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若年性黄斑変性症(シュタルガルト病):パイプライン分析

Juvenile Macular Degeneration (Stargardt Disease) - Pipeline Review, H2 2016

発行 Global Markets Direct 商品コード 257806
出版日 ページ情報 英文 68 Pages
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若年性黄斑変性症(シュタルガルト病):パイプライン分析 Juvenile Macular Degeneration (Stargardt Disease) - Pipeline Review, H2 2016
出版日: 2016年08月24日 ページ情報: 英文 68 Pages
概要

若年性黄斑変性症は児童期・少年期に発症する遺伝性の眼病で、その最も一般的な形態はシュタルガルト病と呼ばれています。シュタルガルト病は先天性の常染色体劣性遺伝の症候群です。主な症状・徴候として、視野がぼやけたり、視野の中心部に暗点が現れたりして、読書や細かい作業ができなくなることが挙げられます。主なリスク要因には、動脈硬化症や高コレステロール血症、喫煙、高血圧などがあります。

当レポートでは、世界各国での若年性黄斑変性症(シュタルガルト病)治療用のパイプライン製品の開発状況について分析し、製品開発・上市の最新動向や、治験の各段階にある製品の一覧、主要企業のプロファイル、主な薬剤の概要、最新の業界動向などの情報をお届けします。

目次

イントロダクション

  • 分析範囲

若年性黄斑変性症の概要

治療薬の開発

  • 若年性黄斑変性症向けパイプライン製品:概要
  • 若年性黄斑変性症向けパイプライン製品:比較分析

企業で開発中の製品

大学/機関で研究中の製品

治療薬開発に従事している企業

  • Acucela Inc.
  • Alkeus Pharmaceuticals, Inc.
  • Ocata Therapeutics, Inc.
  • Sanofi

治療薬の評価

  • 単剤製品
  • 標的別
  • 作用機序別
  • 投与経路別
  • 分子タイプ別

薬剤プロファイル

  • ALK-001
  • echothiophate iodide
  • emixustat hydrochloride
  • Gene Therapy for Stargardt Disease
  • MA09-hRPE
  • SAR-422459
  • Small Molecule for Stargardt Disease
  • Stem Cell Therapy for Ophthalmic Disorders
  • VSM-20R

