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年下半期
Leber Congenital Amaurosis (LCA) - Pipeline Review, H2 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年下半期
出版社名 Global Markets Direct
発刊日 2016-09-30
体裁 PDF / 61 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 5
Introduction 6
Global Markets Direct Report Coverage 6
Leber Congenital Amaurosis (LCA) Overview 7
Therapeutics Development 8
Pipeline Products for Leber Congenital Amaurosis (LCA) - Overview 8
Pipeline Products for Leber Congenital Amaurosis (LCA) - Comparative Analysis 9
Leber Congenital Amaurosis (LCA) - Therapeutics under Development by Companies 10
Leber Congenital Amaurosis (LCA) - Therapeutics under Investigation by Universities/Institutes 11
Leber Congenital Amaurosis (LCA) - Pipeline Products Glance 12
Late Stage Products 12
Clinical Stage Products 13
Early Stage Products 14
Leber Congenital Amaurosis (LCA) - Products under Development by Companies 15
Leber Congenital Amaurosis (LCA) - Products under Investigation by Universities/Institutes 16
Leber Congenital Amaurosis (LCA) - Companies Involved in Therapeutics Development 17
AmpliPhi Biosciences Corporation 17
Applied Genetic Technologies Corporation 18
Editas Medicine, Inc. 19
ProQR Therapeutics N.V. 20
QLT Inc. 21
Spark Therapeutics, Inc. 22
Leber Congenital Amaurosis (LCA) - Therapeutics Assessment 23
Assessment by Monotherapy Products 23
Assessment by Target 24
Assessment by Mechanism of Action 26
Assessment by Route of Administration 28
Assessment by Molecule Type 30
Drug Profiles 32
A-001 - Drug Profile 32
Product Description 32
Mechanism Of Action 32
R&D Progress 32
Gene Therapy for Leber Congenital Amaurosis 10 - Drug Profile 34
Product Description 34
Mechanism Of Action 34
R&D Progress 34
Gene Therapy to Activate Retinal Guanylyl Cyclase-1 for Leber Congenital Amaurosis-1 - Drug Profile 35
Product Description 35
Mechanism Of Action 35
R&D Progress 35
Gene Therapy to Activate Retinal Pigment Epithelium 65 for Leber Congenital Amaurosis - Drug Profile 36
Product Description 36
Mechanism Of Action 36
R&D Progress 36
HORARPE-65 - Drug Profile 38
Product Description 38
Mechanism Of Action 38
R&D Progress 38
QR-110 - Drug Profile 39
Product Description 39
Mechanism Of Action 39
R&D Progress 39
voretigene neparvovec - Drug Profile 40
Product Description 40
Mechanism Of Action 40
R&D Progress 40
zuretinol acetate - Drug Profile 44
Product Description 44
Mechanism Of Action 44
R&D Progress 44
Leber Congenital Amaurosis (LCA) - Dormant Projects 49
Leber Congenital Amaurosis (LCA) - Product Development Milestones 50
Featured News & Press Releases 50
Jun 01, 2016: ProQR’s Drug Candidate QR-110 for Leber’s Congenital Amaurosis Type 10 Receives EMA and FDA Orphan Drug Designation 50
Apr 25, 2016: ProQR Announces an Investor and Analyst Event and Pre-Clinical Data Presentations for QR-110 for LCA10 at the 2016 ARVO Annual Meeting 50
May 04, 2015: Penn Study Indicates that Gene Therapy Efficacy for LCA is Dynamic: Improvement is Followed by Decline in Vision 50
May 01, 2015: Spark Therapeutics to Have Multiple Presentations at 2015 ARVO Meeting 52
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 52
Nov 06, 2014: Spark Therapeutics Receives FDA Breakthrough Therapy Designation for Its Lead Product Candidate, SPK-RPE65 54
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 55
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 56
May 08, 2014: Spark Therapeutics Scientific Advisor to Deliver Keynote at the Association for Research in Vision and Ophthalmology’s 2014 Annual Meeting 57
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 57
Jan 14, 2014: Spark Therapeutics Achieves Recruitment Goal in Phase 3 Gene Therapy Clinical Study for Inherited Blindness 58
Appendix 60
Methodology 60
Coverage 60
Secondary Research 60
Primary Research 60
Expert Panel Validation 60
Contact Us 60
Disclaimer 61

List of Figures
Number of Products under Development for Leber Congenital Amaurosis (LCA), H2 2016 8
Number of Products under Development for Leber Congenital Amaurosis (LCA) - Comparative Analysis, H2 2016 9
Number of Products under Development by Companies, H2 2016 10
Comparative Analysis by Late Stage Development, H2 2016 12
Comparative Analysis by Clinical Stage Development, H2 2016 13
Comparative Analysis by Early Stage Products, H2 2016 14
Assessment by Monotherapy Products, H2 2016 23
Number of Products by Targets, H2 2016 24
Number of Products by Stage and Targets, H2 2016 24
Number of Products by Mechanism of Actions, H2 2016 26
Number of Products by Stage and Mechanism of Actions, H2 2016 26
Number of Products by Routes of Administration, H2 2016 28
Number of Products by Stage and Routes of Administration, H2 2016 28
Number of Products by Molecule Types, H2 2016 30
Number of Products by Stage and Molecule Types, H2 2016 30

List of Tables
Number of Products under Development for Leber Congenital Amaurosis (LCA), H2 2016 8
Number of Products under Development for Leber Congenital Amaurosis (LCA) - Comparative Analysis, H2 2016 9
Number of Products under Development by Companies, H2 2016 10
Number of Products under Investigation by Universities/Institutes, H2 2016 11
Comparative Analysis by Late Stage Development, H2 2016 12
Comparative Analysis by Clinical Stage Development, H2 2016 13
Comparative Analysis by Early Stage Development, H2 2016 14
Products under Development by Companies, H2 2016 15
Products under Investigation by Universities/Institutes, H2 2016 16
Leber Congenital Amaurosis (LCA) - Pipeline by AmpliPhi Biosciences Corporation, H2 2016 17
Leber Congenital Amaurosis (LCA) - Pipeline by Applied Genetic Technologies Corporation, H2 2016 18
Leber Congenital Amaurosis (LCA) - Pipeline by Editas Medicine, Inc., H2 2016 19
Leber Congenital Amaurosis (LCA) - Pipeline by ProQR Therapeutics N.V., H2 2016 20
Leber Congenital Amaurosis (LCA) - Pipeline by QLT Inc., H2 2016 21
Leber Congenital Amaurosis (LCA) - Pipeline by Spark Therapeutics, Inc., H2 2016 22
Assessment by Monotherapy Products, H2 2016 23
Number of Products by Stage and Target, H2 2016 25
Number of Products by Stage and Mechanism of Action, H2 2016 27
Number of Products by Stage and Route of Administration, H2 2016 29
Number of Products by Stage and Molecule Type, H2 2016 31
Leber Congenital Amaurosis (LCA) - Dormant Projects, H2 2016 49
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