Leber Congenital Amaurosis (LCA) - Pipeline Review, H2 2014

Published: Dec, 2014 | Pages: 52 | Publisher: Global Markets Direct
Industry: Healthcare | Report Format: Electronic (PDF)

Leber Congenital Amaurosis (LCA) - Pipeline Review, H2 2014

Summary

Global Markets Direct's, 'Leber Congenital Amaurosis (LCA) - Pipeline Review, H2 2014', 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

 Table of Contents
Table of Contents 2
List of Tables 4
List of Figures 4
Introduction 5
Global Markets Direct Report Coverage 5
Leber Congenital Amaurosis (LCA) Overview 6
Therapeutics Development 7
Pipeline Products for Leber Congenital Amaurosis (LCA) - Overview 7
Pipeline Products for Leber Congenital Amaurosis (LCA) - Comparative Analysis 8
Leber Congenital Amaurosis (LCA) - Therapeutics under Development by Companies 9
Leber Congenital Amaurosis (LCA) - Therapeutics under Investigation by Universities/Institutes 10
Leber Congenital Amaurosis (LCA) - Pipeline Products Glance 11
Late Stage Products 11
Clinical Stage Products 12
Early Stage Products 13
Leber Congenital Amaurosis (LCA) - Products under Development by Companies 14
Leber Congenital Amaurosis (LCA) - Products under Investigation by Universities/Institutes 15
Leber Congenital Amaurosis (LCA) - Companies Involved in Therapeutics Development 16
AmpliPhi Biosciences Corporation 16
Applied Genetic Technologies Corporation 17
BBB Therapeutics B.V. 18
ProQR Therapeutics N.V. 19
QLT Inc. 20
ReGenX Biosciences, LLC 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 25
Assessment by Route of Administration 26
Assessment by Molecule Type 28
Drug Profiles 30
Gene Therapy to Activate Retinal Guanylyl Cyclase-1 for Leber Congenital Amaurosis-1 - Drug Profile 30
Product Description 30
Mechanism of Action 30
R&D Progress 30
Gene Therapy to Activate RPE65 for Leber Congenital Amaurosis (LCA) - Drug Profile 31
Product Description 31
Mechanism of Action 31
R&D Progress 31
QLT-091001 - Drug Profile 32
Product Description 32
Mechanism of Action 32
R&D Progress 32
QR-110 - Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
rAAV2-CB-hRPE65 - Drug Profile 35
Product Description 35
Mechanism of Action 35
R&D Progress 35
Small Molecules for Inherited Retinal Diseases - Drug Profile 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
SPK-RPE-65 - Drug Profile 37
Product Description 37
Mechanism of Action 37
R&D Progress 37
tgAAG-76 - Drug Profile 38
Product Description 38
Mechanism of Action 38
R&D Progress 38
Leber Congenital Amaurosis (LCA) - Recent Pipeline Updates 39
Leber Congenital Amaurosis (LCA) - Dormant Projects 43
Leber Congenital Amaurosis (LCA) - Product Development Milestones 44
Featured News & Press Releases 44
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 44
Nov 06, 2014: Spark Therapeutics Receives FDA Breakthrough Therapy Designation for Its Lead Product Candidate, SPK-RPE65 45
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 46
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 47
May 08, 2014: Spark Therapeutics Scientific Advisor to Deliver Keynote at the Association for Research in Vision and Ophthalmology's 2014 Annual Meeting 48
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 48
Jan 14, 2014: Spark Therapeutics Achieves Recruitment Goal in Phase 3 Gene Therapy Clinical Study for Inherited Blindness 49
Appendix 51
Methodology 51
Coverage 51
Secondary Research 51
Primary Research 51
Expert Panel Validation 51
Contact Us 52
Disclaimer 52

List of Tables
Number of Products under Development for Leber Congenital Amaurosis (LCA), H2 2014 7
Number of Products under Development for Leber Congenital Amaurosis (LCA) - Comparative Analysis, H2 2014 8
Number of Products under Development by Companies, H2 2014 9
Number of Products under Investigation by Universities/Institutes, H2 2014 10
Comparative Analysis by Late Stage Development, H2 2014 11
Comparative Analysis by Clinical Stage Development, H2 2014 12
Comparative Analysis by Early Stage Development, H2 2014 13
Products under Development by Companies, H2 2014 14
Products under Investigation by Universities/Institutes, H2 2014 15
Leber Congenital Amaurosis (LCA) - Pipeline by AmpliPhi Biosciences Corporation, H2 2014 16
Leber Congenital Amaurosis (LCA) - Pipeline by Applied Genetic Technologies Corporation, H2 2014 17
Leber Congenital Amaurosis (LCA) - Pipeline by BBB Therapeutics B.V., H2 2014 18
Leber Congenital Amaurosis (LCA) - Pipeline by ProQR Therapeutics N.V., H2 2014 19
Leber Congenital Amaurosis (LCA) - Pipeline by QLT Inc., H2 2014 20
Leber Congenital Amaurosis (LCA) - Pipeline by ReGenX Biosciences, LLC, H2 2014 21
Leber Congenital Amaurosis (LCA) - Pipeline by Spark Therapeutics, Inc., H2 2014 22
Assessment by Monotherapy Products, H2 2014 23
Number of Products by Stage and Target, H2 2014 24
Number of Products by Stage and Mechanism of Action, H2 2014 25
Number of Products by Stage and Route of Administration, H2 2014 27
Number of Products by Stage and Molecule Type, H2 2014 29
Leber Congenital Amaurosis (LCA) Therapeutics - Recent Pipeline Updates, H2 2014 39
Leber Congenital Amaurosis (LCA) - Dormant Projects, H2 2014 43