世界の医療・医薬品産業レポート

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ムコ多糖症 III (MPS III) (サンフィリポ症候群 ) - パイプラインレビュー 2016年下半期
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Review, H2 2016

レポートタイトル ムコ多糖症 III (MPS III) (サンフィリポ症候群 ) - パイプラインレビュー 2016年下半期
出版社名 Global Markets Direct
発刊日 2016-12-21
体裁 PDF / 71 ページ
シングルユーザ価格 US$ 2,000
レポート分類 疾患別治験・パイプラインレポート
地域 グローバル
領域 遺伝子
疾患名 ムコ多糖症 III (MPS III) (サンフィリポ症候群 )
薬剤名 -
企業名 -
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※ 本レポートは、英文です。PDF(電子媒体)での納品となります。

レポート概要

このレポートは、世界の主要国における上記疾患の治療薬開発についての包括的情報、さまざまな開発ステージでの比較分析、薬剤ターゲット、作用機序(MoA)、投与方法(RoA)、分子タイプ別の薬剤評価、および最新情報、ニュースやプレスリリースなどを提供します。
また、上記疾患における治療薬開発に関わる主要企業概要、開発後期および中止プロジェクトの特集などを提供します。


Global Market Direct社のレポートは、20の治療領域および3000もの適応症をカバーした治験薬を特集しています。
本レポートは、Global Market Direct社独自のデータベース、企業/大学ウェブサイト、SEC書類、企業や大学のウェブサイトからのプレゼンテーションやプレスリリース、および業界特有の第三者情報源、自社分析を基にしたデータや情報を盛り込んでいます。


本レポートは、意思決定能力を高め、競争上の優位性を得るための効果的な戦略を立案するのに役立ちます。
ファーストインクラスやベストインクラス製品を作り出すため、新たなターゲットやMOAを特定することにより、R&Dパイプラインを強化します。


注)本レポートの一部のセクションは、該当疾患におけるデータの可用性や妥当性に基づいて、削除または変更される場合があります。

目次

Table of Contents
Table of Contents 2
List of Tables 5
List of Figures 6
Introduction 7
Global Markets Direct Report Coverage 7
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) Overview 8
Therapeutics Development 9
Pipeline Products for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Overview 9
Pipeline Products for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Comparative Analysis 10
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Therapeutics under Development by Companies 11
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Therapeutics under Investigation by Universities/Institutes 12
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Products Glance 13
Clinical Stage Products 13
Early Stage Products 14
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Products under Development by Companies 15
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Products under Investigation by Universities/Institutes 16
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Companies Involved in Therapeutics Development 17
Abeona Therapeutics Inc 17
Alexion Pharmaceuticals Inc 18
ArmaGen Inc 19
Axcentua Pharmaceuticals AB 20
BioMarin Pharmaceutical Inc 21
Dorphan SA 22
Laboratorios Del Dr Esteve SA 23
Lysogene SAS 24
Swedish Orphan Biovitrum AB 25
UniQure NV 26
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Therapeutics Assessment 27
Assessment by Monotherapy Products 27
Assessment by Target 28
Assessment by Mechanism of Action 30
Assessment by Route of Administration 32
Assessment by Molecule Type 34
Drug Profiles 36
ABO-101 - Drug Profile 36
Product Description 36
Mechanism Of Action 36
R&D Progress 36
ABO-102 - Drug Profile 38
Product Description 38
Mechanism Of Action 38
R&D Progress 38
AGT-184 - Drug Profile 41
Product Description 41
Mechanism Of Action 41
R&D Progress 41
AMT-110 - Drug Profile 42
Product Description 42
Mechanism Of Action 42
R&D Progress 42
AXP-10711 - Drug Profile 44
Product Description 44
Mechanism Of Action 44
R&D Progress 44
BMN-250 - Drug Profile 46
Product Description 46
Mechanism Of Action 46
R&D Progress 46
EGT-101 - Drug Profile 48
Product Description 48
Mechanism Of Action 48
R&D Progress 48
EGT-201 - Drug Profile 49
Product Description 49
Mechanism Of Action 49
R&D Progress 49
lesinidase alfa - Drug Profile 50
Product Description 50
Mechanism Of Action 50
R&D Progress 50
LYSSAF-301 - Drug Profile 53
Product Description 53
Mechanism Of Action 53
R&D Progress 53
LYSSAF-302 - Drug Profile 54
Product Description 54
Mechanism Of Action 54
R&D Progress 54
Proteins for Mucopolysaccharidosis Type IIIC - Drug Profile 55
Product Description 55
Mechanism Of Action 55
R&D Progress 55
Recombinant N-Acetylgalactosamine-6-Sulfatase Replacement for Mucopolysaccharidosis Type IIID - Drug Profile 56
Product Description 56
Mechanism Of Action 56
R&D Progress 56
Small Molecule for Sanfilippo Syndrome - Drug Profile 57
Product Description 57
Mechanism Of Action 57
R&D Progress 57
Small Molecule to Target Alpha-N-Acetylglucosaminidase for MPSIIIB - Drug Profile 58
Product Description 58
Mechanism Of Action 58
R&D Progress 58
SOBI-003 - Drug Profile 59
Product Description 59
Mechanism Of Action 59
R&D Progress 59
Stem Cell Therapy for Mucopolysaccharidosis Type IIIA - Drug Profile 60
Product Description 60
Mechanism Of Action 60
R&D Progress 60
Stem Cell Therapy for Sanfilippo Syndrome - Drug Profile 61
Product Description 61
Mechanism Of Action 61
R&D Progress 61
Stem Cell Therapy to Activate NAGLU for Mucopolysaccharidosis IIIB - Drug Profile 62
Product Description 62
Mechanism Of Action 62
R&D Progress 62
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Dormant Projects 63
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Discontinued Products 64
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Product Development Milestones 65
Featured News & Press Releases 65
Oct 19, 2016: European Commission Grants SOBI003 Orphan Designation for the Treatment of MPS IIIA 65
May 24, 2016: Abeona Therapeutics Announces FDA Allowance of Investigational New Drug for Phase 1/2 Clinical Study With ABO-101 Gene Therapy for Patients With Sanfilippo Syndrome Type B (MPS IIIB) 65
Feb 25, 2016: ESTEVE Provides Update On EGT-201 For Sanfilippo B Syndrome 66
Jan 11, 2016: Abeona Therapeutics Announces Initial European Regulatory Approval for Phase 1/2 Gene Therapy Clinical Study for Patients With Sanfilippo Syndrome 67
May 20, 2015: PlasmaTech Biopharmaceuticals Announces Orphan Drug and Rare Pediatric Disease Designations For ABX-101 From FDA 68
Apr 02, 2014: Orphan designation granted for MPSIII 68
Jul 25, 2013: New stem cell gene therapy gives hope to prevent inherited neurological disease 68
Apr 11, 2013: Axcentua granted patent on ten solid forms of genistein 69
Appendix 70
Methodology 70
Coverage 70
Secondary Research 70
Primary Research 70
Expert Panel Validation 70
Contact Us 70
Disclaimer 71

