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市場調查報告書

克果納傑氏症:開發中產品分析

Crigler-Najjar Syndrome - Pipeline Review, H2 2020

出版商 Global Markets Direct 商品編碼 465226
出版日期 內容資訊 英文 39 Pages
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克果納傑氏症:開發中產品分析 Crigler-Najjar Syndrome - Pipeline Review, H2 2020
出版日期: 2020年08月31日內容資訊: 英文 39 Pages
簡介

克果納傑氏症是一種嚴重的病症,其特徵在於血液中有毒物質膽紅素增加到危及性命(高膽紅素血症)。症狀包括皮膚和眼白發黃(黃疸)。一般是由於體內可將膽紅素轉化為可排出體外型態的酶無法正常運作才引發。治療包括光療,肝移植和輸血。

本報告提供克果納傑氏症的治療藥開發平台現狀及最新更新的各開發階段比較分析,提供您涵括最新的新聞和發表之企業和研究機關開發中治療藥,治療藥評估,後期階段及中止的計劃等相關資訊。

簡介

  • 調查範圍

克果納傑氏症;概要

治療藥的開發

  • 開發中產品;概要
  • 企業開發中的產品
  • 各大學/研究機關的開發中產品
  • 企業開發中的治療藥
  • 大學/研究機關開發中的產品

治療藥的評估

  • 標的別
  • 各作用機制
  • 各給藥途徑
  • 各分子類型

開發治療藥的企業

  • Audentes Therapeutics Inc
  • Genethon SA
  • International Stem Cell Corp
  • Promethera Biosciences SA

藥物簡介

  • ALXN-1540
  • AT-342
  • Gene Therapy to Activate Glucuronosyltransferase for Crigler-Najjar Syndrome
  • Gene Therapy to Activate UGT1A1 for Crigler-Najjar Syndrome
  • HepaStem
  • Stem Cell Therapy for Alpha-Antitrypsin Deficiency, Crigler-Najjar Syndrome and Liver Cirrhosis

產品開發的里程碑

  • 主要消息和新聞稿

附錄

圖表

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目錄
Product Code: GMDHC12429IDB

Summary:

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Crigler-Najjar Syndrome - Pipeline Review, H2 2020, 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 2020, 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 1 and 3 respectively. Similarly, the Universities portfolio in Phase II 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.

Table of Contents

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
  • International Stem Cell Corp
  • Logicbio Therapeutics Inc
  • Promethera Biosciences SA
  • Selecta Biosciences Inc
  • Crigler-Najjar Syndrome - Drug Profiles
  • Gene Therapy 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
  • Crigler-Najjar Syndrome - Dormant Projects
  • Crigler-Najjar Syndrome - Product Development Milestones
  • Featured News & Press Releases
  • 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

List of Tables

  • Number of Products under Development for Crigler-Najjar Syndrome, H2 2020
  • Number of Products under Development by Companies, H2 2020
  • Number of Products under Development by Universities/Institutes, H2 2020
  • Products under Development by Companies, H2 2020
  • Products under Development by Universities/Institutes, H2 2020
  • Number of Products by Stage and Target, H2 2020
  • Number of Products by Stage and Mechanism of Action, H2 2020
  • Number of Products by Stage and Route of Administration, H2 2020
  • Number of Products by Stage and Molecule Type, H2 2020
  • Crigler-Najjar Syndrome - Pipeline by International Stem Cell Corp, H2 2020
  • Crigler-Najjar Syndrome - Pipeline by Logicbio Therapeutics Inc, H2 2020
  • Crigler-Najjar Syndrome - Pipeline by Promethera Biosciences SA, H2 2020
  • Crigler-Najjar Syndrome - Pipeline by Selecta Biosciences Inc, H2 2020
  • Crigler-Najjar Syndrome - Dormant Projects, H2 2020

List of Figures

List of Figures

  • Number of Products under Development for Crigler-Najjar Syndrome, H2 2020
  • Number of Products under Development by Companies, H2 2020
  • Number of Products by Stage and Targets, H2 2020
  • Number of Products by Stage and Mechanism of Actions, H2 2020
  • Number of Products by Routes of Administration, H2 2020
  • Number of Products by Stage and Routes of Administration, H2 2020
  • Number of Products by Molecule Types, H2 2020
  • Number of Products by Stage and Molecule Types, H2 2020