Leber Congenital Amaurosis (LCA) - Pipeline Review, H2 2016 - Global Markets Direct - Market Research

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

Leber Congenital Amaurosis (LCA) - Pipeline Review, H2 2016 - Global Markets Direct - Market Research
Leber Congenital Amaurosis (LCA) - Pipeline Review, H2 2016
Published Sep 30, 2016
61 pages — Published Sep 30, 2016
Price US$ 2,000.00  |  Buy this Report Now

About This Report

  
Abstract:

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 Directs 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.

  
Source:
Document ID
GMDHC8521IDB
Industry
Format:
PDF Adobe Acrobat
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Sections

TitleStarting PageNumber of Pages
Table of Contents24
  List of Tables41
  List of Figures51
Introduction61
  Global Markets Direct Report Coverage61
Leber Congenital Amaurosis (LCA) Overview71
Therapeutics Development82
  Pipeline Products for Leber Congenital Amaurosis (LCA) Overview81
  Pipeline Products for Leber Congenital Amaurosis (LCA) Comparative Analysis91
Leber Congenital Amaurosis (LCA) Therapeutics under Development by Companies101
Leber Congenital Amaurosis (LCA) Therapeutics under Investigation by Universities/Institutes111
Leber Congenital Amaurosis (LCA) Pipeline Products Glance123
  Late Stage Products121
  Clinical Stage Products131
  Early Stage Products141
Leber Congenital Amaurosis (LCA) Products under Development by Companies151
Leber Congenital Amaurosis (LCA) Products under Investigation by Universities/Institutes161
Leber Congenital Amaurosis (LCA) Companies Involved in Therapeutics Development176
  AmpliPhi Biosciences Corporation171
  Applied Genetic Technologies Corporation181
  Editas Medicine, Inc.191
  ProQR Therapeutics N.V.201
  QLT Inc.211
  Spark Therapeutics, Inc.221
Leber Congenital Amaurosis (LCA) Therapeutics Assessment239
  Assessment by Monotherapy Products231
  Assessment by Target242
  Assessment by Mechanism of Action262
  Assessment by Route of Administration282
  Assessment by Molecule Type302
Drug Profiles3217
  A-001 Drug Profile322
  Gene Therapy for Leber Congenital Amaurosis 10 Drug Profile341
  Gene Therapy to Activate Retinal Guanylyl Cyclase-1 for Leber Congenital Amaurosis-1 Drug Profile351
  Gene Therapy to Activate Retinal Pigment Epithelium 65 for Leber Congenital Amaurosis Drug Profile362
  HORARPE-65 Drug Profile381
  QR-110 Drug Profile391
  voretigene neparvovec Drug Profile404
  zuretinol acetate Drug Profile445
Leber Congenital Amaurosis (LCA) Dormant Projects491
Leber Congenital Amaurosis (LCA) Product Development Milestones5010
  Featured News &Press Releases501
    Jun 01, 2016: ProQR s Drug Candidate QR-110 for Leber s Congenital Amaurosis Type 10 Receives EMA and FDA Orphan Drug Designation501
    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 Meeting501
    May 04, 2015: Penn Study Indicates that Gene Therapy Efficacy for LCA is Dynamic: Improvement is Followed by Decline in Vision502
    May 01, 2015: Spark Therapeutics to Have Multiple Presentations at 2015 ARVO Meeting521
    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 Vision522
    Nov 06, 2014: Spark Therapeutics Receives FDA Breakthrough Therapy Designation for Its Lead Product Candidate, SPK-RPE65541
    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 LRAT551
    Jul 14, 2014: QLT Announces Publication in The Lancet of Phase 1b Data for QLT091001 in Leber Congenital Amaurosis Due to LRAT or RPE65 Mutations561
    May 08, 2014: Spark Therapeutics Scientific Advisor to Deliver Keynote at the Association for Research in Vision and Ophthalmology s 2014 Annual Meeting571
    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 RPE65571
    Jan 14, 2014: Spark Therapeutics Achieves Recruitment Goal in Phase 3 Gene Therapy Clinical Study for Inherited Blindness582
Appendix602
  Methodology601
  Coverage601
  Secondary Research601
  Primary Research601
  Expert Panel Validation601
  Contact Us601
  Disclaimer611

Table Of Contents

Global Markets Direct - Market Research—Global Markets Direct is a leading provider of global business intelligence and market analysis. It publishes a range of high quality market reports created by its large research and analysis capability and drawing from its extensive unique databases of industry-specific information.

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Cite this Report

  
MLA:
Global Markets Direct - Market Research. "Leber Congenital Amaurosis (LCA) - Pipeline Review, H2 2016" Sep 30, 2016. Alacra Store. May 05, 2025. <http://www.alacrastore.com/storecontent/Global-Markets-Direct-Market-Research/Leber-Congenital-Amaurosis-LCA-Pipeline-Review-H2-2016-2088-16648>
  
APA:
Global Markets Direct - Market Research. (2016). Leber Congenital Amaurosis (LCA) - Pipeline Review, H2 2016 Sep 30, 2016. New York, NY: Alacra Store. Retrieved May 05, 2025 from <http://www.alacrastore.com/storecontent/Global-Markets-Direct-Market-Research/Leber-Congenital-Amaurosis-LCA-Pipeline-Review-H2-2016-2088-16648>
  
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