Pompe Disease - Pipeline Review, H1 2018

  • ID: 4521326
  • Drug Pipelines
  • 69 pages
  • Global Markets Direct
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FEATURED COMPANIES

  • Amicus Therapeutics Inc
  • Audentes Therapeutics Inc
  • EpiVax Inc
  • Etubics Corp
  • Genzyme Corp
  • greenovation Biotech GmbH
  • MORE
Pompe Disease - Pipeline Review, H1 2018

Summary:

This latest Pharmaceutical and Healthcare disease pipeline guide Pompe Disease - Pipeline Review, H1 2018, 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:

This latest pipeline guide Pompe Disease - Pipeline Review, H1 2018, 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, Phase I, Preclinical and Discovery stages are 1, 2, 1, 12 and 1 respectively. Similarly, the Universities portfolio in Phase II and Phase I stages comprises 1 and 1 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 the researcher'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 for Purchase:
  • 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.
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Note: Product cover images may vary from those shown
2 of 5

FEATURED COMPANIES

  • Amicus Therapeutics Inc
  • Audentes Therapeutics Inc
  • EpiVax Inc
  • Etubics Corp
  • Genzyme Corp
  • greenovation Biotech GmbH
  • MORE
Introduction

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

JCR Pharmaceuticals Co Ltd

NanoMedSyn SAS

Oxyrane Belgium NV

Pharming Group NV

RespireRx Pharmaceuticals Inc

Sarepta Therapeutics Inc

Spark Therapeutics Inc

Pompe Disease - Drug Profiles

(ATB-200 + miglustat) - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

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

avalglucosidase alfa - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

AVRRD-03 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

CX-1739 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

CX-717 - 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

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-GAA - 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

Feb 06, 2018: Audentes Therapeutics Announces Selection of Optimized Clinical Development Candidate for Pompe Disease Program

Sep 20, 2017: AVROBIO Expands Rare Disease Pipeline with Gene Therapy to Treat Pompe Disease

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

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

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

May 04, 2015: Cortex Pharmaceuticals Announces Publication of Research Results on the Use of Ampakines CX1739 in the Treatment of Respiratory Disorders

May 04, 2015: Cortex Pharmaceuticals Announces Publication of Research Results on the Use of Ampakines CX717 in the Treatment of Respiratory Disorders

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

Number of Products under Development for Pompe Disease, H1 2018

Number of Products under Development by Companies, H1 2018

Number of Products under Development by Universities/Institutes, H1 2018

Products under Development by Companies, H1 2018

Products under Development by Universities/Institutes, H1 2018

Number of Products by Stage and Target, H1 2018

Number of Products by Stage and Mechanism of Action, H1 2018

Number of Products by Stage and Route of Administration, H1 2018

Number of Products by Stage and Molecule Type, H1 2018

Pompe Disease - Pipeline by Amicus Therapeutics Inc, H1 2018

Pompe Disease - Pipeline by Audentes Therapeutics Inc, H1 2018

Pompe Disease - Pipeline by EpiVax Inc, H1 2018

Pompe Disease - Pipeline by Etubics Corp, H1 2018

Pompe Disease - Pipeline by Genzyme Corp, H1 2018

Pompe Disease - Pipeline by greenovation Biotech GmbH, H1 2018

Pompe Disease - Pipeline by JCR Pharmaceuticals Co Ltd, H1 2018

Pompe Disease - Pipeline by NanoMedSyn SAS, H1 2018

Pompe Disease - Pipeline by Oxyrane Belgium NV, H1 2018

Pompe Disease - Pipeline by Pharming Group NV, H1 2018

Pompe Disease - Pipeline by RespireRx Pharmaceuticals Inc, H1 2018

Pompe Disease - Pipeline by Sarepta Therapeutics Inc, H1 2018

Pompe Disease - Pipeline by Spark Therapeutics Inc, H1 2018

Pompe Disease - Dormant Projects, H1 2018

Pompe Disease - Discontinued Products, H1 2018

List of Figures

Number of Products under Development for Pompe Disease, H1 2018

Number of Products under Development by Companies, H1 2018

Number of Products under Development by Universities/Institutes, H1 2018

Number of Products by Targets, H1 2018

Number of Products by Stage and Targets, H1 2018

Number of Products by Mechanism of Actions, H1 2018

Number of Products by Stage and Mechanism of Actions, H1 2018

Number of Products by Routes of Administration, H1 2018

Number of Products by Stage and Routes of Administration, H1 2018

Number of Products by Molecule Types, H1 2018

Number of Products by Stage and Molecule Types, H1
Note: Product cover images may vary from those shown
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  • Amicus Therapeutics Inc
  • Audentes Therapeutics Inc
  • EpiVax Inc
  • Etubics Corp
  • Genzyme Corp
  • greenovation Biotech GmbH
  • JCR Pharmaceuticals Co Ltd
  • NanoMedSyn SAS
  • Oxyrane Belgium NV
  • Pharming Group NV
  • RespireRx Pharmaceuticals Inc
  • Sarepta Therapeutics Inc
  • Spark 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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