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サラセミア:パイプライン製品の分析

Thalassemia - Pipeline Review, H1 2017

発行 Global Markets Direct 商品コード 245974
出版日 ページ情報 英文 140 Pages
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サラセミア:パイプライン製品の分析 Thalassemia - Pipeline Review, H1 2017
出版日: 2017年03月22日 ページ情報: 英文 140 Pages
概要

サラセミアは、ヘモグロビンおよび赤血球の産生に影響を及ぼす遺伝的な血液の疾病であり、ぐずつき、顔色不良、頻繁な感染、発育障害、食欲不振および黄疸などが症状として挙げられます。発病要因は遺伝的であり、治療には輸血および骨髄移植などが行われます。

当レポートでは、サラセミアに対する治療薬の開発状況について調査分析し、パイプライン製品の概要、治験の段階別の製品の概要、主要企業プロファイル、薬剤のプロファイル、パイプライン製品の最新動向、最新ニュースとプレスリリースなどについて、体系的な情報を提供しています。

イントロダクション

  • 調査範囲

サラセミアの概要

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

サラセミア:企業で開発中の治療薬

サラセミア:大学/機関で研究中の治療薬

サラセミア:パイプライン製品の概況

  • 後期段階の製品
  • 臨床段階の製品
  • 初期段階の製品

サラセミア:企業で開発中の製品

サラセミア:大学/機関で研究中の製品

サラセミアの治療薬開発に従事している企業

  • Acceleron Pharma, Inc.
  • Acetylon Pharmaceuticals, Inc.
  • Acino Pharma AG
  • Agios Pharmaceuticals, Inc.
  • Alnylam Pharmaceuticals, Inc.
  • Bellicum Pharmaceuticals, Inc.
  • Bluebird bio, Inc.
  • Editas Medicine, Inc.
  • Emmaus Medical, Inc.
  • Errant Gene Therapeutics, LLC
  • Gamida Cell Ltd.
  • Incyte Corporation
  • Ionis Pharmaceuticals, Inc.
  • IRBM Science Park SpA
  • Johnson & Johnson
  • Kiadis Pharma B.V.
  • La Jolla Pharmaceutical Company
  • Merck & Co., Inc.
  • PharmaEssentia Corporation
  • Protagonist Therapeutics Inc.
  • Sangamo BioSciences, Inc.

サラセミア: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

サラセミア:最近のパイプライン動向

サラセミア:休止中のプロジェクト

サラセミア: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

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: GMDHC9080IDB

Summary

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

Thalassemia is a group of inherited blood disorders that affect the body's ability to produce hemoglobin and red blood cells. Symptoms include fussiness, paleness, frequent infections, failure to thrive, poor appetite and jaundice. Predisposing factors include family history. Treatment includes blood transfusions and bone marrow transplant.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Thalassemia - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Thalassemia (Hematological 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 Thalassemia (Hematological Disorders) pipeline guide also reviews of key players involved in therapeutic development for Thalassemia and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, Preclinical and Discovery stages are 2, 5, 6, 17 and 4 respectively. Similarly, the Universities portfolio in Phase II, Preclinical and Discovery stages comprises 1, 2 and 1 molecules, respectively.

Thalassemia (Hematological 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 Thalassemia (Hematological Disorders).
  • The pipeline guide reviews pipeline therapeutics for Thalassemia (Hematological 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 Thalassemia (Hematological Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Thalassemia (Hematological 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 Thalassemia (Hematological 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 Thalassemia (Hematological 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 Thalassemia (Hematological 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

