Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Pipeline Review, H2 2017

  • ID: 4423496
  • Report
  • 62 pages
  • Global Markets Direct
1 of 5

FEATURED COMPANIES

  • Amicus Therapeutics Inc
  • Audentes Therapeutics Inc
  • Etubics Corp
  • Genzyme Corp
  • greenovation Biotech GmbH
  • NanoMedSyn SAS
  • MORE
Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Pipeline Review, H2 2017

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 15 molecules. Out of which approximately 12 molecules are developed by companies and remaining by the universities/institutes. The molecules developed by companies in Phase III, Phase II, IND/CTA Filed, Preclinical and Discovery stages are 1, 2, 1, 7 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.

The latest report Lysosomal Alpha Glucosidase - Pipeline Review, H2 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. 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
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
READ MORE
Note: Product cover images may vary from those shown
2 of 5

FEATURED COMPANIES

  • Amicus Therapeutics Inc
  • Audentes Therapeutics Inc
  • Etubics Corp
  • Genzyme Corp
  • greenovation Biotech GmbH
  • NanoMedSyn SAS
  • 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

JCR Pharmaceuticals Co Ltd

NanoMedSyn SAS

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

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 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

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

Oct 04, 2017: Amicus Therapeutics Announces Additional Positive Data in Pompe Disease Phase 1/2 Study at World Muscle Society

Sep 21, 2017: U.S. FDA Grants Orphan Drug Designation for ATB200/AT2221 for Pompe Disease

Jul 11, 2017: Valerion Initiates VAL-1221 Dosing in Patients with Pompe Disease

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 23, 2017: JCR to Initiate Development of Jr-162, A New Drug Candidate For Pompe Disease Using J-brain Cargo

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

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 2016

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

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

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 2017

Number of Products under Development by Therapy Areas, H2 2017

Number of Products under Development by Indication, H2 2017

Number of Products under Development by Companies, H2 2017

Products under Development by Companies, H2 2017

Number of Products under Investigation by Universities/Institutes, H2 2017

Products under Investigation by Universities/Institutes, H2 2017

Number of Products by Stage and Mechanism of Actions, H2 2017

Number of Products by Stage and Route of Administration, H2 2017

Number of Products by Stage and Molecule Type, H2 2017

Pipeline by Amicus Therapeutics Inc, H2 2017

Pipeline by Audentes Therapeutics Inc, H2 2017

Pipeline by Etubics Corp, H2 2017

Pipeline by Genzyme Corp, H2 2017

Pipeline by greenovation Biotech GmbH, H2 2017

Pipeline by JCR Pharmaceuticals Co Ltd, H2 2017

Pipeline by NanoMedSyn SAS, H2 2017

Pipeline by Oxyrane Belgium NV, H2 2017

Pipeline by Pharming Group NV, H2 2017

Pipeline by Sarepta Therapeutics Inc, H2 2017

Dormant Projects, H2 2017

Discontinued Products, H2 2017

List of Figures

Number of Products under Development by Stage of Development, H2 2017

Number of Products by Mechanism of Actions, H2 2017

Number of Products by Stage and Mechanism of Actions, H2 2017

Number of Products by Routes of Administration, H2 2017

Number of Products by Stage and Routes of Administration, H2 2017

Number of Products by Molecule Types, H2 2017

Number of Products by Stage and Molecule Types, H2
Note: Product cover images may vary from those shown
3 of 5

Loading
LOADING...

4 of 5
  • Amicus Therapeutics Inc
  • Audentes Therapeutics Inc
  • Etubics Corp
  • Genzyme Corp
  • greenovation Biotech GmbH
  • JCR Pharmaceuticals Co Ltd
  • NanoMedSyn SAS
  • 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
Adroll
adroll