymposium 2019
- Jan 29, 2019: Actus Begins Gene Therapy Trial for Pompe Disease
- Jan 07, 2019: Audentes Therapeutics provides update on Pompe disease program AT-845
- 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, H2 2019
- Number of Products under Development by Therapy Areas, H2 2019
- Number of Products under Development by Indication, H2 2019
- Number of Products under Development by Companies, H2 2019
- Products under Development by Companies, H2 2019
- Products under Development by Companies, H2 2019 (Contd..1), H2 2019
- Number of Products under Investigation by Universities/Institutes, H2 2019
- Products under Investigation by Universities/Institutes, H2 2019
- Number of Products by Stage and Mechanism of Actions, H2 2019
- Number of Products by Stage and Route of Administration, H2 2019
- Number of Products by Stage and Molecule Type, H2 2019
- Pipeline by Actus Therapeutics Inc, H2 2019
- Pipeline by Amicus Therapeutics Inc, H2 2019
- Pipeline by Audentes Therapeutics Inc, H2 2019
- Pipeline by AVROBIO Inc, H2 2019
- Pipeline by Etubics Corp, H2 2019
- Pipeline by Genzyme Corp, H2 2019
- Pipeline by Greenovation Biotech GmbH, H2 2019
- Pipeline by JCR Pharmaceuticals Co Ltd, H2 2019
- Pipeline by NanoMedSyn SAS, H2 2019
- Pipeline by Oxyrane Belgium NV, H2 2019
- Pipeline by Pharming Group NV, H2 2019
- Pipeline by Spark Therapeutics Inc, H2 2019
- Pipeline by Valerion Therapeutics LLC, H2 2019
- Dormant Projects, H2 2019
- Discontinued Products, H2 2019
- List of Figures
- Number of Products under Development by Stage of Development, H2 2019
- Number of Products by Mechanism of Actions, H2 2019
- Number of Products by Stage and Mechanism of Actions, H2 2019
- Number of Products by Routes of Administration, H2 2019
- Number of Products by Stage and Routes of Administration, H2 2019
- Number of Products by Molecule Types, H2 2019
- Number of Products by Stage and Molecule Types, H2 2019