  • Table of Contents
    • List of Tables
    • List of Figures
  • Introduction
    • Global Markets Direct Report Coverage
  • Thalassemia - Overview
  • Thalassemia - Therapeutics Development
    • Pipeline Overview
    • Pipeline by Companies
    • Pipeline by Universities/Institutes
    • Products under Development by Companies
    • Products under Development by Universities/Institutes
  • Thalassemia - Therapeutics Assessment
    • Assessment by Target
    • Assessment by Mechanism of Action
    • Assessment by Route of Administration
    • Assessment by Molecule Type
  • Thalassemia - Companies Involved in Therapeutics Development
    • Acceleron Pharma Inc
    • Agios Pharmaceuticals Inc
    • Alnylam Pharmaceuticals Inc
    • bluebird bio Inc
    • Calimmune Inc
    • CRISPR Therapeutics
    • Editas Medicine Inc
    • Errant Gene Therapeutics LLC
    • Gamida Cell Ltd
    • Gilead Sciences Inc
    • Incyte Corp
    • Ionis Pharmaceuticals Inc
    • IRBM Science Park SpA
    • Johnson & Johnson
    • Kiadis Pharma NV
    • La Jolla Pharmaceutical Company
    • Merck & Co Inc
    • PharmaEssentia Corp
    • Protagonist Therapeutics Inc
    • Sangamo Therapeutics Inc
    • Zydus Cadila Healthcare Ltd
  • Thalassemia - Drug Profiles
    • (decitabine + tetrahydrouridine) - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • ACY-957 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • AG-348 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • ALN-TMP - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • ambrisentan - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • ATIR-201 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • BB-305 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • benserazide - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • CAL-H - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • CNTO-530 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • CordIn - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Drugs to Chelate Iron for Bone Degeneration, Postmenopausal Osteoporosis and Thalassemia - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy for Sickle Cell Disease and Thalassemia - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Gene Therapy to Activate IGF2BP1 for Sickle Cell Disease and Thalassemia - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • GSK-2696277 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • IMR-687 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • IONISTMPRSS-6LRx - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • LJPC-401 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • luspatercept - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • M-009 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • M-012 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • NiCord - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • PEG-EPO - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • PHBB-101 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • PTG-300 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • RCY-1497 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • RNAi Gene Therapy for Sickle Cell Disease and Beta Thalassemia - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • ruxolitinib phosphate - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • SCD-101 - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • sirolimus - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecule for Beta Thalassemia and Sickle Cell Anemia - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecule to Inhibit Histone Deacetylase 1 for Beta Thalassemia - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Small Molecules to Inhibit PRMT5 for Haemoglobinopathies and Solid Tumours - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • sotatercept - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Stem Cell Therapy for Beta Thalassemia - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Stem Cell Therapy for Beta Thalassemia and Sickle Cell Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Stem Cell Therapy for Beta Thalassemia and Sickle Cell Disease - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
    • Thalagen - Drug Profile
      • Product Description
      • Mechanism Of Action
      • R&D Progress
  • Thalassemia - Dormant Projects
  • Thalassemia - Product Development Milestones
    • Featured News & Press Releases
      • Feb 06, 2017: Kiadis Pharma Provides Update on ATIR201
      • Dec 15, 2016: Kiadis Pharma announces initiation of Phase I/II clinical trial with ATIR201 for thalassemia
      • Dec 14, 2016: bluebird bio Announces First Patient Treated with LentiGlobin Drug Product in Northstar-2 (HGB-207) Phase 3 Trial of Patients with Transfusion-Dependent B-Thalassemia
      • Dec 06, 2016: bluebird bio Provides Updates on HSC Gene Therapy Programs
      • Dec 05, 2016: Acceleron and Celgene Announce Updated Results from Phase 2 Studies of Luspatercept in Beta-Thalassemia Presented at the 58th Annual Meeting of the American Society of Hematology
      • Dec 04, 2016: Acetylon Presents Preclinical Data Demonstrating the Utility of Selective HDAC1,2 Inhibition by ACY-957 to Induce Gamma Globin (HBG) Protein Expression for the Treatment of Sickle Cell Disease and Beta-Thalassemia
      • Dec 03, 2016: bluebird bio Presents New Data from HGB-205 Study of LentiGlobin Drug Product in Patients with Transfusion-Dependent B-Thalassemia and Severe Sickle Cell Disease at American Society of Hematology (ASH) Annual Meeting
      • Dec 02, 2016: Acetylon to present data on ACY-957 at the American Society of Hematology Annual Meeting
      • Nov 21, 2016: CRISPR Therapeutics Announces Two Presentations Demonstrating the Potential for CRISPR Gene Editing To Treat Sickle Cell Disease and B-Thalassemia
      • Nov 03, 2016: bluebird bio to Present New Data from Three LentiGlobin Clinical Studies at American Society of Hematology (ASH) Annual Meeting
      • Oct 13, 2016: bluebird bio Provides Update on LentiGlobin Programs and Research and Development Strategy at Gene Therapy Day
      • Sep 21, 2016: LentiGlobin Investigational Gene Therapy for Transfusion-Dependent Beta-Thalassemia Accepted into European Medicines Agency's PRIME Program
      • Sep 08, 2016: bluebird bio Opens Phase 3 Study of LentiGlobin Drug Product in Patients with Transfusion-Dependent Beta-Thalassemia
      • Sep 07, 2016: La Jolla Pharmaceutical Company Reports Positive Results from Phase 1 Study of LJPC-401
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
    • Disclaimer

