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球細胞腦白質失養症 (克拉伯病) :開發中產品分析

Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline Review, H2 2019

出版商 Global Markets Direct 商品編碼 408071
出版日期 內容資訊 英文 71 Pages
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球細胞腦白質失養症 (克拉伯病) :開發中產品分析 Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline Review, H2 2019
出版日期: 2019年12月05日內容資訊: 英文 71 Pages
簡介

所謂球細胞腦白質失養症 (克拉伯病),是因缺乏被半乳糖神經酰胺酶的酶所引起的退行性疾病。由於缺乏這種酶損害髓磷脂的生長和維持。其症狀包括癲癇、肌肉痙攣、失去頭部控制、嘔吐、發燒、煩躁和大哭。危險因子有家族病史。治療側重於管理症狀(抗驚厥藥,肌肉鬆弛藥)和提供支持性治療。

k 本報告提供以球細胞腦白質失養症 (克拉伯病) 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,提供您加上最新的新聞和發表之企業和研究機關開發中的治療藥,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

球細胞腦白質失養症 (克拉伯病) 概要

治療藥的開發

  • 開發中產品;概要
  • 開發中產品;比較分析

企業開發中的治療藥

大學/機關研究中的治療藥

開發中產品概況

  • 臨床實驗階段的產品
  • 初期階段的產品

企業開發中的產品

大學/研究機關研究中的產品

開發治療藥的企業

  • Commence Bio Inc
  • 杏林藥業
  • Novartis AG
  • Nuo Therapeutics Inc

治療藥的評估

  • 單劑產品
  • 標的別
  • 各作用機制
  • 各給藥途徑
  • 各分子類型

藥物簡介

暫停中的計劃

開發中止的產品

產品開發的里程碑

  • 主要消息和新聞稿

附錄

圖表

本網頁內容可能與最新版本有所差異。詳細情況請與我們聯繫。

目錄
Product Code: GMDHC11592IDB

Summary:

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline Review, H2 2019, provides an overview of the Globoid Cell Leukodystrophy (Krabbe Disease) (Central Nervous System) pipeline landscape.

Krabbe disease also called globoid cell leukodystrophy is a degenerative disorder caused by the deficiency of an enzyme called galactosylceramidase. This enzyme deficiency impairs the growth and maintenance of myelin. Symptoms include seizures, Muscle spasms, Loss of head control, vomiting, fevers, irritability and excessive crying. Risk factors include family history. Treatment focuses on managing symptoms (anticonvulsant, muscle relaxer) and providing supportive care.

Report Highlights:

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline Review, H2 2019, provides comprehensive information on the therapeutics under development for Globoid Cell Leukodystrophy (Krabbe Disease) (Central Nervous System), 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 Globoid Cell Leukodystrophy (Krabbe Disease) (Central Nervous System) pipeline guide also reviews of key players involved in therapeutic development for Globoid Cell Leukodystrophy (Krabbe 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, Preclinical and Discovery stages are 1, 5 and 3 respectively. Similarly, the Universities portfolio in Phase I and Preclinical stages comprises 1 and 1 molecules, respectively.

Globoid Cell Leukodystrophy (Krabbe Disease) (Central Nervous System) 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 Globoid Cell Leukodystrophy (Krabbe Disease) (Central Nervous System).
  • The pipeline guide reviews pipeline therapeutics for Globoid Cell Leukodystrophy (Krabbe Disease) (Central Nervous System) 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 Globoid Cell Leukodystrophy (Krabbe Disease) (Central Nervous System) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Globoid Cell Leukodystrophy (Krabbe Disease) (Central Nervous System) 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 Globoid Cell Leukodystrophy (Krabbe Disease) (Central Nervous System)

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 Globoid Cell Leukodystrophy (Krabbe Disease) (Central Nervous System).
  • 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 Globoid Cell Leukodystrophy (Krabbe Disease) (Central Nervous System) 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

