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山德霍夫氏病(GM2神經節苷脂儲積症):開發中產品分析

Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Pipeline Review, H2 2019

出版商 Global Markets Direct 商品編碼 465219
出版日期 內容資訊 英文 39 Pages
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山德霍夫氏病(GM2神經節苷脂儲積症):開發中產品分析 Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Pipeline Review, H2 2019
出版日期: 2019年12月27日內容資訊: 英文 39 Pages
簡介

山德霍夫氏病是遺傳性脂質儲存障礙,會逐漸破壞腦和脊髓中的神經細胞(神經元)。是由於缺乏酶β-氨基己糖苷酶所引起。症狀包括進行性神經系統惡化,造成肌肉控制和運動的問題,對聲音的驚嚇反應增加,早期失明,癲癇發作,痙攣和肌肉陣攣。治療包括使用抗驚厥藥以控制癲癇發作。

本報告提供山德霍夫氏病(GM2神經節苷脂儲積症)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

山德霍夫氏病(GM2神經節苷脂儲積症);概要

治療藥的開發

  • 開發中產品;概要
  • 各企業的開發中產品
  • 各大學/研究機關的開發中產品
  • 企業開發中的產品
  • 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

  • Bioasis Technologies Inc
  • Nuo Therapeutics Inc

藥物簡介

  • ALD-601
  • CCP-010
  • DUOC-01
  • MTf-HEXB
  • MTfp-HEXB
  • OT-1001

暫停中的計劃

附錄

圖表

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目錄
Product Code: GMDHC11832IDB

Summary:

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Pipeline Review, H2 2019, provides an overview of the Sandhoff Disease (Jatzkewitz-Pilz Syndrome) (Genetic Disorders) pipeline landscape.

Sandhoff disease is an inherited lipid storage disorder that progressively destroys nerve cells (neurons) in the brain and spinal cord. It is caused by a deficiency of the enzyme beta-hexosaminidase. Symptoms include progressive nervous system deterioration, problems initiating and controlling muscles and movement, increased startle reaction to sound, early blindness, seizures, spasticity, and myoclonus. Treatment includes anticonvulsants to control seizures.

Report Highlights:

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Pipeline Review, H2 2019, provides comprehensive information on the therapeutics under development for Sandhoff Disease (Jatzkewitz-Pilz 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 Sandhoff Disease (Jatzkewitz-Pilz Syndrome) (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Sandhoff Disease (Jatzkewitz-Pilz 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, Preclinical and Discovery stages are 2, 4 and 1 respectively. Similarly, the Universities portfolio in Phase I stages comprises 1 molecules, respectively.

Sandhoff Disease (Jatzkewitz-Pilz 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 Sandhoff Disease (Jatzkewitz-Pilz Syndrome) (Genetic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Sandhoff Disease (Jatzkewitz-Pilz 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 Sandhoff Disease (Jatzkewitz-Pilz Syndrome) (Genetic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Sandhoff Disease (Jatzkewitz-Pilz 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 Sandhoff Disease (Jatzkewitz-Pilz 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 Sandhoff Disease (Jatzkewitz-Pilz 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 Sandhoff Disease (Jatzkewitz-Pilz 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
  • Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Overview
    • Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Therapeutics Development
    • Pipeline Overview
    • Pipeline by Companies
    • Pipeline by Universities/Institutes
    • Products under Development by Companies
    • Products under Development by Universities/Institutes
  • Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Therapeutics Assessment
    • Assessment by Target
    • Assessment by Mechanism of Action
    • Assessment by Route of Administration
    • Assessment by Molecule Type
  • Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Companies Involved in Therapeutics Development
    • Axovant Gene Therapies Ltd
    • IntraBio Ltd
    • Orphi Therapeutics Inc
    • Polaryx Therapeutics Inc
    • Recursion Pharmaceuticals Inc
  • Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Drug Profiles
  • Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Dormant Projects
  • Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Discontinued Products
  • Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Product Development Milestones
  • Appendix

List of Tables

  • Number of Products under Development for Sandhoff Disease (Jatzkewitz-Pilz Syndrome), 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
  • Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Pipeline by Axovant Gene Therapies Ltd, H2 2019
  • Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Pipeline by IntraBio Ltd, H2 2019
  • Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Pipeline by Orphi Therapeutics Inc, H2 2019
  • Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Pipeline by Polaryx Therapeutics Inc, H2 2019
  • Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Pipeline by Recursion Pharmaceuticals Inc, H2 2019
  • Sandhoff Disease (Jatzkewitz-Pilz Syndrome) - Dormant Projects, H2 2019

List of Figures

  • Number of Products under Development for Sandhoff Disease (Jatzkewitz-Pilz Syndrome), H2 2019
  • Number of Products under Development by Companies, 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 Molecule Types, H2 2019
  • Number of Products by Stage and Molecule Types, H2 2019
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