Mucopolysaccharidosis I (Hurler Syndrome) Pipeline Analysis 2018 (H1) - Focusing on Clinical Trials and Results, Drug Profiling, Patents, Collaborations, and Other Recent Developments

  • Published : Mar 2020
  • Report Code : KSI061611396
  • Pages : 114
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Mucopolysaccharidosis I (Hurler Syndrome) Pipeline Analysis 2018 – Focusing on Clinical Trials and Results, Drug Profiling, Patents, Collaborations, and Other Developments.

Mucopolysaccharidosis I (Hurler Syndrome) Pipeline Analysis report covers 11 drugs currently in different phases of development. Hurler Syndrome is an inherited lysosomal storage disease caused by defect in IDUA gene. The defect in the gene results in deficiency of alpha-L iduronidase enzyme which helps in helps break down long chains of sugar molecules called glycosaminoglycans. Without the enzyme glycosaminoglycans build up and damage of tissues and organs. The disease is characterised by skeletal abnormalities, cognitive impairment, heart disease, respiratory problems, enlarged liver and spleen, characteristic facies and reduced life expectancy.

The report provides Mucopolysaccharidosis I (Hurler Syndrome) treatment drugs by company, phases of development including products in early discovery stage and NDA filing, molecule type, route of administration and region. The report will help to evaluate the collaboration, in-licensing and out-licensing opportunities, formulating business development strategies and tracking the activities of the key market players. Epidemiology, major drivers, restraints, and opportunities have been covered to provide an exhaustive picture of the market. The analysis presents in-depth information regarding the development, trends, and industry policies and regulations implemented to offer stakeholders a better understanding of the key factors affecting the overall market environment.

Various databases (for patents and Clinical Trials), studies and data published by industry associations, analyst reports, investor presentations, press releases and journals among others have been taken into consideration while conducting the secondary research.

Market intelligence is presented in the form of analysis, charts, and graphics to help the clients in gaining faster and efficient understanding of the market.

Major industry players profiled as part of the report are ArmaGen,  Angiochem and JCR Pharmaceuticals Co Ltd among others.

Scope:

By Company

By Phase

By Molecule Type

By Region

By Route of Administration

1. Introduction
1.1. Research Methodology
1.2. Research Scope
1.2.1. Analysis by Company
1.2.2. Analysis by Phase
1.2.3. By Molecule Type
1.2.4. By region
2. Disease Overview
2.1.1. Introduction
2.1.2. Classification
2.1.3. Symptoms
2.1.4. Causes
2.1.5. Diagnoses
2.1.6. Treatment
2.1.7. Epidemiology
3. Executive Summary
4. Market Dynamics
4.1. Drivers
4.2. Restraints
4.3. Opportunities
5. Pipeline Analysis/Outlook
5.1. Analysis by Company
5.2. Analysis by Phase
5.2.1. Phase of Development
5.2.1.1. Introduction
5.2.1.1.1. Drug Profiling
• Drug Name
• Generic Name
• Synonyms
• Company
• Collaborator
• Route of administration
• Target 
• Mechanism of Action
• Technology
• Molecule type
• CAS Number
• Weight
• Chemical Formula
• IUPAC name
• ATC code
5.2.1.1.2. Strategic Developments
5.2.1.1.3. Clinical trials
5.2.1.1.4. Clinical trial results
5.2.1.1.5. Patents
5.2.1.1.6. Technology
5.2.2. Comparative Analysis for Trials by Phase (Pie, Bar graph)
5.3. By Molecule type
5.4. Analysis by Region
6. Company profiling
6.1. ArmaGen
6.1.1. Introduction
6.1.2. Financials
6.1.3. Products and Services
6.1.4. SWOT
6.2. Immusoft
6.2.1. Introduction
6.2.2. Financials
6.2.3. Products and Services
6.2.4. SWOT
6.3. Sangamo Therapeutics, Inc.
6.3.1. Introduction
6.3.2. Financials
6.3.3. Products and Services
6.3.4. SWOT
6.4. Eloxx Pharmaceuticals
6.4.1. Introduction
6.4.2. Financials
6.4.3. Products and Services
6.4.4. SWOT
6.5. REGENXBIO Inc.
6.5.1. Introduction
6.5.2. Financials
6.5.3. Products and Services
6.5.4. SWOT
6.6. Angiochem
6.6.1. Introduction
6.6.2. Financials
6.6.3. Products and Services
6.6.4. SWOT
6.7. Crispr Therapeutics
6.7.1. Introduction
6.7.2. Financials
6.7.3. Products and Services
6.7.4. SWOT
6.8. Jupiter Orphan Therapeutics, Inc.
6.8.1. Introduction
6.8.2. Financials
6.8.3. Products and Services
6.8.4. SWOT
6.9. Orchard Therapeutics
6.9.1. Introduction
6.9.2. Financials
6.9.3. Products and Services
6.9.4. SWOT
6.10. ProQR Therapeutics
6.10.1. Introduction
6.10.2. Financials
6.10.3. Products and Services
6.10.4. SWOT
6.11. JCR Pharmaceuticals Co Ltd
6.11.1. Introduction
6.11.2. Financials
6.11.3. Products and Services
6.11.4. SWOT
ArmaGen
Immusoft
Sangamo Therapeutics, Inc.
Eloxx Pharmaceuticals
REGENXBIO Inc.
Angiochem
Crispr Therapeutics
Jupiter Orphan Therapeutics, Inc.
Orchard Therapeutics
ProQR Therapeutics
JCR Pharmaceuticals Co Ltd

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