Adrenoleukodystrophy (ALD) Pipeline Analysis 2019 – Focusing on Clinical Trials and Results, Drug Profiling, Patents, Collaborations, and Other Recent Developments

Adrenoleukodystrophy is a rare genetic disorder characterised by loss or damage of myelin sheath surrounding and insulating the nerve cells. The damage of the myelin sheath further leads to the progressive loss of the fuctioning of the adrenal glands. Symptoms of adrenoleukodystrophy are muscle spasms and pain, impaired vision, trouble balancing the body, deterioration of motor control, Adrenal insufficiency and lethargy and clumsiness. Diagnosis is done using blood tests, Skin biopsy and fibroblast cell culture and imaging tests.

The report provides ALD 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 Applied Genetic Technologies Corporation, Magenta Therapeutics, Orpheris, Inc. and Vertex Pharmaceuticals Incorporated 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. MINORYX THERAPEUTICS SL.

6.1.1. Introduction

6.1.2. Financials

6.1.3. Products and Services

6.1.4. SWOT

6.2. bluebird bio, Inc

6.2.1. Introduction

6.2.2. Financials

6.2.3. Products and Services

6.2.4. SWOT

6.3. Viking Therapeutics

6.3.1. Introduction

6.3.2. Financials

6.3.3. Products and Services

6.3.4. SWOT

6.4. Orpheris, Inc.

6.4.1. Introduction

6.4.2. Financials

6.4.3. Products and Services

6.4.4. SWOT

6.5. Vertex Pharmaceuticals Incorporated

6.5.1. Introduction

6.5.2. Financials

6.5.3. Products and Services

6.5.4. SWOT

6.6. SOM Biotech

6.6.1. Introduction

6.6.2. Financials

6.6.3. Products and Services

6.6.4. SWOT

6.7. Neurovia, Inc.

6.7.1. Introduction

6.7.2. Financials

6.7.3. Products and Services

6.7.4. SWOT

6.8. MedDay Pharmaceuticals SA

6.8.1. Introduction

6.8.2. Financials

6.8.3. Products and Services

6.8.4. SWOT

6.9. Applied Genetic Technologies Corporation

6.9.1. Introduction

6.9.2. Financials

6.9.3. Products and Services

6.9.4. SWOT

6.10. Poxel SA

6.10.1. Introduction

6.10.2. Financials

6.10.3. Products and Services

6.10.4. SWOT

6.11. Magenta Therapeutic

6.11.1. Introduction

6.11.2. Financials

6.11.3. Products and Services

6.11.4. SWOT

List of Tables

List of Figures

MINORYX THERAPEUTICS SL.

bluebird bio, Inc

Viking Therapeutics

Orpheris, Inc.

Vertex Pharmaceuticals Incorporated

SOM Biotech

Neurovia, Inc.

MedDay Pharmaceuticals SA

Applied Genetic Technologies Corporation

Poxel SA

Magenta Therapeutic