最新パイプライン動向

製品開発のマイルストーン

  • 主なニュースとプレスリリース

付録

図表一覧

このページに掲載されている内容は最新版と異なる場合があります。詳細はお問い合わせください。

目次
Product Code: GMDHC8396IDB

Summary

Global Markets Direct's, 'Juvenile Macular Degeneration (Stargardt Disease) - Pipeline Review, H2 2016', provides an overview of the Juvenile Macular Degeneration (Stargardt Disease) pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Juvenile Macular Degeneration (Stargardt 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 Juvenile Macular Degeneration (Stargardt 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 Juvenile Macular Degeneration (Stargardt Disease)
  • The report reviews pipeline therapeutics for Juvenile Macular Degeneration (Stargardt 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 Juvenile Macular Degeneration (Stargardt Disease) therapeutics and enlists all their major and minor projects
  • The report assesses Juvenile Macular Degeneration (Stargardt 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 Juvenile Macular Degeneration (Stargardt 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 Juvenile Macular Degeneration (Stargardt 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 Juvenile Macular Degeneration (Stargardt 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 2
  • List of Tables 5
  • List of Figures 5
  • Introduction 6
  • Global Markets Direct Report Coverage 6
  • Juvenile Macular Degeneration (Stargardt Disease) Overview 7
  • Therapeutics Development 8
  • Pipeline Products for Juvenile Macular Degeneration (Stargardt Disease) - Overview 8
  • Pipeline Products for Juvenile Macular Degeneration (Stargardt Disease) - Comparative Analysis 9
  • Juvenile Macular Degeneration (Stargardt Disease) - Therapeutics under Development by Companies 10
  • Juvenile Macular Degeneration (Stargardt Disease) - Therapeutics under Investigation by Universities/Institutes 11
  • Juvenile Macular Degeneration (Stargardt Disease) - Pipeline Products Glance 12
  • Clinical Stage Products 12
  • Early Stage Products 13
  • Juvenile Macular Degeneration (Stargardt Disease) - Products under Development by Companies 14
  • Juvenile Macular Degeneration (Stargardt Disease) - Products under Investigation by Universities/Institutes 15
  • Juvenile Macular Degeneration (Stargardt Disease) - Companies Involved in Therapeutics Development 16
  • Acucela Inc. 16
  • Alkeus Pharmaceuticals, Inc. 17
  • Astellas Pharma Inc. 18
  • Copernicus Therapeutics, Inc. 19
  • Iris Pharma 20
  • Sanofi 21
  • Juvenile Macular Degeneration (Stargardt Disease) - Therapeutics Assessment 22
  • Assessment by Monotherapy Products 22
  • Assessment by Target 23
  • Assessment by Mechanism of Action 25
  • Assessment by Route of Administration 27
  • Assessment by Molecule Type 29
  • Drug Profiles 31
  • ALK-001 - Drug Profile 31
  • Product Description 31
  • Mechanism Of Action 31
  • R&D Progress 31
  • echothiophate iodide - Drug Profile 32
  • Product Description 32
  • Mechanism Of Action 32
  • R&D Progress 32
  • emixustat hydrochloride - Drug Profile 33
  • Product Description 33
  • Mechanism Of Action 33
  • R&D Progress 33
  • Gene Therapy 1 to Activate ABCA4 for Stargardt Disease - Drug Profile 37
  • Product Description 37
  • Mechanism Of Action 37
  • R&D Progress 37
  • Gene Therapy to Activate ABCA4 for Stargardt Disease - Drug Profile 38
  • Product Description 38
  • Mechanism Of Action 38
  • R&D Progress 38
  • MA09-hRPE - Drug Profile 39
  • Product Description 39
  • Mechanism Of Action 39
  • R&D Progress 39
  • Oligonucleotides to Activate ABCA4 for Stargardt Disease - Drug Profile 49
  • Product Description 49
  • Mechanism Of Action 49
  • R&D Progress 49
  • ramiprilat - Drug Profile 50
  • Product Description 50
  • Mechanism Of Action 50
  • R&D Progress 50
  • SAR-422459 - Drug Profile 51
  • Product Description 51
  • Mechanism Of Action 51
  • R&D Progress 51
  • soraprazan - Drug Profile 53
  • Product Description 53
  • Mechanism Of Action 53
  • R&D Progress 53
  • Stem Cell Therapy for Ophthalmic Disorders - Drug Profile 54
  • Product Description 54
  • Mechanism Of Action 54
  • R&D Progress 54
  • VM-200 - Drug Profile 55
  • Product Description 55
  • Mechanism Of Action 55
  • R&D Progress 55
  • Juvenile Macular Degeneration (Stargardt Disease) - Dormant Projects 56
  • Juvenile Macular Degeneration (Stargardt Disease) - Product Development Milestones 57
  • Featured News & Press Releases 57
  • May 26, 2015: Ocata Therapeutics Receives Three New U.S. Patents for its RPE Therapy for Macular Degenerative Diseases 57
  • May 04, 2015: European Medicines Agency Grants Orphan Designation for Makindus' MI-100 for Stargardt's Disease 57
  • Mar 31, 2015: Ocata Therapeutics Successfully Completes Dosing in Phase 1/2 RPE Studies 58
  • Oct 15, 2014: ACT Announces Positive Results from Two Clinical Trials Published in The Lancet Using Differentiated Stem Cell-Derived Retinal Pigment Epithelium (RPE) Cells for the Treatment of Macular Degeneration 58
  • Sep 25, 2014: Advanced Cell Technology Announces Final Patient Treated in Stargardt's Macular Degeneration Phase 1 Trial in the United Kingdom 59
  • Jul 17, 2013: ACT Secures Approval from Data Safety Monitoring Board to Complete Third Patient Cohort in All Three Clinical Trials 60
  • May 16, 2013: ACT Confirms Clinical Trial Participant Shows Improvement In Vision Following Treatment With Human Embryonic Stem Cells 61
  • Apr 23, 2013: ACT Initiates Higher-Dosage Patient Treatment In European Phase I Study For Macular Degeneration 61
  • Apr 15, 2013: ACT Treats First Patient with Better Vision in Clinical Trial for Stargardt's Macular Dystrophy 61
  • Apr 01, 2013: ACT Initiates Treatment Of Higher-dosage Cohort In Clinical Trials For Dry Age-related Macular Degeneration And Stargardt's Macular Dystrophy 62
  • Mar 14, 2013: Advanced Cell Technology Receives DSMB Approval To Initiate Treatment Of Third Patient Cohort In All Three Clinical Trials Using hESC-derived RPE Cells 63
  • Jan 08, 2013: Advanced Cell Technology Achieves Clinical Milestone 64
  • Dec 17, 2012: ACT Completes Higher-Dosage Cohort In European Clinical Trial For Stargardt's Macular Dystrophy Using Retinal Pigment Epithelial Cells 64
  • Nov 28, 2012: ACT Completes Higher-Dosage Cohort In Both US Clinical Trials Using Retinal Pigment Epithelial Cells 65
  • Nov 08, 2012: Advanced Cell Technology Announces Interim Data From Three Ongoing Macular Degeneration Trials 65
  • Appendix 67
  • Methodology 67
  • Coverage 67
  • Secondary Research 67
  • Primary Research 67
  • Expert Panel Validation 67
  • Contact Us 67
  • Disclaimer 68

