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黏多醣症I(MPS I,賀勒氏症):開發平台分析

Mucopolysaccharidosis I - Pipeline Review, H1 2017

出版商 Global Markets Direct 商品編碼 245985
出版日期 內容資訊 英文 73 Pages
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黏多醣症I(MPS I,賀勒氏症):開發平台分析 Mucopolysaccharidosis I - Pipeline Review, H1 2017
出版日期: 2017年06月13日 內容資訊: 英文 73 Pages
簡介

賀勒氏症是罕見的難治疾病之一,是指人體內無法分解被稱為糖胺聚糖(舊稱:黏多醣體)的多糖(糖分子長連鎖)這樣的遺傳性代謝疾病。賀勒氏症屬於黏多醣症(MPS)的疾病群組。賀勒氏症患者由於無法生產α-L-iduronidase(溶酶體酶的一種)。該酵素的作用是支援糖胺聚糖的分解。這個分子常見於人體各部位、尤其是關節週圍的黏液及體液。其症狀有脊椎骨異常,爪形手,角膜混濁,聽障,心臟瓣膜症等。治療方法有骨髓移植,酵素療法,基因治療等。

本報告提供全球各國的黏多醣症I(MPS I,賀勒氏症)所用的開發中產品之開發情形相關分析,提供您開發中產品的最新趨勢,及臨床實驗的各階段產品一覽,主要企業簡介,主要藥物概要(產品概要,功能機制,研究開發(R&D)的發展情形),最新的產業趨勢等。

簡介

  • 分析範圍

黏多醣症I概要

治療藥的開發

  • 黏多醣症I開發中產品:概要
  • 黏多醣症I開發中產品:比較分析

各企業正在開發的黏多醣症I治療藥

各大學·研究機關研究中的黏多醣症I治療藥

開發中產品的概要

  • 臨床階段的產品
  • 初期階段的產品
  • 開發階段不明的產品

黏多醣症I開發治療藥的企業

  • AngioChem Inc.
  • ArmaGen Inc.
  • Athersys, Inc.
  • Bioasis Technologies Inc.
  • Eloxx Pharmaceuticals, Ltd.
  • Inventiva
  • OPKO Health, Inc.
  • PTC Therapeutics, Inc.
  • RegenxBio Inc.
  • Sangamo BioSciences, Inc.

黏多醣症I:治療藥的評估

  • 單劑治療藥的情況
  • 標的別
  • 各行動機制
  • 各投藥法
  • 各分子類型

藥物簡介

黏多醣症I治療藥:開發暫停的產品

黏多醣症I治療藥:開發中止的產品

黏多醣症I相關產品的開發的里程碑

  • 最新消息及新聞稿

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: GMDHC9430IDB

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Mucopolysaccharidosis I - Pipeline Review, H1 2017, provides an overview of the Mucopolysaccharidosis I (Metabolic Disorders) pipeline landscape.

MPS I (Mucopolysaccharidosis I) is an inherited lysosomal storage disorder caused by a deficiency of alpha-L-iduronidase, a lysosomal enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs). Symptoms include abnormal bones in the spine, claw hand, cloudy corneas, deafness and heart valve problems. Treatment includes bone marrow transplantation, enzyme therapy and gene therapy.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Mucopolysaccharidosis I - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis I (Metabolic 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 Mucopolysaccharidosis I (Metabolic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Mucopolysaccharidosis I (MPS I) (Hurler 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, Phase I, Preclinical and Discovery stages are 5, 1, 6 and 3 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 2 and 1 molecules, respectively.

Mucopolysaccharidosis I (Metabolic 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 Mucopolysaccharidosis I (Metabolic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Mucopolysaccharidosis I (Metabolic 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 Mucopolysaccharidosis I (Metabolic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Mucopolysaccharidosis I (Metabolic 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 Mucopolysaccharidosis I (Metabolic 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 Mucopolysaccharidosis I (Metabolic 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 Mucopolysaccharidosis I (Metabolic 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

