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市場調查報告書

萊伯氏先天性黑蒙症(LCA):開發中產品分析

Leber Congenital Amaurosis (LCA) - Pipeline Review, H2 2016

出版商 Global Markets Direct 商品編碼 293890
出版日期 內容資訊 英文 61 Pages
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萊伯氏先天性黑蒙症(LCA):開發中產品分析 Leber Congenital Amaurosis (LCA) - Pipeline Review, H2 2016
出版日期: 2016年09月30日 內容資訊: 英文 61 Pages
簡介

萊伯氏先天性黑蒙症(LCA)是主要侵襲網膜的眼睛疾病。網膜位於眼球最內側,是感受光與顏色的特殊組織。這是造成兒童期失明最常見的原因。症狀包含失明,眼震,眼球凹陷,發育遲緩,癲癇,運動技能障礙等。

本報告提供萊伯氏先天性黑蒙症(LCA)的治療藥開發情形調查分析,提供您開發中產品概要,臨床實驗的各階段產品概要,主要企業簡介,藥物簡介,開發中產品的最新趨勢,最新消息和新聞稿等相關的系統性資訊。

目錄

簡介

萊伯氏先天性黑蒙症(LCA)概要

治療藥的開發

  • 開發中產品:概要
  • 開發中產品:比較分析

開發中的治療藥:各企業

調查中的治療藥:大學/研究機關別

開發中產品的概要

  • 後期階段的產品
  • 臨床實驗階段的產品
  • 初期階段的產品

開發中的產品:各企業

調查中的產品:大學/研究機關別

開發治療藥的企業

  • QLT Inc.
  • AmpliPhi Biosciences Corporation
  • ReGenX Biosciences, LLC
  • Applied Genetic Technologies Corporation
  • to-BBB technologies BV
  • Spark Therapeutics, Inc.

治療藥的評估

  • 單獨療法的情況
  • 標的別
  • 各作用機制
  • 各給藥途徑
  • 各分子類型

藥物簡介

  • Gene Therapy to Activate Retinal Guanylyl Cyclase-1 for Leber Congenital Amaurosis-1
  • QLT-091001
  • QR-110
  • rAAV2-CB-hRPE65
  • Small Molecules for Inherited Retinal Diseases
  • SPK-RPE-65
  • tgAAG-76

開發中產品的最新趨勢

暫停的計劃

產品開發的里程碑

  • 最新消息和新聞稿

附錄

圖表

本網頁內容可能與最新版本有所差異。詳細情況請與我們聯繫。

目錄
Product Code: GMDHC8521IDB

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Leber Congenital Amaurosis (LCA) - Pipeline Review, H2 2016, provides an overview of the Leber Congenital Amaurosis (LCA) (Genetic Disorders) pipeline landscape.

Leber congenital amaurosis is an eye disorder that primarily affects the retina, which is the specialized tissue at the back of the eye that detects light and color. This condition is the most common cause of inherited blindness in childhood. Symptoms include vision loss, roving eye movements, deep-set eyes, developmental delay, epilepsy, and motor skill impairment.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Leber Congenital Amaurosis (LCA) - Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Leber Congenital Amaurosis (LCA) (Genetic 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 Leber Congenital Amaurosis (LCA) (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Leber Congenital Amaurosis (LCA) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Phase II, Phase I, Preclinical and Discovery stages are 1, 3, 1, 1 and 1 respectively for Similarly, the Universities portfolio in Preclinical stages comprises 1 molecules, respectively for Leber Congenital Amaurosis (LCA).

