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Hereditary Hematological Disorders Drug Development Pipeline Review, 2018

遺伝性血液疾患薬剤開発パイプラインレビュー2018

レポート概要

本レポートは、遺伝性血液疾患のパイプライン展望の概要を提供します。サラセミア、血友病Bおよび鎌状赤血球貧血の治療開発に関わる主要な開発企業および治療中の治療薬に関する包括的な情報を提供し、休止および中止製品も特集しています。

サラセミアとは、身体がヘモグロビンおよび赤血球を生成する能力に影響を及ぼす、遺伝性血液疾患のグループを指します。症状としては、蒼白さ、頻繁な感染症および黄疸が挙げられます。予知因子には家族歴が含まれ、治療には、輸血や骨髄移植が含まれます。この適応症の開発には40の製品があります。

血友病Bは、血液凝固因子IXの欠乏によって引き起こされる遺伝性出血障害です。症状としては、関節出血、関連する痛みおよび腫れ、挫傷、鼻血および傷害、および手術による長期間の出血が挙げられます。治療には、欠陥のある凝固因子の置換が含まれます。この適応症の開発には42の製品があります。

鎌状赤血球貧血は、体の周りに酸素を運ぶ赤血球が異常に発達する遺伝性血液障害です。 徴候および症状には、貧血、成長の遅延、視力の問題、痛みおよび頻繁な感染が含まれます。治療には、抗生物質、鎮痛剤、輸血、幹細胞移植が含まれます。この適応症の開発には68の製品があります。

遺伝性血液疾患のための開発中の製品によって作用する分子標的には、凝固因子、ヒストンデアセチラーゼおよびプロテインキナーゼが含まれます。このパイプラインスペースで活動している企業には、Sangamo Therapeutics社、Gamida Cell社、Bluebird Bio社などがあります。

■調査範囲

  • 各パイプライン内において、最も活発に活動しているのはどの企業でしょうか?
  • 各適応症において、各開発段階で最も優れた薬物療法的アプローチはどれでしょうか?
  • このパイプライン内において、大学や組織は製薬企業と比べてどの程度重要な役割を果たすでしょうか?
  • この疾患分野において起きた最も重要なR&Dの画期的出来事や、データの公表は何でしょうか?

レポート詳細

目次

1 Table of Contents
1.1 List of Tables
1.2 List of Figures

2 Introduction
2.1 Hereditary Hematological Disorders Report Coverage
2.2 Thalassemia - Overview
2.3 Hemophilia B - Overview
2.4 Sickle Cell Disease - Overview

3 Therapeutics Development
3.1 Thalassemia
3.2 Hemophilia B - Therapeutics Development
3.3 Sickle Cell Disease - Therapeutics Development

4 Therapeutics Assessment
4.1 Thalassemia - Therapeutics Assessment
4.2 Hemophilia B - Therapeutics Assessment
4.3 Sickle Cell Disease - Therapeutics Assessment

5 Companies Involved in Therapeutics Development
5.1 Thalassemia - Companies Involved in Therapeutics Development
5.2 Hemophilia B - Companies Involved in Therapeutics Development
5.3 Sickle Cell Disease - Companies Involved in Therapeutics Development

6 Dormant Projects
6.1 Thalassemia - Dormant Projects
6.2 Hemophilia B - Dormant Projects
6.3 Sickle Cell Disease - Dormant Projects

7 Discontinued Products
7.1 Thalassemia - Discontinued Products
7.2 Hemophilia B - Discontinued Products

8 Product Development Milestones
8.1 Thalassemia - Product Development Milestones
8.2 Hemophilia B - Product Development Milestones
8.3 Sickle Cell Disease - Product Development Milestones

9 Appendix
9.1 Methodology
9.2 Coverage
9.3 Secondary Research
9.4 Primary Research
9.5 Expert Panel Validation
9.6 Contact Us
9.7 Disclaimer

