Pompe Disease – Pipeline Review, H1 2020

Global Markets Direct’s, ‘Pompe Disease – Pipeline Review, H1 2020’, 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

Abeona Therapeutics Inc

Actus Therapeutics Inc

Amicus Therapeutics Inc

Audentes Therapeutics Inc

Avidity Biosciences LLC

AVROBIO Inc

EpiVax Inc

Etubics Corp

Genzyme Corp

Greenovation Biotech GmbH

Icagen Inc

JCR Pharmaceuticals Co Ltd

NanoMedSyn SAS

Oxyrane Belgium NV

Pharming Group NV

RespireRx Pharmaceuticals Inc

Sarepta Therapeutics Inc

Spark Therapeutics Inc

Valerion Therapeutics LLC

Table of Contents

Table of Contents

Introduction

Pompe Disease - Overview

Pompe Disease - Therapeutics Development

Pompe Disease - Therapeutics Assessment

Pompe Disease - Companies Involved in Therapeutics Development

Pompe Disease - Drug Profiles

Pompe Disease - Dormant Projects

Pompe Disease - Discontinued Products

Pompe Disease - Product Development Milestones

Appendix

List of Tables

List of Tables

Number of Products under Development for Pompe Disease, H1 2020

Number of Products under Development by Companies, H1 2020

Number of Products under Development by Universities/Institutes, H1 2020

Products under Development by Companies, H1 2020

Products under Development by Companies, H1 2020 (Contd..1), H1 2020

Products under Development by Universities/Institutes, H1 2020

Number of Products by Stage and Target, H1 2020

Number of Products by Stage and Mechanism of Action, H1 2020

Number of Products by Stage and Route of Administration, H1 2020

Number of Products by Stage and Molecule Type, H1 2020

Pompe Disease – Pipeline by Abeona Therapeutics Inc, H1 2020

Pompe Disease – Pipeline by Actus Therapeutics Inc, H1 2020

Pompe Disease – Pipeline by Amicus Therapeutics Inc, H1 2020

Pompe Disease – Pipeline by Audentes Therapeutics Inc, H1 2020

Pompe Disease – Pipeline by Avidity Biosciences LLC, H1 2020

Pompe Disease – Pipeline by AVROBIO Inc, H1 2020

Pompe Disease – Pipeline by EpiVax Inc, H1 2020

Pompe Disease – Pipeline by Etubics Corp, H1 2020

Pompe Disease – Pipeline by Genzyme Corp, H1 2020

Pompe Disease – Pipeline by Greenovation Biotech GmbH, H1 2020

Pompe Disease – Pipeline by Icagen Inc, H1 2020

Pompe Disease – Pipeline by JCR Pharmaceuticals Co Ltd, H1 2020

Pompe Disease – Pipeline by NanoMedSyn SAS, H1 2020

Pompe Disease – Pipeline by Oxyrane Belgium NV, H1 2020

Pompe Disease – Pipeline by Pharming Group NV, H1 2020

Pompe Disease – Pipeline by RespireRx Pharmaceuticals Inc, H1 2020

Pompe Disease – Pipeline by Sarepta Therapeutics Inc, H1 2020

Pompe Disease – Pipeline by Spark Therapeutics Inc, H1 2020

Pompe Disease – Pipeline by Valerion Therapeutics LLC, H1 2020

Pompe Disease – Dormant Projects, H1 2020

Pompe Disease – Discontinued Products, H1 2020

List of Figures

List of Figures

Number of Products under Development for Pompe Disease, H1 2020

Number of Products under Development by Companies, H1 2020

Number of Products under Development by Universities/Institutes, H1 2020

Number of Products by Targets, H1 2020

Number of Products by Stage and Targets, H1 2020

Number of Products by Mechanism of Actions, H1 2020

Number of Products by Stage and Mechanism of Actions, H1 2020

Number of Products by Routes of Administration, H1 2020

Number of Products by Stage and Routes of Administration, H1 2020

Number of Products by Molecule Types, H1 2020

Number of Products by Stage and Top 10 Molecule Types, H1 2020

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