Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Pipeline Review, H1 2017

  • ID: 4284829
  • Report
  • 60 pages
  • Global Markets Direct
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FEATURED COMPANIES

  • Amicus Therapeutics Inc
  • Audentes Therapeutics Inc
  • Etubics Corp
  • Genzyme Corp
  • greenovation Biotech GmbH
  • Huons Co Ltd
  • MORE
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Pipeline Review, H1 2017

Summary:

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) pipeline Target constitutes close to 15 molecules. Out of which approximately 12 molecules are developed by companies and remaining by the universities/institutes. The latest report Lysosomal Alpha Glucosidase - Pipeline Review, H1 2017, 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.

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 molecules developed by companies in Phase III, Phase II, IND/CTA Filed, Preclinical and Discovery stages are 1, 1, 1, 7 and 2 respectively. Similarly, the universities portfolio in Phase II and Preclinical stages comprises 1 and 2 molecules, respectively. Report covers products from therapy areas Metabolic Disorders which include indications Pompe Disease.

Furthermore, this report 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. Driven by 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.

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
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Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • Amicus Therapeutics Inc
  • Audentes Therapeutics Inc
  • Etubics Corp
  • Genzyme Corp
  • greenovation Biotech GmbH
  • Huons Co Ltd
  • MORE
Introduction

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

Audentes Therapeutics Inc

Etubics Corp

Genzyme Corp

greenovation Biotech GmbH

Huons Co Ltd

JCR Pharmaceuticals Co Ltd

Oxyrane Belgium NV

Pharming Group NV

Sarepta Therapeutics Inc

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Drug Profiles

Antisense Oligonucleotide to Activate Lysosomal Alpha-Glucosidase for Pompe Disease - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

AT-982 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ATB-200 + miglustat - 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 Acid Alpha-Glucosidase for Pompe Disease - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

GZ-402666 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

HU-023 - 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

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

May 15, 2017: Amicus Therapeutics Announces Positive Functional Data from Initial Patients in Pompe Phase 1/2 Study

Mar 03, 2017: Valerion Therapeutics Demonstrates a New Mechanism for Treating Pompe Disease

Feb 15, 2017: Amicus Therapeutics Presents Important New Scientific Findings and Preclinical Data for Pompe Program at WORLDSymposium

Dec 08, 2016: Amicus Therapeutics Announces Positive Preliminary Data from Phase 1/2 Study of Novel Treatment Paradigm for Pompe Disease

Nov 17, 2016: Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease to Begin in the UK

Nov 04, 2016: Sanofi Genzyme Begins Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease

Mar 03, 2016: Sanofi Genzyme Presents Results from Phase 1/2 Study of Investigational Second-Generation Therapy for Pompe Disease

Mar 01, 2016: Amicus Therapeutics Highlights Preclinical Pompe Data at WORLDSymposium

Feb 10, 2016: Amicus Therapeutics Announces Oral and Poster Presentations on Chaperone AT2221 at 12th Annual WORLDSymposium

Jan 11, 2016: Amicus Therapeutics Provides Update On Novel ERT for Pompe Disease (ATB200 + Chaperone)

Dec 22, 2015: Amicus Therapeutics Commences Phase 1/2 Study of Novel ERT for Treatment of Pompe Disease

Sep 03, 2015: Valerion Therapeutics Announces Positive Results Of Non-Clinical Research Study at SSIEM Annual Symposium

May 11, 2015: Audentes Therapeutics To Present New Data On AT-002 at American Society of Gene and Cell Therapy Annual Meeting

Feb 10, 2015: Amicus Therapeutics Presents Preclinical Data on Next-Generation Pompe ERT at WORLD Symposium

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, H1

Number of Products under Development by Therapy Areas, H1

Number of Products under Development by Indication, H1

Number of Products under Development by Companies, H1

Products under Development by Companies, H1

Number of Products under Investigation by Universities/Institutes, H1

Products under Investigation by Universities/Institutes, H1

Number of Products by Stage and Mechanism of Actions, H1

Number of Products by Stage and Route of Administration, H1

Number of Products by Stage and Molecule Type, H1

Pipeline by Amicus Therapeutics Inc, H1

Pipeline by Audentes Therapeutics Inc, H1

Pipeline by Etubics Corp, H1

Pipeline by Genzyme Corp, H1

Pipeline by greenovation Biotech GmbH, H1

Pipeline by Huons Co Ltd, H1

Pipeline by JCR Pharmaceuticals Co Ltd, H1

Pipeline by Oxyrane Belgium NV, H1

Pipeline by Pharming Group NV, H1

Pipeline by Sarepta Therapeutics Inc, H1

Dormant Projects, H1

Discontinued Products, H1

List of Figures:

Number of Products under Development by Stage of Development, H1

Number of Products by Mechanism of Actions, H1

Number of Products by Stage and Mechanism of Actions, H1

Number of Products by Routes of Administration, H1

Number of Products by Stage and Routes of Administration, H1

Number of Products by Molecule Types, H1

Number of Products by Stage and Molecule Types, H
Note: Product cover images may vary from those shown
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  • Amicus Therapeutics Inc
  • Audentes Therapeutics Inc
  • Etubics Corp
  • Genzyme Corp
  • greenovation Biotech GmbH
  • Huons Co Ltd
  • JCR Pharmaceuticals Co Ltd
  • Oxyrane Belgium NV
  • Pharming Group NV
  • Sarepta Therapeutics Inc
Note: Product cover images may vary from those shown
5 of 5
Note: Product cover images may vary from those shown
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