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 2017年下半期
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Review, H2 2017

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 2017年下半期
出版社名 Global Markets Direct
発刊日 2017-12-29
体裁 PDF / 58 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 -
備考 ※ ご請求は、お申込日のTTSレートで円換算し、消費税を加えた金額になります。
※ 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
List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Overview
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Products under Development by Companies
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Companies Involved in Therapeutics Development
Abeona Therapeutics Inc
ArmaGen Inc
Axcentua Pharmaceuticals AB
BioMarin Pharmaceutical Inc
Laboratorios Del Dr Esteve SA
Lysogene SAS
Swedish Orphan Biovitrum AB
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Drug Profiles
ABO-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ABO-102 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AGT-184 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AXP-10711 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
BMN-250 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
EGT-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
EGT-201 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
LYSSAF-301 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
LYSSAF-302 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
OTL-201 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Proteins for Mucopolysaccharidosis Type IIIC - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant N-Acetylgalactosamine-6-Sulfatase Replacement for Mucopolysaccharidosis Type IIID - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecule for Sanfilippo Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SOBI-003 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Stem Cell Therapy to Activate NAGLU for Mucopolysaccharidosis IIIB - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Stem Cell Therapy to Activate NAGLU for Sanfilippo Syndrome - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Dormant Projects
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Discontinued Products
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Product Development Milestones
Featured News & Press Releases
Dec 20, 2017: Abeona Therapeutics Enrolls First Patient in ABO-101 Phase 1/2 Clinical Trial for MPS IIIB
Jul 05, 2017: FDA grants SOBI003 Orphan Drug Designation for the treatment of MPS IIIA
Jan 19, 2017: Abeona Therapeutics Receives Orphan Drug Designation in The European Union for ABO-101 Gene Therapy in Sanfilippo Syndrome Type B
Oct 19, 2016: European Commission Grants SOBI003 Orphan Designation for the Treatment of MPS IIIA
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)
Feb 25, 2016: ESTEVE Provides Update On EGT-201 For Sanfilippo B Syndrome
Jan 11, 2016: Abeona Therapeutics Announces Initial European Regulatory Approval for Phase 1/2 Gene Therapy Clinical Study for Patients With Sanfilippo Syndrome
May 20, 2015: PlasmaTech Biopharmaceuticals Announces Orphan Drug and Rare Pediatric Disease Designations For ABX-101 From FDA
Apr 02, 2014: Orphan designation granted for MPSIII
Jul 25, 2013: New stem cell gene therapy gives hope to prevent inherited neurological disease
Apr 11, 2013: Axcentua granted patent on ten solid forms of genistein
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List of Figures
Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), H2 2017
Number of Products under Development by Companies, H2 2017
Number of Products by Targets, H2 2017
Number of Products by Stage and Targets, H2 2017
Number of Products by Mechanism of Actions, H2 2017
Number of Products by Stage and Mechanism of Actions, H2 2017
Number of Products by Routes of Administration, H2 2017
Number of Products by Stage and Routes of Administration, H2 2017
Number of Products by Molecule Types, H2 2017
Number of Products by Stage and Molecule Types, H2 2017

List of Tables
Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), H2 2017
Number of Products under Development by Companies, H2 2017
Products under Development by Companies, H2 2017
Products under Development by Companies, H2 2017 (Contd..1), H2 2017
Number of Products by Stage and Target, H2 2017
Number of Products by Stage and Mechanism of Action, H2 2017
Number of Products by Stage and Route of Administration, H2 2017
Number of Products by Stage and Molecule Type, H2 2017
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Abeona Therapeutics Inc, H2 2017
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by ArmaGen Inc, H2 2017
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Axcentua Pharmaceuticals AB, H2 2017
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by BioMarin Pharmaceutical Inc, H2 2017
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Laboratorios Del Dr Esteve SA, H2 2017
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Lysogene SAS, H2 2017
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Swedish Orphan Biovitrum AB, H2 2017
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Dormant Projects, H2 2017
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Discontinued Products, H2 2017
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