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Gaucher Disease-Pipeline Review, H2 2017

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Restricted to one authorized user; Open on 1 PC/ mobile / Tablet only.
Print Option: Yes
Copy Option: No
Deliverable Format : PDF Via Email

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Gaucher Disease-Pipeline Review, H2 2017

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Gaucher Disease-Pipeline Review, H2 2017, 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, H2 2017, 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 3, 1, 9 and 1 respectively. Similarly, the Universities portfolio in Phase I, Preclinical and Discovery stages comprises 1, 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'sproprietary 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'smost 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.

READ MORE

Table Of Content

Scope

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

Genzyme Corp

JCR Pharmaceuticals Co Ltd

Johnson & Johnson

Lixte Biotechnology Holdings Inc

Orphazyme ApS

Pharming Group NV

Protalix BioTherapeutics Inc

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

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

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

PRX-112-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Recombinant Beta Glucosidase 2 Replacement for Gaucher's Disease and Male Infertility-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Recombinant Beta-Cerebrosidase Replacement for Gaucher's Disease-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 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-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

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

Jun 18, 2014: Protalix BioTherapeutics Initiates Phase II Study With PRX-112, an Orally-Administered Enzyme Replacement Therapy for the Treatment of Gaucher Disease

Feb 12, 2014: Protalix BioTherapeutics Announces Oral GCD Data to be Presented at WORLD Symposium 2014

Oct 14, 2013: Protalix BioTherapeutics Announces Positive Phase I Clinical Trial Results for Oral GCD in Gaucher Disease Patients

Apr 02, 2013: Protalix BioTherapeutics Treats First Gaucher Patient In Phase I Study With PRX-112

Mar 07, 2013: Protalix BioTherapeutics Receives Approval From Israeli Ministry Of Health To Initiate Phase I Clinical Trial Of PRX-112 For Treatment Of Gaucher Disease

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 Figure

List of Figures

Number of Products under Development for Gaucher Disease, H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products under Development by Universities/Institutes, H2 2017

Number of Products by Targets, H2 2017

Number of Products by Stage and Targets, H2 2017

Number of Products by Mechanism of Actions, H2 2017

Number of Products by Stage and Mechanism of Actions, H2 2017

Number of Products by Routes of Administration, H2 2017

Number of Products by Stage and Routes of Administration, H2 2017

Number of Products by Molecule Types, H2 2017

Number of Products by Stage and Molecule Types, H2 2017


List Of Table

List of Tables

Number of Products under Development for Gaucher Disease, H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products under Development by Universities/Institutes, H2 2017

Products under Development by Companies, H2 2017

Products under Development by Universities/Institutes, H2 2017

Number of Products by Stage and Target, H2 2017

Number of Products by Stage and Mechanism of Action, H2 2017

Number of Products by Stage and Route of Administration, H2 2017

Number of Products by Stage and Molecule Type, H2 2017

Gaucher Disease-Pipeline by Genzyme Corp, H2 2017

Gaucher Disease-Pipeline by JCR Pharmaceuticals Co Ltd, H2 2017

Gaucher Disease-Pipeline by Johnson & Johnson, H2 2017

Gaucher Disease-Pipeline by Lixte Biotechnology Holdings Inc, H2 2017

Gaucher Disease-Pipeline by Orphazyme ApS, H2 2017

Gaucher Disease-Pipeline by Pharming Group NV, H2 2017

Gaucher Disease-Pipeline by Protalix BioTherapeutics Inc, H2 2017

Gaucher Disease-Dormant Projects, H2 2017

Gaucher Disease-Dormant Projects, H2 2017 (Contd..1), H2 2017

Gaucher Disease-Discontinued Products, H2 2017

Licence Rights

Single User License:
Report can be used by individual purchaser only

Site License: 
Report can be shared by unlimited users within one corporate location, e.g. a regional office

Corporate User License:  
Report can be shared globally by unlimited users within the purchasing corporation e.g. all employees of a single company

Section Purchase

To know more information on Purchase by Section, please send a mail to support@kenresearch.com

Products and Companies


Companies

Genzyme Corp

JCR Pharmaceuticals Co Ltd

Johnson & Johnson

Lixte Biotechnology Holdings Inc

Orphazyme ApS

Pharming Group NV

Protalix BioTherapeutics Inc

Gaucher Disease-Pipeline Review, H2 2017

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Gaucher Disease-Pipeline Review, H2 2017, 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, H2 2017, 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 3, 1, 9 and 1 respectively. Similarly, the Universities portfolio in Phase I, Preclinical and Discovery stages comprises 1, 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'sproprietary 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'smost 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.

