Pompe Disease R&D Pipeline Analysis Report, Q4 2020
Pompe Disease Pipeline Overview
The Q4 Pompe Disease pipeline report is part of the VPA Metabolic Disorder disease pipeline service. This quarterly review describes the pipeline of drugs under development for Pompe Disease, provides an update on the mechanism of action and targets, licensing and collaboration opportunities, company profiles including their business information, and market developments.
Pompe Disease Disease Types, Symptoms, Causes, and Available Treatment Options
The comprehensive report on the indication presents Pompe Disease disease overview, Pompe Disease types, Pompe Disease symptoms, causes, and FDA/EMA approved treatment options.
Pompe Disease Pipeline Trends and Insights
The year 2020 witnessed progress in innovations and the number of pipeline agents researched for Pompe Disease indication. The report presents near-term and long-term pipeline development trends and potential insights.
Pompe Disease Company Trends and Insights
The report analyzes the development progress, current status, investments, partnerships, and other developments of 18 companies. Business profiles and contact details of the companies actively perusing Pompe Disease pipeline are assessed.
Pompe Disease R&D Pipeline Development Phase Trends and Insights
From the discovery stage of development to phase 3 development, all the potential pipeline drug candidates are analyzed in the report. Pompe Disease discovery stage, research stage, Phase 1, phase 2, Phase 3, IND enabling studies are provided in the research.
Small Pompe Disease companies look for licensing and collaboration partners
High R&D costs and long duration pose challenges for small companies operating in the industry. Further, the presence of large companies and stringent regulations act as barriers for Pompe Disease pipeline development companies. Accordingly, a significant portion of small companies presents potential licensing and collaboration opportunities.
Pompe Disease Pipeline Market News and Developments during 2020
The Pompe Disease industry news, pipeline research, market developments including advancement in phases, pre-clinical study results, clinical trial launches, acquisitions and mergers, regulatory approvals, and others are included in the report.
Pompe Disease Mechanism of Action and Targets
Leading Mechanism of Action and targets being researched by all the companies and their progress is included. New mechanisms of actions introduced during the year, route of administration, orphan drug designation, and others are provided in the study.
New molecular entity details
Drug candidates identified as new molecular entities by FDA, EMA, and other regulators are included in the research.
Sources and Research Methodology
Data is updated daily across diseases in the infectious pipeline service. We review our quarterly pipeline reports for the latest 5 years to present longitudinal insights.
Scope and Coverage
The Q4 Pompe Disease pipeline report is part of the VPA Metabolic Disorder disease pipeline service. This quarterly review describes the pipeline of drugs under development for Pompe Disease, provides an update on the mechanism of action and targets, licensing and collaboration opportunities, company profiles including their business information, and market developments.
Pompe Disease Disease Types, Symptoms, Causes, and Available Treatment Options
The comprehensive report on the indication presents Pompe Disease disease overview, Pompe Disease types, Pompe Disease symptoms, causes, and FDA/EMA approved treatment options.
Pompe Disease Pipeline Trends and Insights
The year 2020 witnessed progress in innovations and the number of pipeline agents researched for Pompe Disease indication. The report presents near-term and long-term pipeline development trends and potential insights.
Pompe Disease Company Trends and Insights
The report analyzes the development progress, current status, investments, partnerships, and other developments of 18 companies. Business profiles and contact details of the companies actively perusing Pompe Disease pipeline are assessed.
Pompe Disease R&D Pipeline Development Phase Trends and Insights
From the discovery stage of development to phase 3 development, all the potential pipeline drug candidates are analyzed in the report. Pompe Disease discovery stage, research stage, Phase 1, phase 2, Phase 3, IND enabling studies are provided in the research.
Small Pompe Disease companies look for licensing and collaboration partners
High R&D costs and long duration pose challenges for small companies operating in the industry. Further, the presence of large companies and stringent regulations act as barriers for Pompe Disease pipeline development companies. Accordingly, a significant portion of small companies presents potential licensing and collaboration opportunities.
Pompe Disease Pipeline Market News and Developments during 2020
The Pompe Disease industry news, pipeline research, market developments including advancement in phases, pre-clinical study results, clinical trial launches, acquisitions and mergers, regulatory approvals, and others are included in the report.
Pompe Disease Mechanism of Action and Targets
Leading Mechanism of Action and targets being researched by all the companies and their progress is included. New mechanisms of actions introduced during the year, route of administration, orphan drug designation, and others are provided in the study.
New molecular entity details
Drug candidates identified as new molecular entities by FDA, EMA, and other regulators are included in the research.
Sources and Research Methodology
Data is updated daily across diseases in the infectious pipeline service. We review our quarterly pipeline reports for the latest 5 years to present longitudinal insights.
Scope and Coverage
- Pompe Disease pipeline development activities across the early phase, mid-stage, and late phase pipelines including therapeutic candidates
- Drug profiles comprising of drug overview, mechanism of action, co-developers, originators, route of administration, molecule type, orphan drug status, new molecular entity, area, and other details.
