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 2017年下半期
Leber Congenital Amaurosis (LCA) - Pipeline Review, H2 2017

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 2017年下半期
出版社名 Global Markets Direct
発刊日 2017-09-19
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
List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Leber Congenital Amaurosis (LCA) - Overview
Leber Congenital Amaurosis (LCA) - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Leber Congenital Amaurosis (LCA) - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Leber Congenital Amaurosis (LCA) - Companies Involved in Therapeutics Development
AmpliPhi Biosciences Corp
Editas Medicine Inc
Novelion Therapeutics Inc
ProQR Therapeutics NV
Spark Therapeutics Inc
Leber Congenital Amaurosis (LCA) - Drug Profiles
A-001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy for Leber Congenital Amaurosis 10 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Retinal Guanylyl Cyclase-1 for Leber Congenital Amaurosis-1 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Retinoid Isomerohydrolase for Leber Congenital Amaurosis - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate RPE65 for Leber Congenital Amaurosis - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate RPE65 for Leber Congenital Amaurosis - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
HORARPE-65 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
QR-110 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
voretigene neparvovec - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
zuretinol acetate - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Leber Congenital Amaurosis (LCA) - Dormant Projects
Leber Congenital Amaurosis (LCA) - Product Development Milestones
Featured News & Press Releases
Jul 31, 2017: Spark Therapeutics Submits Marketing Authorization Application to European Medicines Agency for Investigational LUXTURNA (voretigene neparvovec)
May 31, 2017: ProQR Receives Fast Track Designation from the FDA for QR-110 for Leber's Congenital Amaurosis Type 10
May 01, 2017: ProQR to Present its Ophthalmology Pipeline at ARVO, Highlighting Programs in Leber’s Congenital Amaurosis Type 10 (LCA 10)
Apr 27, 2017: ProQR Announces Clearance of IND Application to Start Clinical Trial for QR-110 in Leber’s Congenital Amaurosis Type 10 Patients
Jan 09, 2017: Editas Medicine Reports on Recent Progress and 2017 Goals at J.P. Morgan Healthcare Conference
Jun 01, 2016: ProQR’s Drug Candidate QR-110 for Leber’s Congenital Amaurosis Type 10 Receives EMA and FDA Orphan Drug Designation
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
May 04, 2015: Penn Study Indicates that Gene Therapy Efficacy for LCA is Dynamic: Improvement is Followed by Decline in Vision
May 01, 2015: Spark Therapeutics to Have Multiple Presentations at 2015 ARVO Meeting
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
Nov 06, 2014: Spark Therapeutics Receives FDA Breakthrough Therapy Designation for Its Lead Product Candidate, SPK-RPE65
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
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
May 08, 2014: Spark Therapeutics Scientific Advisor to Deliver Keynote at the Association for Research in Vision and Ophthalmology’s 2014 Annual Meeting
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
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List of Figures
Number of Products under Development for Leber Congenital Amaurosis (LCA), H2 2017
Number of Products under Development by Companies, H2 2017
Number of Products under Development by Universities/Institutes, H2 2017
Number of Products by Targets, H2 2017
Number of Products by Stage and Targets, H2 2017
Number of Products by Mechanism of Actions, H2 2017
Number of Products by Stage and Mechanism of Actions, H2 2017
Number of Products by Routes of Administration, H2 2017
Number of Products by Stage and Routes of Administration, H2 2017
Number of Products by Molecule Types, H2 2017
Number of Products by Stage and Molecule Types, H2 2017

List of Tables
Number of Products under Development for Leber Congenital Amaurosis (LCA), H2 2017
Number of Products under Development by Companies, H2 2017
Number of Products under Development by Universities/Institutes, H2 2017
Products under Development by Companies, H2 2017
Products under Development by Universities/Institutes, H2 2017
Number of Products by Stage and Target, H2 2017
Number of Products by Stage and Mechanism of Action, H2 2017
Number of Products by Stage and Route of Administration, H2 2017
Number of Products by Stage and Molecule Type, H2 2017
Leber Congenital Amaurosis (LCA) - Pipeline by AmpliPhi Biosciences Corp, H2 2017
Leber Congenital Amaurosis (LCA) - Pipeline by Editas Medicine Inc, H2 2017
Leber Congenital Amaurosis (LCA) - Pipeline by Novelion Therapeutics Inc, H2 2017
Leber Congenital Amaurosis (LCA) - Pipeline by ProQR Therapeutics NV, H2 2017
Leber Congenital Amaurosis (LCA) - Pipeline by Spark Therapeutics Inc, H2 2017
Leber Congenital Amaurosis (LCA) - Dormant Projects, H2 2017
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