ムコ多糖症III型(MPS III)(サンフィリッポ症候群)(Mucopolysaccharidosis III (Sanfilippo Syndrome)):治療薬開発パイプライン動向(2014年下半期版)

◆英語タイトル:Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline Review, H2 2014
◆商品コード:GMDHC5826IDB
◆発行会社(リサーチ会社):Global Markets Direct
◆発行日:2014年12月10日
◆ページ数:59
◆レポート言語:英語
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当調査レポートでは、世界におけるムコ多糖症III型(MPS III)(サンフィリッポ症候群)(Mucopolysaccharidosis III (Sanfilippo Syndrome))の治療薬開発パイプライン動向について調査・分析し、企業で開発中の治療薬、大学/研究機関で研究中の治療薬、後期段階の製品、治験段階の製品、初期段階の製品、単剤治療薬/併用治療薬/標的別/作用機序別/投与経路別/分子の種類別治療薬の評価、各薬剤のプロファイル(説明、作用機序、研究開発の進展状況)などのデータをお届けいたします。

・イントロダクション
・ムコ多糖症III型(MPS III)(サンフィリッポ症候群)(Mucopolysaccharidosis III (Sanfilippo Syndrome))の概要
・ムコ多糖症III型(MPS III)(サンフィリッポ症候群)(Mucopolysaccharidosis III (Sanfilippo Syndrome))治療薬の開発
  - パイプライン製品の概要
  - パイプライン製品の比較分析
・企業で開発中の治療薬
・大学/研究機関で研究中の治療薬
・ムコ多糖症III型(MPS III)(サンフィリッポ症候群)(Mucopolysaccharidosis III (Sanfilippo Syndrome))パイプライン製品の概略
  - 後期段階の製品
  - 治験段階の製品
  - 初期段階の製品
・製品
・大学/研究機関別、研究中の製品
・ムコ多糖症III型(MPS III)(サンフィリッポ症候群)(Mucopolysaccharidosis III (Sanfilippo Syndrome))治療薬開発に取り組んでいる企業:企業別製品パイプライン
・ムコ多糖症III型(MPS III)(サンフィリッポ症候群)(Mucopolysaccharidosis III (Sanfilippo Syndrome))治療薬の評価
  - 単剤治療薬
  - 併用治療薬
  - 標的別
  - 作用機序(MoA)別
  - 投与経路(RoA)別
  - 分子の種類別
・各薬剤のプロファイル:説明、作用機序、研究開発(R&D)の進展状況
・パイプライン製品の最新動向
・開発休止中の製品/プロジェクト
・開発が中止された製品
・製品開発のマイルストーン
【レポートの概要】

Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Pipeline Review, H2 2014

Summary

Global Markets Direct’s, ‘Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Pipeline Review, H2 2014’, provides an overview of the Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)’s therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome), complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) and special features on late-stage 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, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct’s team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis.

The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products.

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

Scope

- The report provides a snapshot of the global therapeutic landscape of Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
- The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities
- The report reviews key players involved in the therapeutics development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) and enlists all their major and minor projects
- The report summarizes all the dormant and discontinued pipeline projects
- A review of the Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) products under development by companies and universities/research institutes based on information derived from company and industry-specific sources
- Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages
- A detailed assessment of monotherapy and combination therapy pipeline projects
- Coverage of the Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) pipeline on the basis of target, MoA, route of administration and molecule type
- Latest news and deals relating related to pipeline products

Reasons to buy

- Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
- Develop strategic initiatives by understanding the focus areas of leading companies
- Identify and understand important and diverse types of therapeutics under development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome)
- Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
- Devise corrective measures for pipeline projects by understanding Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) 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

