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

Global Markets Direct’s, ‘Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) – Pipeline Review, H2 2017’, provides in depth analysis on Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted pipeline therapeutics. The report provides comprehensive information complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics development and features dormant and discontinued projects. The report analyses the pipeline products from therapy areas Metabolic Disorders under development targeting Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20).

Global Markets Direct’s report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Direct’s 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. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.

*Updated report will be delivered in 48 hours of order confirmation.

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

Companies mentioned

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

Table of Contents

Table of Contents

Table of Contents 2

List of Tables 5

List of Figures 5

Introduction 6

Global Markets Direct Report Coverage 6

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC

3.2.1.20) - Overview 7

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC

3.2.1.20) - Therapeutics Development 8

Products under Development by Stage of Development 8

Products under Development by Therapy Area 9

Products under Development by Indication 10

Products under Development by Companies 11

Products under Development by Universities/Institutes 13

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC

3.2.1.20) - Therapeutics Assessment 15

Assessment by Mechanism of Action 15

Assessment by Route of Administration 17

Assessment by Molecule Type 19

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC

3.2.1.20) - Companies Involved in Therapeutics Development 21

Amicus Therapeutics Inc 21

Audentes Therapeutics Inc 21

Etubics Corp 22

Genzyme Corp 22

greenovation Biotech GmbH 22

JCR Pharmaceuticals Co Ltd 23

NanoMedSyn SAS 23

Oxyrane Belgium NV 23

Pharming Group NV 24

Sarepta Therapeutics Inc 24

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC

3.2.1.20) - Drug Profiles 26

Antisense Oligonucleotide to Activate Lysosomal Alpha-Glucosidase for Pompe Disease - Drug Profile 26

Product Description 26

Mechanism Of Action 26

R&D Progress 26

AT-982 - Drug Profile 27

Product Description 27

Mechanism Of Action 27

R&D Progress 27

ATB-200 + miglustat - Drug Profile 29

Product Description 29

Mechanism Of Action 29

R&D Progress 29

AVRRD-03 - Drug Profile 32

Product Description 32

Mechanism Of Action 32

R&D Progress 32

Gene Therapy 1 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile 33

Product Description 33

Mechanism Of Action 33

R&D Progress 33

Gene Therapy 2 to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile 34

Product Description 34

Mechanism Of Action 34

R&D Progress 34

Gene Therapy to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile 35

Product Description 35

Mechanism Of Action 35

R&D Progress 35

Gene Therapy to Activate Acid Alpha-Glucosidase for Pompe Disease - Drug Profile 36

Product Description 36

Mechanism Of Action 36

R&D Progress 36

GZ-402666 - Drug Profile 37

Product Description 37

Mechanism Of Action 37

R&D Progress 37

JR-162 - Drug Profile 39

Product Description 39

Mechanism Of Action 39

R&D Progress 39

MOSS-GAA - Drug Profile 40

Product Description 40

Mechanism Of Action 40

R&D Progress 40

OXY-2810 - Drug Profile 41

Product Description 41

Mechanism Of Action 41

R&D Progress 41

PGN-004 - Drug Profile 42

Product Description 42

Mechanism Of Action 42

R&D Progress 42

Recombinant Alpha Glucosidase Replacement for Pompe Disease - Drug Profile 43

Product Description 43

Mechanism Of Action 43

R&D Progress 43

VAL-1221 - Drug Profile 44

Product Description 44

Mechanism Of Action 44

R&D Progress 44

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC

3.2.1.20) - Dormant Products 46

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC

3.2.1.20) - Discontinued Products 47

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC

3.2.1.20) - Product Development Milestones 48

Featured News & Press Releases 48

Oct 04, 2017: Amicus Therapeutics Announces Additional Positive Data in Pompe Disease Phase 1/2 Study at World Muscle Society 48

Sep 21, 2017: U.S. FDA Grants Orphan Drug Designation for ATB200/AT2221 for Pompe Disease 49

Jul 11, 2017: Valerion Initiates VAL-1221 Dosing in Patients with Pompe Disease 50

May 15, 2017: Amicus Therapeutics Announces Positive Functional Data from Initial Patients in Pompe Phase 1/2 Study 51

Mar 03, 2017: Valerion Therapeutics Demonstrates a New Mechanism for Treating Pompe Disease 52

Feb 23, 2017: JCR to Initiate Development of Jr-162, A New Drug Candidate For Pompe Disease Using J-brain Cargo 53

Feb 15, 2017: Amicus Therapeutics Presents Important New Scientific Findings and Preclinical Data for Pompe Program at WORLDSymposium 2017 53

Dec 08, 2016: Amicus Therapeutics Announces Positive Preliminary Data from Phase 1/2 Study of Novel Treatment Paradigm for Pompe Disease 54

Nov 17, 2016: Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease to Begin in the UK 55

Nov 04, 2016: Sanofi Genzyme Begins Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease 56

Mar 03, 2016: Sanofi Genzyme Presents Results from Phase 1/2 Study of Investigational Second-Generation Therapy for Pompe Disease 57

Mar 01, 2016: Amicus Therapeutics Highlights Preclinical Pompe Data at WORLDSymposium 2016 58

Feb 10, 2016: Amicus Therapeutics Announces Oral and Poster Presentations on Chaperone AT2221 at 12th Annual WORLDSymposium 2016 58

Jan 11, 2016: Amicus Therapeutics Provides Update On Novel ERT for Pompe Disease (ATB200 + Chaperone) 59

Dec 22, 2015: Amicus Therapeutics Commences Phase 1/2 Study of Novel ERT for Treatment of Pompe Disease 60

Appendix 61

Methodology 61

Coverage 61

Secondary Research 61

Primary Research 61

Expert Panel Validation 61

Contact Us 61

Disclaimer 6

List of Tables

List of Tables

Number of Products under Development by Stage of Development, H2 2017 8

Number of Products under Development by Therapy Areas, H2 2017 9

Number of Products under Development by Indication, H2 2017 10

Number of Products under Development by Companies, H2 2017 11

Products under Development by Companies, H2 2017 12

Number of Products under Investigation by Universities/Institutes, H2 2017 13

Products under Investigation by Universities/Institutes, H2 2017 14

Number of Products by Stage and Mechanism of Actions, H2 2017 16

Number of Products by Stage and Route of Administration, H2 2017 18

Number of Products by Stage and Molecule Type, H2 2017 20

Pipeline by Amicus Therapeutics Inc, H2 2017 21

Pipeline by Audentes Therapeutics Inc, H2 2017 21

Pipeline by Etubics Corp, H2 2017 22

Pipeline by Genzyme Corp, H2 2017 22

Pipeline by greenovation Biotech GmbH, H2 2017 23

Pipeline by JCR Pharmaceuticals Co Ltd, H2 2017 23

Pipeline by NanoMedSyn SAS, H2 2017 23

Pipeline by Oxyrane Belgium NV, H2 2017 24

Pipeline by Pharming Group NV, H2 2017 24

Pipeline by Sarepta Therapeutics Inc, H2 2017 25

Dormant Projects, H2 2017 46

Discontinued Products, H2 2017 47

List of Figures

List of Figures

Number of Products under Development by Stage of Development, H2 2017 8

Number of Products by Mechanism of Actions, H2 2017 15

Number of Products by Stage and Mechanism of Actions, H2 2017 15

Number of Products by Routes of Administration, H2 2017 17

Number of Products by Stage and Routes of Administration, H2 2017 17

Number of Products by Molecule Types, H2 2017 19

Number of Products by Stage and Molecule Types, H2 2017 19

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