Canavan Disease - Pipeline Review, H2 2017

Canavan Disease - Pipeline Review, H2 2017

Summary

Global Markets Directs latest Pharmaceutical and Healthcare disease pipeline guide Canavan Disease - Pipeline Review, H2 2017, provides an overview of the Canavan Disease (Central Nervous System) pipeline landscape.

Canavan disease is a gene-linked neurological disorder in which the brain degenerates into spongy tissue riddled with microscopic fluid-filled spaces. Cause includes a mutation in the gene which directs the production of the enzyme aspartoacylase (ASPA) allows the buildup of N-acetylaspartic acid (NAA) in the brain. The buildup of NAA causes damage to myelin. Symptoms include lack of motor development, feeding difficulties, abnormal muscle tone, and an abnormally large, poorly controlled head, paralysis, blindness, or hearing loss.

Report Highlights

Global Markets Directs Pharmaceutical and Healthcare latest pipeline guide Canavan Disease - Pipeline Review, H2 2017, provides comprehensive information on the therapeutics under development for Canavan Disease (Central Nervous System), 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 Canavan Disease (Central Nervous System) pipeline guide also reviews of key players involved in therapeutic development for Canavan Disease and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase II and Preclinical stages are 1 and 2 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 3 molecules, respectively.

Canavan Disease (Central Nervous System) 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 Directs 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 Canavan Disease (Central Nervous System).
- The pipeline guide reviews pipeline therapeutics for Canavan Disease (Central Nervous System) 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 Canavan Disease (Central Nervous System) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Canavan Disease (Central Nervous System) 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 Canavan Disease (Central Nervous System)

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 Canavan Disease (Central Nervous System).
- 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 its most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Canavan Disease (Central Nervous System) 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.

2
List of Tables 3
List of Figures 3
Introduction 4
Global Markets Direct Report Coverage 4
Canavan Disease - Overview 5
Canavan Disease - Therapeutics Development 6
Pipeline Overview 6
Pipeline by Companies 7
Pipeline by Universities/Institutes 8
Products under Development by Companies 9
Products under Development by Universities/Institutes 10
Canavan Disease - Therapeutics Assessment 11
Assessment by Target 11
Assessment by Mechanism of Action 12
Assessment by Molecule Type 14
Canavan Disease - Companies Involved in Therapeutics Development 16
Pfizer Inc 16
Turing Pharmaceuticals AG 16
Canavan Disease - Drug Profiles 17
Gene Therapy 2 to Activate Aspartoacylase for Canavan Disease - Drug Profile 17
Product Description 17
Mechanism Of Action 17
R&D Progress 17
Gene therapy to Activate Aspartoacylase for Canavan Disease - Drug Profile 18
Product Description 18
Mechanism Of Action 18
R&D Progress 18
Gene Therapy to Activate Aspartoacylase for Canavan Disease - Drug Profile 19
Product Description 19
Mechanism Of Action 19
R&D Progress 19
Recombinant Enzyme to Replace Aspartoacylase for Canavan Disease - Drug Profile 20
Product Description 20
Mechanism Of Action 20
R&D Progress 20
Stem Cell Therapy to Activate Aspartoacylase for Canavan Disease - Drug Profile 21
Product Description 21
Mechanism Of Action 21
R&D Progress 21
TUR-007 - Drug Profile 22
Product Description 22
Mechanism Of Action 22
R&D Progress 22
Canavan Disease - Dormant Projects 23
Canavan Disease - Discontinued Products 24
Canavan Disease - Product Development Milestones 25
Featured News & Press Releases 25
Nov 09, 2017: New gene therapy developed with UNSW researchers targets brain disorders 25
Appendix 26
Methodology 26
Coverage 26
Secondary Research 26
Primary Research 26
Expert Panel Validation 26
Contact Us 26
Disclaimer 27

List Of Tables

List of Tables
Number of Products under Development for Canavan Disease, H2 2017 6
Number of Products under Development by Companies, H2 2017 7
Number of Products under Development by Universities/Institutes, H2 2017 8
Products under Development by Companies, H2 2017 9
Products under Development by Universities/Institutes, H2 2017 10
Number of Products by Stage and Target, H2 2017 11
Number of Products by Stage and Mechanism of Action, H2 2017 13
Number of Products by Stage and Molecule Type, H2 2017 15
Canavan Disease - Pipeline by Pfizer Inc, H2 2017 16
Canavan Disease - Pipeline by Turing Pharmaceuticals AG, H2 2017 16
Canavan Disease - Dormant Projects, H2 2017 23
Canavan Disease - Discontinued Products, H2 2017 24

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