List of Tables

  • Number of Products under Development for Thalassemia, H1 2017
  • Number of Products under Development by Companies, H1 2017
  • Number of Products under Development by Companies, H1 2017 (Contd..1), H1 2017
  • Number of Products under Development by Universities/Institutes, H1 2017
  • Products under Development by Companies, H1 2017
  • Products under Development by Companies, H1 2017 (Contd..1), 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
  • Thalassemia - Pipeline by Acceleron Pharma Inc, H1 2017
  • Thalassemia - Pipeline by Agios Pharmaceuticals Inc, H1 2017
  • Thalassemia - Pipeline by Alnylam Pharmaceuticals Inc, H1 2017
  • Thalassemia - Pipeline by bluebird bio Inc, H1 2017
  • Thalassemia - Pipeline by Calimmune Inc, H1 2017
  • Thalassemia - Pipeline by CRISPR Therapeutics, H1 2017
  • Thalassemia - Pipeline by Editas Medicine Inc, H1 2017
  • Thalassemia - Pipeline by Errant Gene Therapeutics LLC, H1 2017
  • Thalassemia - Pipeline by Gamida Cell Ltd, H1 2017
  • Thalassemia - Pipeline by Gilead Sciences Inc, H1 2017
  • Thalassemia - Pipeline by Incyte Corp, H1 2017
  • Thalassemia - Pipeline by Ionis Pharmaceuticals Inc, H1 2017
  • Thalassemia - Pipeline by IRBM Science Park SpA, H1 2017
  • Thalassemia - Pipeline by Johnson & Johnson, H1 2017
  • Thalassemia - Pipeline by Kiadis Pharma NV, H1 2017
  • Thalassemia - Pipeline by La Jolla Pharmaceutical Company, H1 2017
  • Thalassemia - Pipeline by Merck & Co Inc, H1 2017
  • Thalassemia - Pipeline by PharmaEssentia Corp, H1 2017
  • Thalassemia - Pipeline by Protagonist Therapeutics Inc, H1 2017
  • Thalassemia - Pipeline by Sangamo Therapeutics Inc, H1 2017
  • Thalassemia - Pipeline by Zydus Cadila Healthcare Ltd, H1 2017
  • Thalassemia - Dormant Projects, H1 2017

List of Figures

  • Number of Products under Development for Thalassemia, 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 Top 10 Targets, H1 2017
  • Number of Products by Stage and Top 10 Targets, H1 2017
  • Number of Products by Top 10 Mechanism of Actions, H1 2017
  • Number of Products by Stage and Top 10 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 Top 10 Molecule Types, H1 2017
  • Number of Products by Stage and Top 10 Molecule Types, H1 2017
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