Pompe Disease - Pipeline Review, H2 2018

Global Markets Direct’s, ‘Pompe Disease - Pipeline Review, H2 2018’, provides an overview of the Pompe Disease pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Pompe Disease, complete with analysis by stage of development, drug target, 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 therapeutic development for Pompe Disease and features dormant and discontinued projects.

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.

Scope

- The report provides a snapshot of the global therapeutic landscape of Pompe Disease

- The report reviews pipeline therapeutics for Pompe Disease 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 Pompe Disease therapeutics and enlists all their major and minor projects

- The report assesses Pompe Disease therapeutics based on drug target, 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 related to pipeline therapeutics for Pompe Disease

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 important and diverse types of therapeutics under development for Pompe Disease

- 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 Pompe Disease 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

- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline

Companies mentioned

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 ...

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

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Pompe Disease - Overview

Pompe Disease - Therapeutics Development

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

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

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 ...

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

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 ...

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

    Pricing

Discounts available for multiple report purchases.

reportstore@globalmarketsdirect.com
+44 (0) 161 359 5414

Saved reports