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Pompe Disease-Pipeline Review, H1 2017

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Pompe Disease-Pipeline Review, H1 2017



Executive Summary

Pompe Disease-Pipeline Review, H1 2017

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Pompe Disease-Pipeline Review, H1 2017, provides an overview of the Pompe Disease (Metabolic Disorders) pipeline landscape.

Pompe disease is an inherited disorder caused by defect in a gene called GAA. The GAA gene is responsible for the production of the GAA enzyme (acid alpha-glucosidase). This enzyme is needed to break down glycogen, a form of sugar stored in muscle cells. When too much glycogen builds up in the muscle cells, the cells become damaged and the muscles cannot function properly. Symptoms include respiratory failure, muscle pain, proximal muscle weakness, respiratory tract infections, headache and difficulty chewing or jaw muscle fatigue.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Pompe Disease-Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Pompe Disease (Metabolic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Pompe Disease (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Pompe Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, IND/CTA Filed, Preclinical and Discovery stages are 1, 1, 1, 8 and 2 respectively. Similarly, the Universities portfolio in Phase II and Preclinical stages comprises 1 and 2 molecules, respectively.

Pompe Disease (Metabolic Disorders) pipeline guide 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. The guide 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

The pipeline guide provides a snapshot of the global therapeutic landscape of Pompe Disease (Metabolic Disorders).

The pipeline guide reviews pipeline therapeutics for Pompe Disease (Metabolic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.

The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.

The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.

The pipeline guide reviews key companies involved in Pompe Disease (Metabolic Disorders) therapeutics and enlists all their major and minor projects.

The pipeline guide evaluates Pompe Disease (Metabolic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.

The pipeline guide encapsulates all the dormant and discontinued pipeline projects.

The pipeline guide reviews latest news related to pipeline therapeutics for Pompe Disease (Metabolic Disorders)

Reasons to buy

Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.

Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.

Find and recognize significant and varied types of therapeutics under development for Pompe Disease (Metabolic Disorders).

Classify potential new clients or partners in the target demographic.

Develop tactical initiatives by understanding the focus areas of leading companies.

Plan mergers and acquisitions meritoriously by identifying key players and it's most promising pipeline therapeutics.

Formulate corrective measures for pipeline projects by understanding Pompe Disease (Metabolic Disorders) pipeline depth and focus of Indication therapeutics.

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.

Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Pompe Disease-Overview

Pompe Disease-Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Pompe Disease-Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Pompe Disease-Companies Involved in Therapeutics Development

Amicus Therapeutics Inc

Audentes Therapeutics Inc

EpiVax Inc

Etubics Corp

Genzyme Corp

greenovation Biotech GmbH

Huons Co Ltd

JCR Pharmaceuticals Co Ltd

Oxyrane Belgium NV

Pharming Group NV

Sarepta Therapeutics Inc

Pompe Disease-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

Synthetic Peptides 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

Pompe Disease-Dormant Projects

Pompe Disease-Discontinued Products

Pompe Disease-Product Development Milestones

Featured News & Press Releases

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

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

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

To know more information on Purchase by Section, please send a mail to sales@kenresearch.com

List of Figures

Number of Products under Development for Pompe Disease, H1 2017

Number of Products under Development by Companies, H1 2017

Number of Products under Development by Universities/Institutes, H1 2017

Number of Products by Stage and Top 10 Targets, H1 2017

Number of Products by Mechanism of Actions, H1 2017

Number of Products by Stage and Mechanism of Actions, H1 2017

Number of Products by Routes of Administration, H1 2017

Number of Products by Stage and Routes of Administration, H1 2017

Number of Products by Molecule Types, H1 2017

Number of Products by Stage and Molecule Types, H1 2017

List of Tables

Number of Products under Development for Pompe Disease, H1 2017

Number of Products under Development by Companies, H1 2017

Number of Products under Development by Universities/Institutes, H1 2017

Products under Development by Companies, H1 2017

Products under Development by Universities/Institutes, H1 2017

Number of Products by Stage and Target, H1 2017

Number of Products by Stage and Mechanism of Action, H1 2017

Number of Products by Stage and Route of Administration, H1 2017

Number of Products by Stage and Molecule Type, H1 2017

Pompe Disease-Pipeline by Amicus Therapeutics Inc, H1 2017

Pompe Disease-Pipeline by Audentes Therapeutics Inc, H1 2017

Pompe Disease-Pipeline by EpiVax Inc, H1 2017

Pompe Disease-Pipeline by Etubics Corp, H1 2017

Pompe Disease-Pipeline by Genzyme Corp, H1 2017

Pompe Disease-Pipeline by greenovation Biotech GmbH, H1 2017

Pompe Disease-Pipeline by Huons Co Ltd, H1 2017

Pompe Disease-Pipeline by JCR Pharmaceuticals Co Ltd, H1 2017

Pompe Disease-Pipeline by Oxyrane Belgium NV, H1 2017

Pompe Disease-Pipeline by Pharming Group NV, H1 2017

Pompe Disease-Pipeline by Sarepta Therapeutics Inc, H1 2017

Pompe Disease-Dormant Projects, H1 2017

Pompe Disease-Discontinued Products, H1 2017

Single User License:
Report can be used by individual purchaser only

Site License:
Report can be shared by unlimited users within one corporate location, e.g. a regional office

Corporate User License:
Report can be shared globally by unlimited users within the purchasing corporation e.g. all employees of a single company

Amicus Therapeutics Inc, Audentes Therapeutics Inc, EpiVax Inc, Etubics Corp, Genzyme Corp, greenovation Biotech GmbH, Huons Co Ltd, JCR Pharmaceuticals Co Ltd, Oxyrane Belgium NV, Pharming Group NV, Sarepta Therapeutics Inc

Pompe Disease Therapeutic Products under Development, Key Players in Pompe Disease Therapeutics, Pompe Disease Pipeline Overview, Pompe Disease Pipeline, Pompe Disease Pipeline Assessment


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