Leber Congenital Amaurosis (LCA) (Genetic 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 Leber Congenital Amaurosis (LCA) (Genetic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Leber Congenital Amaurosis (LCA) (Genetic 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 Leber Congenital Amaurosis (LCA) (Genetic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Leber Congenital Amaurosis (LCA) (Genetic 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 Leber Congenital Amaurosis (LCA) (Genetic 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 Leber Congenital Amaurosis (LCA) (Genetic 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 Leber Congenital Amaurosis (LCA) (Genetic 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
  • Leber Congenital Amaurosis (LCA) Overview
  • Therapeutics Development
    • Pipeline Products for Leber Congenital Amaurosis (LCA) - Overview
    • Pipeline Products for Leber Congenital Amaurosis (LCA) - Comparative Analysis
  • Leber Congenital Amaurosis (LCA) - Therapeutics under Development by Companies
  • Leber Congenital Amaurosis (LCA) - Therapeutics under Investigation by Universities/Institutes
  • Leber Congenital Amaurosis (LCA) - Pipeline Products Glance
    • Late Stage Products
    • Clinical Stage Products
    • Early Stage Products
  • Leber Congenital Amaurosis (LCA) - Products under Development by Companies
  • Leber Congenital Amaurosis (LCA) - Products under Investigation by Universities/Institutes
  • Leber Congenital Amaurosis (LCA) - Companies Involved in Therapeutics Development
    • AmpliPhi Biosciences Corporation
    • Applied Genetic Technologies Corporation
    • Editas Medicine, Inc.
    • ProQR Therapeutics N.V.
    • QLT Inc.
    • Spark Therapeutics, Inc.
  • Leber Congenital Amaurosis (LCA) - Therapeutics Assessment
    • Assessment by Monotherapy Products
    • Assessment by Target
    • Assessment by Mechanism of Action
    • Assessment by Route of Administration
    • Assessment by Molecule Type
  • 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 Retinal Pigment Epithelium 65 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
      • 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
      • Jan 14, 2014: Spark Therapeutics Achieves Recruitment Goal in Phase 3 Gene Therapy Clinical Study for Inherited Blindness
  • Appendix
    • Methodology
    • Coverage
    • Secondary Research
    • Primary Research
    • Expert Panel Validation
    • Contact Us
    • Disclaimer

List of Tables

  • Number of Products under Development for Leber Congenital Amaurosis (LCA), H2 2016
  • Number of Products under Development for Leber Congenital Amaurosis (LCA) - Comparative Analysis, H2 2016
  • Number of Products under Development by Companies, H2 2016
  • Number of Products under Investigation by Universities/Institutes, H2 2016
  • Comparative Analysis by Late Stage Development, H2 2016
  • Comparative Analysis by Clinical Stage Development, H2 2016
  • Comparative Analysis by Early Stage Development, H2 2016
  • Products under Development by Companies, H2 2016
  • Products under Investigation by Universities/Institutes, H2 2016
  • Leber Congenital Amaurosis (LCA) - Pipeline by AmpliPhi Biosciences Corporation, H2 2016
  • Leber Congenital Amaurosis (LCA) - Pipeline by Applied Genetic Technologies Corporation, H2 2016
  • Leber Congenital Amaurosis (LCA) - Pipeline by Editas Medicine, Inc., H2 2016
  • Leber Congenital Amaurosis (LCA) - Pipeline by ProQR Therapeutics N.V., H2 2016
  • Leber Congenital Amaurosis (LCA) - Pipeline by QLT Inc., H2 2016
  • Leber Congenital Amaurosis (LCA) - Pipeline by Spark Therapeutics, Inc., H2 2016
  • Assessment by Monotherapy Products, H2 2016
  • Number of Products by Stage and Target, H2 2016
  • Number of Products by Stage and Mechanism of Action, H2 2016
  • Number of Products by Stage and Route of Administration, H2 2016
  • Number of Products by Stage and Molecule Type, H2 2016
  • Leber Congenital Amaurosis (LCA) - Dormant Projects, H2 2016

List of Figures

  • Number of Products under Development for Leber Congenital Amaurosis (LCA), H2 2016
  • Number of Products under Development for Leber Congenital Amaurosis (LCA) - Comparative Analysis, H2 2016
  • Number of Products under Development by Companies, H2 2016
  • Comparative Analysis by Late Stage Development, H2 2016
  • Comparative Analysis by Clinical Stage Development, H2 2016
  • Comparative Analysis by Early Stage Products, H2 2016
  • Assessment by Monotherapy Products, H2 2016
  • Number of Products by Targets, H2 2016
  • Number of Products by Stage and Targets, H2 2016
  • Number of Products by Mechanism of Actions, H2 2016
  • Number of Products by Stage and Mechanism of Actions, H2 2016
  • Number of Products by Routes of Administration, H2 2016
  • Number of Products by Stage and Routes of Administration, H2 2016
  • Number of Products by Molecule Types, H2 2016
  • Number of Products by Stage and Molecule Types, H2 2016
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