- Company-company partnerships, company-institute partnerships, and investment details of companies are included
- 18 companies are included including 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, JCR Pharmaceuticals Co Ltd, NanoMedSyn SAS, Oxyrane Belgium NV, Pharming Group NV, RespireRx Pharmaceuticals Inc, Sarepta Therapeutics Inc, Spark Therapeutics Inc, Valerion Therapeutics LLC,
- Disease overview, Pipeline trends, market analysis, and other developments
- Potential licensing/new business opportunities in Pompe Disease pipeline market
- Global coverage of companies and pipeline agents
- 2020 Trends, market analysis, and developments
- Potential growth opportunities
- Comprehensive details of drug candidates
- Drive pipeline research and commercial assessment
- Assess most promising drug candidates and stay ahead of the competition
- Strengthen your pipeline through identifying business expansion and acquisition opportunities
- Develop new candidates based on most focused targets and mechanism of actions
1. TABLE OF CONTENTS
1.1 List of Tables
1.2 List of Figures
2. POMPE DISEASE PIPELINE TRENDS AND INSIGHTS
2.1 Dominant Phase type of Pompe Disease Pipeline, 2020
2.2 Most focused Mechanism of Action in Pompe Disease Pipeline
2.3 Leading type of Route of Administration during 2020
2.4 Proportion of New Molecular Entities in Pompe Disease pipeline
2.5 Active Companies Developing Pompe Disease pipeline
3. POMPE DISEASE DISEASE TYPES, SYMPTOMS, CAUSES, AND AVAILABLE TREATMENT OPTIONS
3.1 Disease Overview
3.2 Types
3.3 Symptoms
3.4 Approved Treatment Options
4. POMPE DISEASE PIPELINE COMPANY TRENDS AND INSIGHTS - BUSINESS PROFILES, DRUG CANDIDATES AND CONTACTS
4.1 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
JCR Pharmaceuticals Co Ltd
NanoMedSyn SAS
Oxyrane Belgium NV
Pharming Group NV
RespireRx Pharmaceuticals Inc
Sarepta Therapeutics Inc
Spark Therapeutics Inc
Valerion Therapeutics LLC
5. POMPE DISEASE PIPELINE DRUG PROFILES
5.1 Current Status of Drug and Vaccine Candidates
5.2 Drug Snapshot
5.2.1 Drug
5.2.2 Mechanism of Action
5.2.3 Phase
5.2.4 Co-Developer
5.2.5 Originator
5.2.6 Synonym
5.2.7 Route of Administration
5.2.8 Type of Molecule
5.2.9 Orphan Drug Status
5.2.10 New Molecular Entity
5.2.11 Area
5.3 Drug Details
5.4 Mechanism of Action
5.5 Licensing/Collaboration Agreements
5.6 Clinical Trial Details
6. POMPE DISEASE PIPELINE MARKET NEWS AND DEVELOPMENTS DURING 2020
7. APPENDIX
7.1 About the Publisher
7.2 Sources and Research Methodology
1.1 List of Tables
1.2 List of Figures
2. POMPE DISEASE PIPELINE TRENDS AND INSIGHTS
2.1 Dominant Phase type of Pompe Disease Pipeline, 2020
2.2 Most focused Mechanism of Action in Pompe Disease Pipeline
2.3 Leading type of Route of Administration during 2020
2.4 Proportion of New Molecular Entities in Pompe Disease pipeline
2.5 Active Companies Developing Pompe Disease pipeline
3. POMPE DISEASE DISEASE TYPES, SYMPTOMS, CAUSES, AND AVAILABLE TREATMENT OPTIONS
3.1 Disease Overview
3.2 Types
3.3 Symptoms
3.4 Approved Treatment Options
4. POMPE DISEASE PIPELINE COMPANY TRENDS AND INSIGHTS - BUSINESS PROFILES, DRUG CANDIDATES AND CONTACTS
4.1 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
JCR Pharmaceuticals Co Ltd
NanoMedSyn SAS
Oxyrane Belgium NV
Pharming Group NV
RespireRx Pharmaceuticals Inc
Sarepta Therapeutics Inc
Spark Therapeutics Inc
Valerion Therapeutics LLC
5. POMPE DISEASE PIPELINE DRUG PROFILES
5.1 Current Status of Drug and Vaccine Candidates
5.2 Drug Snapshot
5.2.1 Drug
5.2.2 Mechanism of Action
5.2.3 Phase
5.2.4 Co-Developer
5.2.5 Originator
5.2.6 Synonym
5.2.7 Route of Administration
5.2.8 Type of Molecule
5.2.9 Orphan Drug Status
5.2.10 New Molecular Entity
5.2.11 Area
5.3 Drug Details
5.4 Mechanism of Action
5.5 Licensing/Collaboration Agreements
5.6 Clinical Trial Details
6. POMPE DISEASE PIPELINE MARKET NEWS AND DEVELOPMENTS DURING 2020
7. APPENDIX
7.1 About the Publisher
7.2 Sources and Research Methodology
