Crigler-Najjar Syndrome - Pipeline Review, H1 2018

  • ID: 4473097
  • Report
  • 34 pages
  • Global Markets Direct
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FEATURED COMPANIES

  • Audentes Therapeutics Inc
  • Genethon SA
  • Promethera Biosciences SA
  • MORE
Crigler-Najjar Syndrome - Pipeline Review, H1 2018

Summary:

This latest Pharmaceutical and Healthcare disease pipeline guide Crigler-Najjar Syndrome - Pipeline Review, H1 2018, 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:

Pharmaceutical and Healthcare latest pipeline guide Crigler-Najjar Syndrome - Pipeline Review, H1 2018, 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 and Preclinical stages are 3 and 1 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 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 Purchase:
  • 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.
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Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • Audentes Therapeutics Inc
  • Genethon SA
  • Promethera Biosciences SA
  • MORE
Introduction

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

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

GNT-0003 - 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 - Dormant Projects

Crigler-Najjar Syndrome - Product Development Milestones

Featured News & Press Releases

Feb 12, 2018: Audentes Therapeutics Announces Dosing of First Patient in VALENS, a Phase 1/2 Clinical Trial of AT342 for the treatment of Crigler-Najjar Syndrome

Nov 13, 2017: Audentes Therapeutics Announces MHRA Approval of Clinical Trial Authorisation Application for AT342 to Treat Crigler-Najjar Syndrome

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

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, 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

Crigler-Najjar Syndrome - Pipeline by Audentes Therapeutics Inc, H1 2018

Crigler-Najjar Syndrome - Pipeline by Genethon SA, H1 2018

Crigler-Najjar Syndrome - Pipeline by International Stem Cell Corp, H1 2018

Crigler-Najjar Syndrome - Pipeline by Promethera Biosciences SA, H1 2018

Crigler-Najjar Syndrome - Dormant Projects, H1 2018

List of Figures:

Number of Products under Development for Crigler-Najjar Syndrome, H1 2018

Number of Products under Development by Companies, 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 Stage and Routes of Administration, H1 2018

Number of Products by Molecule Types, H1 2018

Number of Products by Stage and Molecule Types, H1
Note: Product cover images may vary from those shown
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  • Audentes Therapeutics Inc
  • Genethon SA
  • International Stem Cell Corp
  • Promethera Biosciences SA
Note: Product cover images may vary from those shown
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Note: Product cover images may vary from those shown
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