【レポートの目次】

Table of Contents
Table of Contents 2
List of Tables 5
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) Overview 7
Therapeutics Development 8
Pipeline Products for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Overview 8
Pipeline Products for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Comparative Analysis 9
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Therapeutics under Development by Companies 10
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Therapeutics under Investigation by Universities/Institutes 11
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Pipeline Products Glance 12
Clinical Stage Products 12
Early Stage Products 13
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Products under Development by Companies 14
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Products under Investigation by Universities/Institutes 15
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Companies Involved in Therapeutics Development 16
ArmaGen Technologies, Inc. 16
Axcentua Pharmaceuticals AB 17
BioMarin Pharmaceutical Inc. 18
Dorphan S.A. 19
Laboratorios Del Dr. Esteve S.A. 20
Shire Plc 21
Synageva BioPharma Corp. 22
uniQure N.V. 23
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Therapeutics Assessment 24
Assessment by Monotherapy Products 24
Assessment by Target 25
Assessment by Mechanism of Action 26
Assessment by Route of Administration 28
Assessment by Molecule Type 30
Drug Profiles 32
ABX-A – Drug Profile 32
Product Description 32
Mechanism of Action 32
R&D Progress 32
ABX-B – Drug Profile 33
Product Description 33
Mechanism of Action 33
R&D Progress 33
AGT-184 – Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
AMT-110 – Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
AXP-10711 – Drug Profile 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
BMN-250 – Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
Gene Therapy for Sanfilippo A Syndrome – Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
Gene Therapy to Activate Alpha-N-Acetylglucosaminidase for Sanfilippo Type B Syndrome – Drug Profile 40
Product Description 40
Mechanism of Action 40
R&D Progress 40
HGT-3010 – Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
SAF-301 – Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
SBC-103 – Drug Profile 43
Product Description 43
Mechanism of Action 43
R&D Progress 43
SHP-610 – Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
Small Molecule for Sanfilippo Syndrome – Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
Small Molecule to Target Alpha-N-Acetylglucosaminidase for MPSIIIB – Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
Stem Cell Therapy for Sanfilippo Syndrome – Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Recent Pipeline Updates 48
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Dormant Projects 51
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Product Development Milestones 52
Featured News & Press Releases 52
Apr 02, 2014: Orphan designation granted for MPSIII 52
Feb 12, 2014: Synageva BioPharma Highlights SBC-103 Data at the Lysosomal Disease Network WORLD Symposium 52
Feb 11, 2014: BioMarin Announces Selection of NAGLU Fusion Protein Drug Development Candidate BMN 250 for the Treatment of Sanfilippo B 54
Jan 29, 2014: Synageva BioPharma Announces Data Presentations On SBC-103 at the 10th Annual Lysosomal Disease Network World Symposium 55
Jul 25, 2013: New stem cell gene therapy gives hope to prevent inherited neurological disease 55
Apr 11, 2013: Axcentua granted patent on ten solid forms of genistein 57
Jan 30, 2013: Synageva BioPharma To Present Scientific Data On SBC-103 At 9th Annual LDN WORLD Symposium 57
Jan 30, 2012: Synageva To Present Preclinical Data Of SBC-103 At Lysosomal Disease Network World Symposium 57
Appendix 58
Methodology 58
Coverage 58
Secondary Research 58
Primary Research 58
Expert Panel Validation 58
Contact Us 59
Disclaimer 59

[List of Tables]
Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome), H2 2014 8
Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Comparative Analysis, H2 2014 9
Number of Products under Development by Companies, H2 2014 10
Number of Products under Investigation by Universities/Institutes, H2 2014 11
Comparative Analysis by Clinical Stage Development, H2 2014 12
Comparative Analysis by Early Stage Development, H2 2014 13
Products under Development by Companies, H2 2014 14
Products under Investigation by Universities/Institutes, H2 2014 15
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by ArmaGen Technologies, Inc., H2 2014 16
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Axcentua Pharmaceuticals AB, H2 2014 17
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by BioMarin Pharmaceutical Inc., H2 2014 18
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Dorphan S.A., H2 2014 19
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Laboratorios Del Dr. Esteve S.A., H2 2014 20
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Shire Plc, H2 2014 21
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by Synageva BioPharma Corp., H2 2014 22
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Pipeline by uniQure N.V., H2 2014 23
Assessment by Monotherapy Products, H2 2014 24
Number of Products by Stage and Target, H2 2014 25
Number of Products by Stage and Mechanism of Action, H2 2014 27
Number of Products by Stage and Route of Administration, H2 2014 29
Number of Products by Stage and Molecule Type, H2 2014 31
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) Therapeutics - Recent Pipeline Updates, H2 2014 48
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Dormant Projects, H2 2014 51

[List of Figures]
Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome), H2 2014 8
Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Comparative Analysis, H2 2014 9
Number of Products under Development by Companies, H2 2014 10
Number of Products under Investigation by Universities/Institutes, H2 2014 11
Comparative Analysis by Clinical Stage Development, H2 2014 12
Comparative Analysis by Early Stage Products, H2 2014 13
Assessment by Monotherapy Products, H2 2014 24
Number of Products by Stage and Top 10 Targets, H2 2014 25
Number of Products by Top 10 Mechanism of Actions, H2 2014 26
Number of Products by Stage and Top 10 Mechanism of Actions, H2 2014 27
Number of Products by Top 10 Routes of Administration, H2 2014 28
Number of Products by Stage and Top 10 Routes of Administration, H2 2014 29
Number of Products by Top 10 Molecule Types, H2 2014 30
Number of Products by Stage and Top 10 Molecule Types, H2 2014 31

【掲載企業】

ArmaGen Technologies, Inc.
Axcentua Pharmaceuticals AB
BioMarin Pharmaceutical Inc.
Dorphan S.A.
Laboratorios Del Dr. Esteve S.A.
Shire Plc
Synageva BioPharma Corp.
uniQure N.V.

【免責事項】
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