Pompe Disease (Musculoskeletal) – Drugs In Development, 2021

Global Markets Direct'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

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Pompe Disease – Drugs In Development, 2021, 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 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.

Companies mentioned

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

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, 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

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

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