List of Tables

List of Figures

  • Introduction
  • Global Markets Direct Report Coverage
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Overview
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics Development
  • Pipeline Overview
  • Pipeline by Companies
  • Pipeline by Universities/Institutes
  • Products under Development by Companies
  • Products under Development by Universities/Institutes
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Therapeutics Assessment
  • Assessment by Target
  • Assessment by Mechanism of Action
  • Assessment by Route of Administration
  • Assessment by Molecule Type
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Companies Involved in Therapeutics Development
  • AngioChem Inc
  • ArmaGen Inc
  • Bioasis Technologies Inc
  • CRISPR Therapeutics
  • Eloxx Pharmaceuticals Ltd
  • Immusoft Corp
  • Inventiva
  • OPKO Health Inc
  • PTC Therapeutics Inc
  • RegenxBio Inc
  • Sangamo Therapeutics Inc
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Drug Profiles
  • AGT-181 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • ataluren - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Cell Therapy for Mucopolysaccharidosis I - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Cell Therapy to Activate Alpha L-Iduronidase for Hurler Syndrome - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • ELX-02 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • JOT-102 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • MGTA-456 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • odiparcil - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Oligonucleotide for Mucopolysaccharidosis I - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • pentosan polysulfate sodium - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Recombinant Alpha-L-Iduronidase Replacement for Hurler Syndrome - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • RGX-111 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • SB-318 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Stem Cell Therapy for Hurler Sydrome - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Stem Cell Therapy for Type1 Mucopolysaccharidosis - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • X-372 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • R&D Progress
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Discontinued Products
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Product Development Milestones
  • Featured News & Press Releases
  • Feb 16, 2017: ArmaGen Reports Preliminary Evidence of Cognitive Improvement in Children with Hurler Syndrome (MPS I) Treated with AGT-181
  • Feb 07, 2017: ArmaGen Announces Oral Presentation of Preliminary Results from its Phase 2 Clinical Trial of AGT-181 in Patients with MPS 1 to be Presented at WORLDSymposium 2017
  • Nov 07, 2016: Eloxx Pharmaceuticals Announces Orphan Drug Designation in the U.S. and Europe for ELX-02 in Mucopolysacchardisis Type 1 (MPS 1)
  • Sep 13, 2016: REGENXBIO Publishes Data from Ongoing Preclinical Studies of NAV Gene Therapy RGX-111
  • Jul 05, 2016: REGENXBIO Provides Update On Gene Therapy Development Program RGX-111
  • Mar 31, 2016: ArmaGen Announces Initiation of Phase 2 Proof-of-Concept Clinical Trial in Brazil to Study AGT-181 for the Treatment of Hurler Syndrome
  • Dec 30, 2015: FDA Grants Rare Pediatric Disease Designation to REGENXBIO RGX-111 Gene Therapy for the Treatment of Mucopolysaccharidosis Type I (MPS I)
  • Nov 05, 2015: ArmaGen Receives Rare Pediatric Disease Designation from FDA for AGT-181 for the Potential Treatment of Hurler Syndrome
  • Oct 01, 2015: FDA Grants Orphan Drug Designation to REGENXBIO's RGX-111 Gene Therapy for the Treatment of Mucopolysaccharidosis Type I
  • Sep 02, 2015: ArmaGen Announces First Patient Dosed in Phase 1/2a Clinical Trial of AGT-181 for the Treatment of Hurler Syndrome
  • Apr 08, 2015: ArmaGen Announces FDA Acceptance of IND Application for AGT-181 for the Treatment of Hurler Syndrome
  • Dec 19, 2014: Translarna Granted Orphan Drug Designation in the U.S. and Europe for the Treatment of Mucopolysaccharidosis I
  • Feb 05, 2014: Mouse Study Shows Gene Therapy May Be Possible Cure for Hurler Syndrome
  • Appendix
  • Methodology
  • Coverage
  • Secondary Research
  • Primary Research
  • Expert Panel Validation
  • Contact Us
  • Disclaimer

List of Tables

  • Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H1 2017
  • Number of Products under Development by Companies, H1 2017
  • Number of Products under Development by Universities/Institutes, H1 2017
  • Products under Development by Companies, H1 2017
  • Products under Development by Universities/Institutes, H1 2017
  • Number of Products by Stage and Target, H1 2017
  • Number of Products by Stage and Mechanism of Action, H1 2017
  • Number of Products by Stage and Route of Administration, H1 2017
  • Number of Products by Stage and Molecule Type, H1 2017
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by AngioChem Inc, H1 2017
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by ArmaGen Inc, H1 2017
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Bioasis Technologies Inc, H1 2017
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by CRISPR Therapeutics, H1 2017
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Eloxx Pharmaceuticals Ltd, H1 2017
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Immusoft Corp, H1 2017
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Inventiva, H1 2017
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by OPKO Health Inc, H1 2017
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by PTC Therapeutics Inc, H1 2017
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by RegenxBio Inc, H1 2017
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Pipeline by Sangamo Therapeutics Inc, H1 2017
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Dormant Projects, H1 2017
  • Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Discontinued Products, H1 2017

List of Figures

  • Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), H1 2017
  • Number of Products under Development by Companies, H1 2017
  • Number of Products under Development by Universities/Institutes, H1 2017
  • Number of Products by Targets, H1 2017
  • Number of Products by Stage and Targets, H1 2017
  • Number of Products by Mechanism of Actions, H1 2017
  • Number of Products by Stage and Mechanism of Actions, H1 2017
  • Number of Products by Routes of Administration, H1 2017
  • Number of Products by Stage and Routes of Administration, H1 2017
  • Number of Products by Molecule Types, H1 2017
  • Number of Products by Stage and Molecule Types, H1 2017
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