1.1 List of Tables
Table 1: Number of Products under Development for Thalassemia
Table 2: Number of Products under Development by Companies, Thalassemia
Table 3: Number of Products under Development by Universities/Institutes, Thalassemia
Table 4: Products under Development by Companies, Thalassemia
Table 5: Products under Development by Universities/Institutes, Thalassemia
Table 6: Number of Products under Development for Hemophilia B
Table 7: Number of Products under Development by Companies, Hemophilia B
Table 8: Number of Products under Development by Universities/Institutes, Hemophilia B
Table 9: Products under Development by Companies, Hemophilia B
Table 10: Products under Development by Universities/Institutes, Hemophilia B
Table 11: Number of Products under Development for Sickle Cell Disease
Table 12: Number of Products under Development by Companies, Sickle Cell Disease
Table 13: Number of Products under Development by Universities/Institutes, Sickle Cell Disease
Table 14: Products under Development by Companies, Sickle Cell Disease
Table 15: Products under Development by Universities/Institutes, Sickle Cell Disease
Table 16: Number of Products by Stage and Target, Thalassemia
Table 17: Number of Products by Stage and Mechanism of Action, Thalassemia
Table 18: Number of Products by Stage and Route of Administration, Thalassemia
Table 19: Number of Products by Stage and Molecule Type, Thalassemia
Table 20: Number of Products by Stage and Target, Hemophilia B
Table 21: Number of Products by Stage and Mechanism of Action, Hemophilia B
Table 22: Number of Products by Stage and Route of Administration, Hemophilia B
Table 23: Number of Products by Stage and Molecule Type, Hemophilia B
Table 24: Number of Products by Stage and Target, H2 2018, Sickle Cell Disease
Table 25: Number of Products by Stage and Mechanism of Action, Sickle Cell Disease
Table 26: Number of Products by Stage and Route of Administration, Sickle Cell Disease
Table 27: Number of Products by Stage and Molecule Type, Sickle Cell Disease
Table 28: Thalassemia - Pipeline by Acceleron Pharma Inc
Table 29: Thalassemia - Pipeline by Agios Pharmaceuticals Inc
Table 30: Thalassemia - Pipeline by bluebird bio Inc
Table 31: Thalassemia - Pipeline by Cadila Healthcare Ltd,
Table 32: Thalassemia - Pipeline by Calimmune Inc
Table 33: Thalassemia - Pipeline by CRISPR Therapeutics
Table 34: Thalassemia - Pipeline by Editas Medicine Inc
Table 35: Thalassemia - Pipeline by Errant Gene Therapeutics LLC
Table 36: Thalassemia - Pipeline by Gamida Cell Ltd
Table 37: Thalassemia - Pipeline by Gilead Sciences Inc
Table 38: Thalassemia - Pipeline by Incyte Corp
Table 39: Thalassemia - Pipeline by Ionis Pharmaceuticals Inc
Table 40: Thalassemia - Pipeline by Kiadis Pharma NV
Table 41: Thalassemia - Pipeline by La Jolla Pharmaceutical Company
Table 42: Thalassemia - Pipeline by Merck & Co Inc
Table 43: Thalassemia - Pipeline by Poseida Therapeutics Inc
Table 44: Thalassemia - Pipeline by Protagonist Therapeutics Inc
Table 45: Thalassemia - Pipeline by Sangamo Therapeutics Inc
Table 46: Thalassemia - Pipeline by Silence Therapeutics Plc
Table 47: Thalassemia - Pipeline by Vifor Pharma AG
Table 48: Hemophilia B - Pipeline by Amarna Therapeutics BV
Table 49: Hemophilia B - Pipeline by Bayer AG
Table 50: Hemophilia B - Pipeline by Bioverativ Inc
Table 51: Hemophilia B - Pipeline by Catalyst Biosciences Inc
Table 52: Hemophilia B - Pipeline by China Biologic Products Inc
Table 53: Hemophilia B - Pipeline by CSL Ltd

1.2 List of Figures
Figure 1: Number of Products under Development for Thalassemia
Figure 2: Number of Products under Development by Companies, Thalassemia
Figure 3: Number of Products under Development by Universities/Institutes, Thalassemia
Figure 4: Number of Products under Development for Hemophilia B
Figure 5: Number of Products under Development by Companies, Hemophilia B
Figure 6: Number of Products under Development by Universities/Institutes, Hemophilia B
Figure 7: Number of Products under Development for Sickle Cell Disease
Figure 8: Number of Products under Development by Companies, Sickle Cell Disease
Figure 9: Number of Products under Development by Universities/Institutes, Sickle Cell Disease
Figure 10: Number of Products by Top 10 Targets, Thalassemia
Figure 11: Number of Products by Stage and Top 10 Targets, Thalassemia
Figure 12: Number of Products by Top 10 Mechanism of Actions, Thalassemia
Figure 13: Number of Products by Stage and Top 10 Mechanism of Actions, Thalassemia
Figure 14: Number of Products by Routes of Administration, Thalassemia
Figure 15: Number of Products by Stage and Routes of Administration, Thalassemia
Figure 16: Number of Products by Molecule Types, Thalassemia
Figure 17: Number of Products by Stage and Molecule Types, Thalassemia
Figure 18: Number of Products by Targets, Hemophilia B
Figure 19: Number of Products by Stage and Targets, Hemophilia B
Figure 20: Number of Products by Mechanism of Actions, Hemophilia B
Figure 21: Number of Products by Stage and Mechanism of Actions, Hemophilia B
Figure 22: Number of Products by Routes of Administration, Hemophilia B
Figure 23: Number of Products by Stage and Routes of Administration, Hemophilia B
Figure 24: Number of Products by Molecule Types, Hemophilia B
Figure 25: Number of Products by Stage and Molecule Types, Hemophilia B
Figure 26: Number of Products by Top 10 Targets, Sickle Cell Disease
Figure 27: Number of Products by Stage and Top 10 Targets, Sickle Cell Disease
Figure 28: Number of Products by Top 10 Mechanism of Actions, Sickle Cell Disease
Figure 29: Number of Products by Stage and Top 10 Mechanism of Actions, Sickle Cell Disease
Figure 30: Number of Products by Routes of Administration, Sickle Cell Disease
Figure 31: Number of Products by Stage and Routes of Administration, Sickle Cell Disease
Figure 32: Number of Products by Top 10 Molecule Types, Sickle Cell Disease
Figure 33: Number of Products by Stage and Top 10 Molecule Types, Sickle Cell Disease

発刊日

2018/10/25

体裁

PDF / 143ページ

販売価格

3,995USD

発行

GBI Research

備考

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