Gaucher Disease - Pipeline Review, H1 2018

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Date: 12-Jun-2018
No. of pages: 61
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Gaucher Disease - Pipeline Review, H1 2018

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Gaucher Disease - Pipeline Review, H1 2018, provides an overview of the Gaucher Disease (Genetic Disorders) pipeline landscape.

Gaucher disease is a rare genetic disorder occurs due to lacks an enzyme called glucocerebrosidase. Symptoms include cognitive impairment, enlarged spleen, severe swelling, lung disease, fatigue and seizures. Treatment includes enzyme replacement therapy.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Gaucher Disease - Pipeline Review, H1 2018, provides comprehensive information on the therapeutics under development for Gaucher Disease (Genetic 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 Gaucher Disease (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Gaucher 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, Phase I, Preclinical and Discovery stages are 2, 3, 8 and 3 respectively. Similarly, the Universities portfolio in Phase I and Discovery stages comprises 1 and 5 molecules, respectively.

Gaucher Disease (Genetic 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 Gaucher Disease (Genetic Disorders).

- The pipeline guide reviews pipeline therapeutics for Gaucher Disease (Genetic 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 Gaucher Disease (Genetic Disorders) therapeutics and enlists all their major and minor projects.

- The pipeline guide evaluates Gaucher Disease (Genetic 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 Gaucher Disease (Genetic 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 Gaucher Disease (Genetic 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 Gaucher Disease (Genetic 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.

Gaucher Disease - Pipeline Review, H1 2018

Table of Contents
List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Gaucher Disease - Overview
Gaucher Disease - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Gaucher Disease - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Gaucher Disease - Companies Involved in Therapeutics Development
Bioasis Technologies Inc
Genzyme Corp
JCR Pharmaceuticals Co Ltd
Johnson & Johnson
Lixte Biotechnology Holdings Inc
Orphazyme A/S
Gaucher Disease - Drug Profiles
ADN-LYS - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
arimoclomol - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AVRRD-02 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Biologic to Activate Glucocerebrosidase for Genetic Disorders - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy for Gaucher Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy for Gaucher Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ibiglustat - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
imiglucerase biosimilar - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
LB-201 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
LB-205 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
LTI-291 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
miglustat - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NCGC-607 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Pcgin - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant Protein for Type I and Type III Gaucher's Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
RND-001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecule to Activate Beta Galactosidase for Gaucher Disease and GM1 Gangliosidosis - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Activate GBA for Gaucher Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Inhibit Glucocerebrosidase for Gaucher Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Inhibit Glucosidase for Diabetes and Gaucher's Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
taliglucerase alfa biosimilar - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
xB-3007 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gaucher Disease - Dormant Projects
Gaucher Disease - Discontinued Products
Gaucher Disease - Product Development Milestones
Featured News & Press Releases
Feb 06, 2018: Poster presentation at WORLDSymposium 2018 - arimoclomol as clinical candidate for treatment of Gaucher disease
Mar 22, 2017: AVROBIO Expands Rare Disease Pipeline with Gene Therapy to Treat Gaucher Disease
Oct 25, 2016: Asthma research unexpectedly yields new treatment approach for inherited enzyme disease
Jul 12, 2016: Researchers make advance in possible treatments for Gaucher, Parkinson's diseases
Dec 19, 2011: Lixte's LB-205 Shows Therapeutic Potential For Gaucher Disease
Nov 09, 2010: Actelion Receives Approval For Zavesca For Reimbursement Under Ontario Public Drug Program
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List of Tables
Number of Products under Development for Gaucher Disease, H1 2018
Number of Products under Development by Companies, H1 2018
Number of Products under Development by Universities/Institutes, H1 2018
Products under Development by Companies, H1 2018
Products under Development by Universities/Institutes, H1 2018
Number of Products by Stage and Target, H1 2018
Number of Products by Stage and Mechanism of Action, H1 2018
Number of Products by Stage and Route of Administration, H1 2018
Number of Products by Stage and Molecule Type, H1 2018
Gaucher Disease - Pipeline by Bioasis Technologies Inc, H1 2018
Gaucher Disease - Pipeline by Genzyme Corp, H1 2018
Gaucher Disease - Pipeline by JCR Pharmaceuticals Co Ltd, H1 2018
Gaucher Disease - Pipeline by Johnson & Johnson, H1 2018
Gaucher Disease - Pipeline by Lixte Biotechnology Holdings Inc, H1 2018
Gaucher Disease - Pipeline by Orphazyme A/S, H1 2018
Gaucher Disease - Dormant Projects, H1 2018
Gaucher Disease - Dormant Projects, H1 2018 (Contd..1), H1 2018
Gaucher Disease - Discontinued Products, H1 2018

List of Figures
Number of Products under Development for Gaucher Disease, H1 2018
Number of Products under Development by Companies, H1 2018
Number of Products under Development by Universities/Institutes, H1 2018
Number of Products by Targets, H1 2018
Number of Products by Stage and Targets, H1 2018
Number of Products by Mechanism of Actions, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Routes of Administration, H1 2018
Number of Products by Stage and Routes of Administration, H1 2018
Number of Products by Molecule Types, H1 2018
Number of Products by Stage and Molecule Types, H1 2018
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