READ MORE

Scope

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

Genzyme Corp

JCR Pharmaceuticals Co Ltd

Johnson & Johnson

Lixte Biotechnology Holdings Inc

Orphazyme ApS

Pharming Group NV

Protalix BioTherapeutics Inc

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

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

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

PRX-112-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Recombinant Beta Glucosidase 2 Replacement for Gaucher's Disease and Male Infertility-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Recombinant Beta-Cerebrosidase Replacement for Gaucher's Disease-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 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-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

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

Jun 18, 2014: Protalix BioTherapeutics Initiates Phase II Study With PRX-112, an Orally-Administered Enzyme Replacement Therapy for the Treatment of Gaucher Disease

Feb 12, 2014: Protalix BioTherapeutics Announces Oral GCD Data to be Presented at WORLD Symposium 2014

Oct 14, 2013: Protalix BioTherapeutics Announces Positive Phase I Clinical Trial Results for Oral GCD in Gaucher Disease Patients

Apr 02, 2013: Protalix BioTherapeutics Treats First Gaucher Patient In Phase I Study With PRX-112

Mar 07, 2013: Protalix BioTherapeutics Receives Approval From Israeli Ministry Of Health To Initiate Phase I Clinical Trial Of PRX-112 For Treatment Of Gaucher Disease

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 Figure

List of Figures

Number of Products under Development for Gaucher Disease, H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products under Development by Universities/Institutes, H2 2017

Number of Products by Targets, H2 2017

Number of Products by Stage and Targets, H2 2017

Number of Products by Mechanism of Actions, H2 2017

Number of Products by Stage and Mechanism of Actions, H2 2017

Number of Products by Routes of Administration, H2 2017

Number of Products by Stage and Routes of Administration, H2 2017

Number of Products by Molecule Types, H2 2017

Number of Products by Stage and Molecule Types, H2 2017


List Of Table

List of Tables

Number of Products under Development for Gaucher Disease, H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products under Development by Universities/Institutes, H2 2017

Products under Development by Companies, H2 2017

Products under Development by Universities/Institutes, H2 2017

Number of Products by Stage and Target, H2 2017

Number of Products by Stage and Mechanism of Action, H2 2017

Number of Products by Stage and Route of Administration, H2 2017

Number of Products by Stage and Molecule Type, H2 2017

Gaucher Disease-Pipeline by Genzyme Corp, H2 2017

Gaucher Disease-Pipeline by JCR Pharmaceuticals Co Ltd, H2 2017

Gaucher Disease-Pipeline by Johnson & Johnson, H2 2017

Gaucher Disease-Pipeline by Lixte Biotechnology Holdings Inc, H2 2017

Gaucher Disease-Pipeline by Orphazyme ApS, H2 2017

Gaucher Disease-Pipeline by Pharming Group NV, H2 2017

Gaucher Disease-Pipeline by Protalix BioTherapeutics Inc, H2 2017

Gaucher Disease-Dormant Projects, H2 2017

Gaucher Disease-Dormant Projects, H2 2017 (Contd..1), H2 2017

Gaucher Disease-Discontinued Products, H2 2017

Single User License:
Report can be used by individual purchaser only

Site License: 
Report can be shared by unlimited users within one corporate location, e.g. a regional office

Corporate User License:  
Report can be shared globally by unlimited users within the purchasing corporation e.g. all employees of a single company

To know more information on Purchase by Section, please send a mail to support@kenresearch.com

Companies

Genzyme Corp

JCR Pharmaceuticals Co Ltd

Johnson & Johnson

Lixte Biotechnology Holdings Inc

Orphazyme ApS

Pharming Group NV

Protalix BioTherapeutics Inc