List of Figures
Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), H2 2016 9
Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Comparative Analysis, H2 2016 10
Number of Products under Development by Companies, H2 2016 11
Comparative Analysis by Early Stage Products, H2 2016 14
Assessment by Monotherapy Products, H2 2016 27
Number of Products by Targets, H2 2016 28
Number of Products by Stage and Targets, H2 2016 28
Number of Products by Top 10 Mechanism of Actions, H2 2016 30
Number of Products by Stage and Top 10 Mechanism of Actions, H2 2016 30
Number of Products by Routes of Administration, H2 2016 32
Number of Products by Stage and Routes of Administration, H2 2016 32
Number of Products by Molecule Types, H2 2016 34
Number of Products by Stage and Molecule Types, H2 2016 34

List of Tables
Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), H2 2016 9
Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Comparative Analysis, H2 2016 10
Number of Products under Development by Companies, H2 2016 11
Number of Products under Investigation by Universities/Institutes, H2 2016 12
Comparative Analysis by Clinical Stage Development, H2 2016 13
Comparative Analysis by Early Stage Development, H2 2016 14
Products under Development by Companies, H2 2016 15
Products under Investigation by Universities/Institutes, H2 2016 16
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Abeona Therapeutics Inc, H2 2016 17
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Alexion Pharmaceuticals Inc, H2 2016 18
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by ArmaGen Inc, H2 2016 19
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Axcentua Pharmaceuticals AB, H2 2016 20
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by BioMarin Pharmaceutical Inc, H2 2016 21
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Dorphan SA, H2 2016 22
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Laboratorios Del Dr Esteve SA, H2 2016 23
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Lysogene SAS, H2 2016 24
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Swedish Orphan Biovitrum AB, H2 2016 25
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by UniQure NV, H2 2016 26
Assessment by Monotherapy Products, H2 2016 27
Number of Products by Stage and Target, H2 2016 29
Number of Products by Stage and Mechanism of Action, H2 2016 31
Number of Products by Stage and Route of Administration, H2 2016 33
Number of Products by Stage and Molecule Type, H2 2016 35
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Dormant Projects, H2 2016 63
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Discontinued Products, H2 2016 64
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