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Leber Congenital Amaurosis (LCA) - Pipeline Review, H2 2015

Leber Congenital Amaurosis (LCA) - Pipeline Review, H2 2015

Summary

Global Markets Direct’s, ‘Leber Congenital Amaurosis (LCA) - Pipeline Review, H2 2015’, provides an overview of the Leber Congenital Amaurosis (LCA)’s therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Leber Congenital Amaurosis (LCA), complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Leber Congenital Amaurosis (LCA) and special features on late-stage and discontinued projects.

Global Markets Direct’s report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Direct’s proprietary databases, Company/University websites, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct’s team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis.

The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease.

Scope

  • The report provides a snapshot of the global therapeutic landscape of Leber Congenital Amaurosis (LCA)
  • The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities
  • The report reviews key players involved in the therapeutics development for Leber Congenital Amaurosis (LCA) and enlists all their major and minor projects
  • The report summarizes all the dormant and discontinued pipeline projects
  • A review of the Leber Congenital Amaurosis (LCA) products under development by companies and universities/research institutes based on information derived from company and industry-specific sources
  • Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages
  • A detailed assessment of monotherapy and combination therapy pipeline projects
  • Coverage of the Leber Congenital Amaurosis (LCA) pipeline on the basis of target, MoA, route of administration and molecule type
  • Latest news and deals relating related to pipeline products
Reasons to buy
  • Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies
  • Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
  • Develop strategic initiatives by understanding the focus areas of leading companies
  • Identify and understand important and diverse types of therapeutics under development for Leber Congenital Amaurosis (LCA)
  • Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
  • Devise corrective measures for pipeline projects by understanding Leber Congenital Amaurosis (LCA) 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
  • Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline


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
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
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 RPE65 for Leber Congenital Amaurosis - Drug Profile
Product Description
Mechanism of Action
R&D Progress
QR-110 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
SPKRPE-65 - Drug Profile
Product Description
Mechanism of Action
R&D Progress
tgAAG-76 - 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) - Recent Pipeline Updates
Leber Congenital Amaurosis (LCA) - Dormant Projects
Leber Congenital Amaurosis (LCA) - Discontinued Products
Leber Congenital Amaurosis (LCA) - Product Development Milestones
Featured News & Press Releases
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 2015
Number of Products under Development for Leber Congenital Amaurosis (LCA) - Comparative Analysis, H2 2015
Number of Products under Development by Companies, H2 2015
Number of Products under Investigation by Universities/Institutes, H2 2015
Comparative Analysis by Late Stage Development, H2 2015
Comparative Analysis by Clinical Stage Development, H2 2015
Comparative Analysis by Early Stage Development, H2 2015
Products under Development by Companies, H2 2015
Products under Investigation by Universities/Institutes, H2 2015
Leber Congenital Amaurosis (LCA) - Pipeline by AmpliPhi Biosciences Corporation, H2 2015
Leber Congenital Amaurosis (LCA) - Pipeline by ProQR Therapeutics N.V., H2 2015
Leber Congenital Amaurosis (LCA) - Pipeline by QLT Inc., H2 2015
Leber Congenital Amaurosis (LCA) - Pipeline by Spark Therapeutics, Inc., H2 2015
Assessment by Monotherapy Products, H2 2015
Number of Products by Stage and Target, H2 2015
Number of Products by Stage and Mechanism of Action, H2 2015
Number of Products by Stage and Route of Administration, H2 2015
Number of Products by Stage and Molecule Type, H2 2015
Leber Congenital Amaurosis (LCA) Therapeutics - Recent Pipeline Updates, H2 2015
Leber Congenital Amaurosis (LCA) - Dormant Projects, H2 2015
Leber Congenital Amaurosis (LCA) - Discontinued Products, H2 2015
List of Figures
Number of Products under Development for Leber Congenital Amaurosis (LCA), H2 2015
Number of Products under Development for Leber Congenital Amaurosis (LCA) - Comparative Analysis, H2 2015
Number of Products under Development by Companies, H2 2015
Number of Products under Investigation by Universities/Institutes, H2 2015
Comparative Analysis by Clinical Stage Development, H2 2015
Comparative Analysis by Early Stage Products, H2 2015
Assessment by Monotherapy Products, H2 2015
Number of Products by Targets, H2 2015
Number of Products by Stage and Targets, H1 2015
Number of Products by Mechanism of Actions, H2 2015
Number of Products by Stage and Mechanism of Actions, H2 2015
Number of Products by Routes of Administration, H2 2015
Number of Products by Stage and Routes of Administration, H2 2015
Number of Products by Molecule Types, H2 2015
Number of Products by Stage and Molecule Types, H2 2015

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