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Pompe Disease (Musculoskeletal) - Drugs In Development, 2021

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    Report

  • 121 Pages
  • August 2021
  • Region: Global
  • Global Markets Direct
  • ID: 5438183
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The publisher's latest Pharmaceutical and Healthcare disease pipeline guide 'Pompe Disease - Drugs In Development, 2021', 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 report 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 Pre-Registration, Phase II, Preclinical and Discovery stages are 2, 3, 20 and 2 respectively. Similarly, the Universities portfolio in Phase II, Phase I, Preclinical and Discovery stages comprises 1, 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 the publisher’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 the 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 portfolios 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 their 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

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
  • Number of Products under Development for Pompe Disease, 2021
  • Number of Products under Development by Companies, 2021
  • Number of Products under Development by Universities/Institutes, 2021
  • Products under Development by Companies, 2021
  • Products under Development by Universities/Institutes, 2021
  • Number of Products by Stage and Target, 2021
  • Number of Products by Stage and Mechanism of Action, 2021
  • Number of Products by Stage and Route of Administration, 2021
  • Number of Products by Stage and Molecule Type, 2021
  • Pompe Disease - Pipeline by AavantiBio Inc, 2021
  • Pompe Disease - Pipeline by Amicus Therapeutics Inc, 2021
  • Pompe Disease - Pipeline by Asklepios BioPharmaceutical Inc, 2021
  • Pompe Disease - Pipeline by Astellas Gene Therapies, 2021
  • Pompe Disease - Pipeline by Avidity Biosciences Inc, 2021
  • Pompe Disease - Pipeline by AVROBIO Inc, 2021
  • Pompe Disease - Pipeline by CANbridge Life Sciences Ltd, 2021
  • Pompe Disease - Pipeline by Denali Therapeutics Inc, 2021
  • Pompe Disease - Pipeline by eleva GmbH, 2021
  • Pompe Disease - Pipeline by EpiVax Inc, 2021
  • Pompe Disease - Pipeline by Etubics Corp, 2021
  • Pompe Disease - Pipeline by Genzyme Corp, 2021
  • Pompe Disease - Pipeline by Icagen Inc, 2021
  • Pompe Disease - Pipeline by JCR Pharmaceuticals Co Ltd, 2021
  • Pompe Disease - Pipeline by M6P Therapeutics, 2021
  • Pompe Disease - Pipeline by Maze Therapeutics Inc, 2021
  • Pompe Disease - Pipeline by NanoMedSyn SAS, 2021
  • Pompe Disease - Pipeline by Oxyrane Belgium NV, 2021
  • Pompe Disease - Pipeline by Pharming Group NV, 2021
  • Pompe Disease - Pipeline by Provention Bio Inc, 2021
  • Pompe Disease - Pipeline by RespireRx Pharmaceuticals Inc, 2021
  • Pompe Disease - Pipeline by Sarepta Therapeutics Inc, 2021
  • Pompe Disease - Pipeline by Spark Therapeutics Inc, 2021
  • Pompe Disease - Pipeline by Takeda Pharmaceutical Co Ltd, 2021
  • Pompe Disease - Dormant Projects, 2021
  • Pompe Disease - Discontinued Products, 2021

List of Figures
  • Number of Products under Development for Pompe Disease, 2021
  • Number of Products under Development by Companies, 2021
  • Number of Products under Development by Universities/Institutes, 2021
  • Number of Products by Targets, 2021
  • Number of Products by Stage and Targets, 2021
  • Number of Products by Mechanism of Actions, 2021
  • Number of Products by Stage and Mechanism of Actions, 2021
  • Number of Products by Routes of Administration, 2021
  • Number of Products by Stage and Routes of Administration, 2021
  • Number of Products by Molecule Types, 2021
  • Number of Products by Stage and Molecule Types, 2021

Companies Mentioned

A selection of companies mentioned in this report includes:

  • AavantiBio Inc
  • Amicus Therapeutics Inc
  • Asklepios BioPharmaceutical Inc
  • Astellas Gene Therapies
  • Avidity Biosciences Inc
  • AVROBIO Inc
  • CANbridge Life Sciences Ltd
  • Denali Therapeutics Inc
  • eleva GmbH
  • EpiVax Inc
  • Etubics Corp
  • Genzyme Corp
  • Icagen Inc
  • JCR Pharmaceuticals Co Ltd
  • M6P Therapeutics
  • Maze Therapeutics Inc
  • NanoMedSyn SAS
  • Oxyrane Belgium NV
  • Pharming Group NV
  • Provention Bio Inc
  • RespireRx Pharmaceuticals Inc
  • Sarepta Therapeutics Inc
  • Spark Therapeutics Inc
  • Takeda Pharmaceutical Co Ltd