Crigler-Najjar Syndrome - Pipeline Review, H2 2017

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Date: 08-Aug-2017
No. of pages: 36
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Crigler-Najjar Syndrome - Pipeline Review, H2 2017

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Crigler-Najjar Syndrome - Pipeline Review, H2 2017, provides an overview of the Crigler-Najjar Syndrome (Genetic Disorders) pipeline landscape.

Crigler-Najjar syndrome is a severe condition characterized by high levels of a toxic substance called bilirubin in the blood (hyperbilirubinemia). Symptoms include yellow skin (jaundice) and yellow in the whites of the eyes (icterus). It occurs when the enzyme that normally converts bilirubin into a form that can easily be removed from the body does not work correctly. Treatment includes phototherapy, liver transplantation and blood transfusions.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Crigler-Najjar Syndrome - Pipeline Review, H2 2017, provides comprehensive information on the therapeutics under development for Crigler-Najjar Syndrome (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 Crigler-Najjar Syndrome (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Crigler-Najjar Syndrome and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase II, IND/CTA Filed and Preclinical stages are 1, 1 and 4 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 1 molecules, respectively.

Crigler-Najjar Syndrome (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 Crigler-Najjar Syndrome (Genetic Disorders).

- The pipeline guide reviews pipeline therapeutics for Crigler-Najjar Syndrome (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 Crigler-Najjar Syndrome (Genetic Disorders) therapeutics and enlists all their major and minor projects.

- The pipeline guide evaluates Crigler-Najjar Syndrome (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 Crigler-Najjar Syndrome (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 Crigler-Najjar Syndrome (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 Crigler-Najjar Syndrome (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.

Crigler-Najjar Syndrome - Pipeline Review, H2 2017

Table of Contents
List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Crigler-Najjar Syndrome - Overview
Crigler-Najjar Syndrome - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Crigler-Najjar Syndrome - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Crigler-Najjar Syndrome - Companies Involved in Therapeutics Development
Audentes Therapeutics Inc
Genethon SA
International Stem Cell Corp
Promethera Biosciences SA
Crigler-Najjar Syndrome - Drug Profiles
ALXN-1540 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ALXN-1630 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AT-342 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Glucuronosyltransferase for Crigler-Najjar Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate UGT1A1 for Crigler-Najjar Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
HepaStem - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Stem Cell Therapy for Alpha-Antitrypsin Deficiency, Crigler-Najjar Syndrome and Liver Cirrhosis - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Crigler-Najjar Syndrome - Product Development Milestones
Featured News & Press Releases
May 03, 2017: Audentes Therapeutics Announces Presentation of Data on at the American Society of Gene and Cell Therapy Annual Meeting
Mar 01, 2017: Audentes Therapeutics Announces Commencement of LUSTRO, a Clinical Assessment and Phase 1/2 Run-in Study of Patients with Crigler-Najjar Syndrome
Feb 01, 2017: Audentes Therapeutics Announces FDA Clearance of Investigational New Drug Application for AT342 to Treat Crigler-Najjar Syndrome
May 07, 2015: Evidence of efficacy of gene therapy in rodents affected by a rare genetic liver disease, Crigler-Najjar syndrome
Feb 12, 2013: International Stem Cell Demonstrates Positive Animal Efficacy Results In Metabolic Liver Disease Program
Jan 07, 2013: International Stem Cell Announces Positive Animal Efficacy Results In Liver Disease Program
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List of Tables
Number of Products under Development for Crigler-Najjar Syndrome, 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
Crigler-Najjar Syndrome - Pipeline by Audentes Therapeutics Inc, H2 2017
Crigler-Najjar Syndrome - Pipeline by Genethon SA, H2 2017
Crigler-Najjar Syndrome - Pipeline by International Stem Cell Corp, H2 2017
Crigler-Najjar Syndrome - Pipeline by Promethera Biosciences SA, H2 2017

List of Figures
Number of Products under Development for Crigler-Najjar Syndrome, H2 2017
Number of Products under Development by Companies, 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 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
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