世界の医療・医薬品産業レポート

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レーバー先天黒内障 (LCA) - パイプラインレビュー 2016年上半期
Leber Congenital Amaurosis (LCA) - Pipeline Review, H1 2016

レポートタイトル レーバー先天黒内障 (LCA) - パイプラインレビュー 2016年上半期
出版社名 Global Markets Direct
発刊日 2016-04-20
体裁 PDF / 57 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 レーバー先天黒内障 (LCA)
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
Table of Contents 2
List of Tables 4
List of Figures 4
Introduction 5
Global Markets Direct Report Coverage 5
Leber Congenital Amaurosis (LCA) Overview 6
Therapeutics Development 7
Pipeline Products for Leber Congenital Amaurosis (LCA) ? Overview 7
Pipeline Products for Leber Congenital Amaurosis (LCA) ? Comparative Analysis 8
Leber Congenital Amaurosis (LCA) ? Therapeutics under Development by Companies 9
Leber Congenital Amaurosis (LCA) ? Therapeutics under Investigation by Universities/Institutes 10
Leber Congenital Amaurosis (LCA) ? Pipeline Products Glance 11
Late Stage Products 11
Clinical Stage Products 12
Early Stage Products 13
Leber Congenital Amaurosis (LCA) ? Products under Development by Companies 14
Leber Congenital Amaurosis (LCA) ? Products under Investigation by Universities/Institutes 15
Leber Congenital Amaurosis (LCA) ? Companies Involved in Therapeutics Development 16
AmpliPhi Biosciences Corporation 16
Editas Medicine, Inc. 17
ProQR Therapeutics N.V. 18
QLT Inc. 19
Spark Therapeutics, Inc. 20
Leber Congenital Amaurosis (LCA) ? Therapeutics Assessment 21
Assessment by Monotherapy Products 21
Assessment by Target 22
Assessment by Mechanism of Action 24
Assessment by Route of Administration 26
Assessment by Molecule Type 28
Drug Profiles 30
Gene Therapy for Leber Congenital Amaurosis - Drug Profile 30
Product Description 30
Mechanism of Action 30
R&D Progress 30
Gene Therapy to Activate Retinal Guanylyl Cyclase-1 for Leber Congenital Amaurosis-1 - Drug Profile 31
Product Description 31
Mechanism of Action 31
R&D Progress 31
Gene Therapy to Activate RPE65 for Leber Congenital Amaurosis - Drug Profile 32
Product Description 32
Mechanism of Action 32
R&D Progress 32
QR-110 - Drug Profile 33
Product Description 33
Mechanism of Action 33
R&D Progress 33
tgAAG-76 - Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
voretigene neparvovec - Drug Profile 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
zuretinol acetate - Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
Leber Congenital Amaurosis (LCA) ? Recent Pipeline Updates 41
Leber Congenital Amaurosis (LCA) - Dormant Projects 45
Leber Congenital Amaurosis (LCA) ? Discontinued Products 46
Leber Congenital Amaurosis (LCA) ? Product Development Milestones 47
Featured News & Press Releases 47
May 04, 2015: Penn Study Indicates that Gene Therapy Efficacy for LCA is Dynamic: Improvement is Followed by Decline in Vision 47
May 01, 2015: Spark Therapeutics to Have Multiple Presentations at 2015 ARVO Meeting 48
Dec 05, 2014: QLT Announces Results From Proof-of-Concept Trial With Oral Synthetic cis-Retinoid (QLT091001) in Adult Subjects With Impaired Dark Adaptation and/or Impaired Low Luminance Vision 49
Nov 06, 2014: Spark Therapeutics Receives FDA Breakthrough Therapy Designation for Its Lead Product Candidate, SPK-RPE65 50
Sep 12, 2014: QLT Announces Positive Final Results From Phase 1b Retreatment Trial With Oral Synthetic cis-Retinoid (QLT091001) in Subjects With LCA or RP Due to Mutations in RPE65 or LRAT 51
Jul 14, 2014: QLT Announces Publication in The Lancet of Phase 1b Data for QLT091001 in Leber Congenital Amaurosis Due to LRAT or RPE65 Mutations 52
May 08, 2014: Spark Therapeutics Scientific Advisor to Deliver Keynote at the Association for Research in Vision and Ophthalmology’s 2014 Annual Meeting 53
Feb 27, 2014: QLT Announces Positive Preliminary Results From Phase 1b Retreatment Trial of QLT091001 in Subjects With Leber Congenital Amaurosis and Retinitis Pigmentosa Due to Mutations in LRAT or RPE65 53
Jan 14, 2014: Spark Therapeutics Achieves Recruitment Goal in Phase 3 Gene Therapy Clinical Study for Inherited Blindness 54
Appendix 56
Methodology 56
Coverage 56
Secondary Research 56
Primary Research 56
Expert Panel Validation 56
Contact Us 56
Disclaimer 57

List of Tables
Number of Products under Development for Leber Congenital Amaurosis (LCA), H1 2016 7
Number of Products under Development for Leber Congenital Amaurosis (LCA) ? Comparative Analysis, H1 2016 8
Number of Products under Development by Companies, H1 2016 9
Number of Products under Investigation by Universities/Institutes, H1 2016 10
Comparative Analysis by Late Stage Development, H1 2016 11
Comparative Analysis by Clinical Stage Development, H1 2016 12
Comparative Analysis by Early Stage Development, H1 2016 13
Products under Development by Companies, H1 2016 14
Products under Investigation by Universities/Institutes, H1 2016 15
Leber Congenital Amaurosis (LCA) ? Pipeline by AmpliPhi Biosciences Corporation, H1 2016 16
Leber Congenital Amaurosis (LCA) ? Pipeline by Editas Medicine, Inc., H1 2016 17
Leber Congenital Amaurosis (LCA) ? Pipeline by ProQR Therapeutics N.V., H1 2016 18
Leber Congenital Amaurosis (LCA) ? Pipeline by QLT Inc., H1 2016 19
Leber Congenital Amaurosis (LCA) ? Pipeline by Spark Therapeutics, Inc., H1 2016 20
Assessment by Monotherapy Products, H1 2016 21
Number of Products by Stage and Target, H1 2016 23
Number of Products by Stage and Mechanism of Action, H1 2016 25
Number of Products by Stage and Route of Administration, H1 2016 27
Number of Products by Stage and Molecule Type, H1 2016 29
Leber Congenital Amaurosis (LCA) Therapeutics ? Recent Pipeline Updates, H1 2016 41
Leber Congenital Amaurosis (LCA) ? Dormant Projects, H1 2016 45
Leber Congenital Amaurosis (LCA) ? Discontinued Products, H1 2016 46

List of Figures
Number of Products under Development for Leber Congenital Amaurosis (LCA), H1 2016 7
Number of Products under Development for Leber Congenital Amaurosis (LCA) ? Comparative Analysis, H1 2016 8
Number of Products under Development by Companies, H1 2016 9
Number of Products under Investigation by Universities/Institutes, H1 2016 10
Comparative Analysis by Clinical Stage Development, H1 2016 12
Comparative Analysis by Early Stage Products, H1 2016 13
Assessment by Monotherapy Products, H1 2016 21
Number of Products by Targets, H1 2016 22
Number of Products by Stage and Targets, H1 2016 22
Number of Products by Mechanism of Actions, H1 2016 24
Number of Products by Stage and Mechanism of Actions, H1 2016 24
Number of Products by Routes of Administration, H1 2016 26
Number of Products by Stage and Routes of Administration, H1 2016 26
Number of Products by Molecule Types, H1 2016 28
Number of Products by Stage and Molecule Types, H1 2016 28
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