Summary
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Pompe Disease - Pipeline Review, H2 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
Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Pompe Disease - Pipeline Review, H2 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, IND/CTA Filed and Preclinical stages are 1, 2, 1, 1 and 12 respectively. Similarly, the Universities portfolio in Phase II, Phase I and Preclinical stages comprises 1, 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 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.
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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
Avrobio 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
(alglucosidase alfa + miglustat) - 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 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
Gene Therapy to Activate GAA 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-3006 - 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
Oct 08, 2018: Spark Therapeutics Announces New Preclinical Data for Pompe Disease Gene Therapy Candidate
Sep 24, 2018: Audentes Therapeutics provides update on AT982 for Pompe disease
Apr 30, 2018: Audentes Therapeutics Announces Data on AT982 at the 21st Annual Meeting of the American Society of Gene and Cell Therapy
Apr 26, 2018: AskBio Spins Out New Gene Therapy Company, Actus Therapeutics
Feb 07, 2018: Sanofi Announces New Safety Data for Investigational Avalglucosidase Alfa in Patients with Pompe Disease
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, H2 2018
Number of Products under Development by Companies, H2 2018
Number of Products under Development by Universities/Institutes, H2 2018
Products under Development by Companies, H2 2018
Products under Development by Universities/Institutes, H2 2018
Number of Products by Stage and Target, H2 2018
Number of Products by Stage and Mechanism of Action, H2 2018
Number of Products by Stage and Route of Administration, H2 2018
Number of Products by Stage and Molecule Type, H2 2018
Pompe Disease - Pipeline by Amicus Therapeutics Inc, H2 2018
Pompe Disease - Pipeline by Audentes Therapeutics Inc, H2 2018
Pompe Disease - Pipeline by Avrobio Inc, H2 2018
Pompe Disease - Pipeline by EpiVax Inc, H2 2018
Pompe Disease - Pipeline by Etubics Corp, H2 2018
Pompe Disease - Pipeline by Genzyme Corp, H2 2018
Pompe Disease - Pipeline by greenovation Biotech GmbH, H2 2018
Pompe Disease - Pipeline by JCR Pharmaceuticals Co Ltd, H2 2018
Pompe Disease - Pipeline by NanoMedSyn SAS, H2 2018
Pompe Disease - Pipeline by Oxyrane Belgium NV, H2 2018
Pompe Disease - Pipeline by Pharming Group NV, H2 2018
Pompe Disease - Pipeline by RespireRx Pharmaceuticals Inc, H2 2018
Pompe Disease - Pipeline by Sarepta Therapeutics Inc, H2 2018
Pompe Disease - Pipeline by Spark Therapeutics Inc, H2 2018
Pompe Disease - Dormant Projects, H2 2018
Pompe Disease - Discontinued Products, H2 2018
List of Figures
Number of Products under Development for Pompe Disease, H2 2018
Number of Products under Development by Companies, H2 2018
Number of Products under Development by Universities/Institutes, H2 2018
Number of Products by Targets, H2 2018
Number of Products by Stage and Targets, H2 2018
Number of Products by Mechanism of Actions, H2 2018
Number of Products by Stage and Mechanism of Actions, H2 2018
Number of Products by Routes of Administration, H2 2018
Number of Products by Stage and Top 10 Routes of Administration, H2 2018
Number of Products by Molecule Types, H2 2018
Number of Products by Stage and Molecule Types, H2 2018