List of Tables

  • Number of Products under Development for Juvenile Macular Degeneration (Stargardt Disease), H2 2016 8
  • Number of Products under Development for Juvenile Macular Degeneration (Stargardt Disease) - Comparative Analysis, H2 2016 9
  • Number of Products under Development by Companies, H2 2016 10
  • Number of Products under Investigation by Universities/Institutes, H2 2016 11
  • Comparative Analysis by Clinical Stage Development, H2 2016 12
  • Comparative Analysis by Early Stage Development, H2 2016 13
  • Products under Development by Companies, H2 2016 14
  • Products under Investigation by Universities/Institutes, H2 2016 15
  • Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Acucela Inc., H2 2016 16
  • Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Alkeus Pharmaceuticals, Inc., H2 2016 17
  • Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Astellas Pharma Inc., H2 2016 18
  • Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Copernicus Therapeutics, Inc., H2 2016 19
  • Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Iris Pharma, H2 2016 20
  • Juvenile Macular Degeneration (Stargardt Disease) - Pipeline by Sanofi, H2 2016 21
  • Assessment by Monotherapy Products, H2 2016 22
  • Number of Products by Stage and Target, H2 2016 24
  • Number of Products by Stage and Mechanism of Action, H2 2016 26
  • Number of Products by Stage and Route of Administration, H2 2016 28
  • Number of Products by Stage and Molecule Type, H2 2016 30
  • Juvenile Macular Degeneration (Stargardt Disease) - Dormant Projects, H2 2016 56

List of Figures

  • Number of Products under Development for Juvenile Macular Degeneration (Stargardt Disease), H2 2016 8
  • Number of Products under Development for Juvenile Macular Degeneration (Stargardt Disease) - Comparative Analysis, H2 2016 9
  • Number of Products under Development by Companies, H2 2016 10
  • Comparative Analysis by Early Stage Products, H2 2016 13
  • Assessment by Monotherapy Products, H2 2016 22
  • Number of Products by Targets, H2 2016 23
  • Number of Products by Stage and Targets, H2 2016 23
  • Number of Products by Mechanism of Actions, H2 2016 25
  • Number of Products by Stage and Mechanism of Actions, H2 2016 25
  • Number of Products by Routes of Administration, H2 2016 27
  • Number of Products by Stage and Routes of Administration, H2 2016 27
  • Number of Products by Molecule Types, H2 2016 29
  • Number of Products by Stage and Molecule Types, H2 2016 29
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