  • List of Tables
  • List of Figures
  • Introduction
    • Global Markets Direct Report Coverage
  • Globoid Cell Leukodystrophy (Krabbe Disease) - Overview
  • Globoid Cell Leukodystrophy (Krabbe Disease) - Therapeutics Development
    • Pipeline Overview
    • Pipeline by Companies
    • Pipeline by Universities/Institutes
    • Products under Development by Companies
    • Products under Development by Universities/Institutes
  • Globoid Cell Leukodystrophy (Krabbe Disease) - Therapeutics Assessment
    • Assessment by Target
    • Assessment by Mechanism of Action
    • Assessment by Route of Administration
    • Assessment by Molecule Type
  • Globoid Cell Leukodystrophy (Krabbe Disease) - Companies Involved in Therapeutics Development
    • BioXcel Corp
    • Commence Bio Inc
    • Kyorin Pharmaceutical Co Ltd
    • Magenta Therapeutics Inc
    • Passage Bio Inc
    • Polaryx Therapeutics Inc
    • Ranedis Pharmaceuticals LLC
    • Vascumab LLC
  • Globoid Cell Leukodystrophy (Krabbe Disease) - Drug Profiles
    • Drug for Krabbe Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • DUOC-01 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • gemfibrozil - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy for Globoid Cell Leukodystrophy - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • GTP-206 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • ibudilast - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • MGTA-456 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • MSC-2 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • PLX-300 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • RND-001 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecules to Activate GALC for Krabbe Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
  • Globoid Cell Leukodystrophy (Krabbe Disease) - Dormant Projects
  • Globoid Cell Leukodystrophy (Krabbe Disease) - Discontinued Products
  • Globoid Cell Leukodystrophy (Krabbe Disease) - Product Development Milestones
    • Featured News & Press Releases
      • Sep 04, 2019: Magenta Therapeutics announces FDA regenerative medicine advanced therapy (RMAT) designation granted to MGTA-456 for the treatment of Inherited Metabolic Disorders
      • May 30, 2019: Passage Bio announces third gene therapy development program in Krabbe Disease and supports million dreams fundraising gala
      • May 09, 2019: Magenta Therapeutics presents updated phase 2 clinical data on MGTA-456 cell therapy at American Academy of Neurology Annual Meeting
      • Feb 25, 2019: Magenta Therapeutics Presents Clinical and Preclinical Data on MGTA-456 Cell Therapy in Best Abstracts Sessions at Transplant and Cellular Therapy (TCT) Annual Meeting
      • Apr 05, 2018: Magenta Therapeutics Announces First Patient Transplanted with MGTA-456 in Phase 2 Study in Inherited Metabolic Disorders
      • Jan 18, 2016: FDA Grants Rare Pediatric Disease Designation to MediciNova's MN-166 (ibudilast) for the Treatment of Krabbe Disease
      • Jun 03, 2015: MediciNova Announces FDA Granted Orphan Drug Designation to MN-166 (ibudilast) for Krabbe Disease
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
    • Disclaimer

List of Tables

  • Number of Products under Development for Globoid Cell Leukodystrophy (Krabbe Disease), H2 2019
  • Number of Products under Development by Companies, H2 2019
  • Number of Products under Development by Universities/Institutes, H2 2019
  • Products under Development by Companies, H2 2019
  • Products under Development by Universities/Institutes, H2 2019
  • Number of Products by Stage and Target, H2 2019
  • Number of Products by Stage and Mechanism of Action, H2 2019
  • Number of Products by Stage and Route of Administration, H2 2019
  • Number of Products by Stage and Molecule Type, H2 2019
  • Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by BioXcel Corp, H2 2019
  • Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by Commence Bio Inc, H2 2019
  • Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by Kyorin Pharmaceutical Co Ltd, H2 2019
  • Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by Magenta Therapeutics Inc, H2 2019
  • Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by Passage Bio Inc, H2 2019
  • Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by Polaryx Therapeutics Inc, H2 2019
  • Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by Ranedis Pharmaceuticals LLC, H2 2019
  • Globoid Cell Leukodystrophy (Krabbe Disease) - Pipeline by Vascumab LLC, H2 2019
  • Globoid Cell Leukodystrophy (Krabbe Disease) - Dormant Projects, H2 2019
  • Globoid Cell Leukodystrophy (Krabbe Disease) - Discontinued Products, H2 2019

List of Figures

  • Number of Products under Development for Globoid Cell Leukodystrophy (Krabbe Disease), H2 2019
  • Number of Products under Development by Companies, H2 2019
  • Number of Products under Development by Universities/Institutes, H2 2019
  • Number of Products by Targets, H2 2019
  • Number of Products by Stage and Targets, H2 2019
  • Number of Products by Mechanism of Actions, H2 2019
  • Number of Products by Stage and Mechanism of Actions, H2 2019
  • Number of Products by Routes of Administration, H2 2019
  • Number of Products by Stage and Routes of Administration, H2 2019
  • Number of Products by Top 10 Molecule Types, H2 2019
  • Number of Products by Stage and Top 10 Molecule Types, H2 2019
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