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市場調查報告書

貝登氏症 - 開發中產品分析

Batten Disease - Pipeline Review, H2 2016

出版商 Global Markets Direct 商品編碼 277747
出版日期 內容資訊 英文 72 Pages
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貝登氏症 - 開發中產品分析 Batten Disease - Pipeline Review, H2 2016
出版日期: 2016年08月31日 內容資訊: 英文 72 Pages
簡介

貝登氏症是惡化型代謝性神經退化性疾病的一種,又稱為神經元蠟樣脂褐質儲積症。症狀有從5歲到15歲之間的失明,肌肉的協調運動失調,精神遲緩或精神功能低,情緒障礙、抽筋、肌肉的痙攣、肌肉張力低落、異動症等。

本報告提供貝登氏症的治療藥開發情形調查分析,提供您開發中產品概要,臨床實驗的各階段產品概要,主要企業簡介,藥物簡介,開發中產品的最新趨勢,最新消息和新聞稿等相關的系統性資訊。

目錄

簡介

貝登氏症概要

治療藥的開發

  • 開發中產品:概要
  • 開發中產品:比較分析

貝登氏症 - 開發中的治療藥:各企業

貝登氏症 - 調查中的治療藥:大學/研究機關別

貝登氏症 - 開發中產品的概要

  • 臨床實驗階段的產品
  • 初期階段的產品
  • 開發階段不明的產品

貝登氏症 - 開發中的產品:各企業

貝登氏症 - 調查中的產品:大學/研究機關別

貝登氏症 - 開發治療藥的企業

  • Abeona Therapeutics, Inc.
  • BioMarin Pharmaceutical Inc.
  • CereSpir Incorporated
  • RegenxBio Inc.
  • Spark Therapeutics, Inc.

貝登氏症 -治療藥的評估

  • 單獨療法的情況
  • 標的別
  • 各作用機制
  • 各給藥途徑
  • 各分子類型

藥物簡介

  • ABO-201
  • cerliponase alfa
  • CSP-1103
  • Gene Therapy to Activate Palmitoyl Protein Thioesterase 1 for Batten Disease
  • Gene Therapy to Activate Tripeptidyl Peptidase 1 for Batten Disease
  • INI-0602
  • MP-101
  • Small Molecules to Inhibit CDC42 for Batten Disease
  • SNB-2401
  • SNB-4050
  • SPKTPP-1
  • XN-001

貝登氏症 - 開發中產品的最新趨勢

貝登氏症 - 開發暫停中的計劃

貝登氏症 - 開發中止的產品

貝登氏症 - 產品開發的里程碑

  • 最新消息和新聞稿

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: GMDHC8413IDB

Summary

Global Markets Direct's, 'Batten Disease - Pipeline Review, H2 2016', provides an overview of the Batten Disease pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Batten Disease, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Batten Disease and features dormant and discontinued projects.

Global Markets Direct's report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report 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. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report 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.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.

Scope

  • The report provides a snapshot of the global therapeutic landscape of Batten Disease
  • The report reviews pipeline therapeutics for Batten Disease by companies and universities/research institutes based on information derived from company and industry-specific sources
  • The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages
  • The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities
  • The report reviews key players involved Batten Disease therapeutics and enlists all their major and minor projects
  • The report assesses Batten Disease therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type
  • The report summarizes all the dormant and discontinued pipeline projects
  • The report reviews latest news related to pipeline therapeutics for Batten Disease

Reasons to buy

  • Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
  • Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
  • Identify and understand important and diverse types of therapeutics under development for Batten Disease
  • Identify potential new clients or partners in the target demographic
  • Develop strategic initiatives by understanding the focus areas of leading companies
  • Plan mergers and acquisitions effectively by identifying key players and it's most promising pipeline therapeutics
  • Devise corrective measures for pipeline projects by understanding Batten Disease 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
  • Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline

Table of Contents

  • Table of Contents
    • List of Tables
    • List of Figures
  • Introduction
    • Global Markets Direct Report Coverage
  • Batten Disease Overview
  • Therapeutics Development
    • Pipeline Products for Batten Disease - Overview
    • Pipeline Products for Batten Disease - Comparative Analysis
  • Batten Disease - Therapeutics under Development by Companies
  • Batten Disease - Therapeutics under Investigation by Universities/Institutes
  • Batten Disease - Pipeline Products Glance
    • Late Stage Products
    • Clinical Stage Products
    • Early Stage Products
    • Unknown Stage Products
  • Batten Disease - Products under Development by Companies
  • Batten Disease - Products under Investigation by Universities/Institutes
  • Batten Disease - Companies Involved in Therapeutics Development
    • Abeona Therapeutics, Inc.
    • BioMarin Pharmaceutical Inc.
    • CereSpir Incorporated
    • Evotec AG
    • Ionis Pharmaceuticals, Inc.
    • Mitochon Pharmaceuticals, Inc.
    • RegenxBio Inc.
    • Retrotope, Inc
    • Spark Therapeutics, Inc.
  • Batten Disease - Therapeutics Assessment
    • Assessment by Monotherapy Products
    • Assessment by Target
    • Assessment by Mechanism of Action
    • Assessment by Route of Administration
    • Assessment by Molecule Type
  • Drug Profiles
    • ABO-201 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Antisense Oligonucleotides to Activate CLN3 for Batten Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • cerliponase alfa - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • CSP-1103 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • DUOC-01 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy to Activate CLN1 and CLN2 for Batten Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy to Activate CLN6 for Batten Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy to Activate Palmitoyl Protein Thioesterase 1 for Batten Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy to Activate Tripeptidyl Peptidase 1 for Batten Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • INI-0602 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • MP-101 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • RT-002 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecule for Batten Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecules for Juvenile Batten Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecules to Inhibit CDC42 for Batten Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • SNB-2401 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • SNB-4050 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • SPKTPP-1 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • XN-001 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
  • Batten Disease - Dormant Projects
  • Batten Disease - Discontinued Products
  • Batten Disease - Product Development Milestones
    • Featured News & Press Releases
      • Jul 27, 2016: FDA Accepts BLA for BioMarins Cerliponase Alfa for CLN2 Disease, Form of Batten Disease
      • May 03, 2016: BioMarin Announces EMA Grants Accelerated Assessment for Cerliponase Alfa, Experimental Treatment for a Form of Batten Disease
      • Mar 02, 2016: Abeona Therapeutics Highlights New Preclinical Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) Data at WORLDSymposium 2016
      • Mar 02, 2016: BioMarin Announces Positive Data From Cerliponase Alfa Program for Treatment of CLN2 Disease, a Form of Batten Disease, at 12th Annual WORLDSymposium 2016
      • Nov 11, 2015: Spark Therapeutics Announces SPK-TPP1, Its First Program Targeting Neurodegenerative Disease
      • Jan 12, 2015: BioMarin Provides Preliminary Data From Ongoing Phase 1/2 Pivotal Study of BMN 190 for Treatment of CLN2 Disorder, a Form of Batten Disease
      • Sep 23, 2013: BioMarin Doses First Patient in Phase 1/2 Trial With BMN 190 for the Treatment of Neuronal Ceroid Lipofuscinosis Type 2, a Form of Batten Disease
      • Apr 01, 2013: BioMarin Pharma Submits CTA For BMN-190 For Batten Disease
      • Mar 15, 2012: Combination treatment in mice shows promise for fatal neurological disorder in kids
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
    • Disclaimer

List of Tables

  • Number of Products under Development for Batten Disease, H2 2016
  • Number of Products under Development for Batten Disease - Comparative Analysis, H2 2016
  • Number of Products under Development by Companies, H2 2016
  • Number of Products under Investigation by Universities/Institutes, H2 2016
  • Comparative Analysis by Late Stage Development, H2 2016
  • Comparative Analysis by Clinical Stage Development, H2 2016
  • Comparative Analysis by Early Stage Development, H2 2016
  • Comparative Analysis by Unknown Stage Development, H2 2016
  • Products under Development by Companies, H2 2016
  • Products under Investigation by Universities/Institutes, H2 2016
  • Batten Disease - Pipeline by Abeona Therapeutics, Inc. , H2 2016
  • Batten Disease - Pipeline by BioMarin Pharmaceutical Inc., H2 2016
  • Batten Disease - Pipeline by CereSpir Incorporated, H2 2016
  • Batten Disease - Pipeline by Evotec AG, H2 2016
  • Batten Disease - Pipeline by Ionis Pharmaceuticals, Inc., H2 2016
  • Batten Disease - Pipeline by Mitochon Pharmaceuticals, Inc., H2 2016
  • Batten Disease - Pipeline by RegenxBio Inc., H2 2016
  • Batten Disease - Pipeline by Retrotope, Inc, H2 2016
  • Batten Disease - Pipeline by Spark Therapeutics, Inc., H2 2016
  • Assessment by Monotherapy Products, H2 2016
  • Number of Products by Stage and Target, H2 2016
  • Number of Products by Stage and Mechanism of Action, H2 2016
  • Number of Products by Stage and Route of Administration, H2 2016
  • Number of Products by Stage and Molecule Type, H2 2016
  • Batten Disease - Dormant Projects, H2 2016
  • Batten Disease - Discontinued Products, H2 2016

List of Figures

  • Number of Products under Development for Batten Disease, H2 2016
  • Number of Products under Development for Batten Disease - Comparative Analysis, H2 2016
  • Number of Products under Development by Companies, H2 2016
  • Number of Products under Investigation by Universities/Institutes, H2 2016
  • Comparative Analysis by Clinical Stage Development, H2 2016
  • Comparative Analysis by Early Stage Products, H2 2016
  • Assessment by Monotherapy Products, H2 2016
  • Number of Products by Targets, H2 2016
  • Number of Products by Stage and Targets, H2 2016
  • Number of Products by Mechanism of Actions, H2 2016
  • Number of Products by Stage and Mechanism of Actions, H2 2016
  • Number of Products by Routes of Administration, H2 2016
  • Number of Products by Stage and Routes of Administration, H2 2016
  • Number of Products by Molecule Types, H2 2016
  • Number of Products by Stage and Molecule Types, H2 2016
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