Leber Congenital Amaurosis (LCA) - Pipeline Review, H1 2016

Published: Apr, 2016 | Pages: 57 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Leber Congenital Amaurosis (LCA) - Pipeline Review, H1 2016

Summary

Global Markets Direct's, 'Leber Congenital Amaurosis (LCA) - Pipeline Review, H1 2016', provides an overview of the Leber Congenital Amaurosis (LCA) pipeline landscape. 

The report provides comprehensive information on the therapeutics under development for Leber Congenital Amaurosis (LCA), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Leber Congenital Amaurosis (LCA) and features dormant 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, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report 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.

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

Scope

- The report provides a snapshot of the global therapeutic landscape of Leber Congenital Amaurosis (LCA)
- The report reviews pipeline therapeutics for Leber Congenital Amaurosis (LCA) by companies and universities/research institutes based on information derived from company and industry-specific sources 
- The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages 
- The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities 
- The report reviews key players involved Leber Congenital Amaurosis (LCA) therapeutics and enlists all their major and minor projects
- The report assesses Leber Congenital Amaurosis (LCA) therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type 
- The report summarizes all the dormant and discontinued pipeline projects 
- The report reviews latest news related to pipeline therapeutics for Leber Congenital Amaurosis (LCA)

Reasons To Buy

- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage 
- Identify and understand important and diverse types of therapeutics under development for Leber Congenital Amaurosis (LCA)
- Identify potential new clients or partners in the target demographic
- Develop strategic initiatives by understanding the focus areas of leading companies 
- Plan mergers and acquisitions effectively by identifying key players and it's most promising pipeline therapeutics
- 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 Content 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
Editas Medicine, Inc. 17
ProQR Therapeutics N.V. 18
QLT Inc. 19
Spark Therapeutics, Inc. 20
Leber Congenital Amaurosis (LCA) - Therapeutics Assessment 21
Assessment by Monotherapy Products 21
Assessment by Target 22
Assessment by Mechanism of Action 24
Assessment by Route of Administration 26
Assessment by Molecule Type 28
Drug Profiles 30
Gene Therapy for Leber Congenital Amaurosis - Drug Profile 30
Product Description 30
Mechanism of Action 30
R&D Progress 30
Gene Therapy to Activate Retinal Guanylyl Cyclase-1 for Leber Congenital Amaurosis-1 - Drug Profile 31
Product Description 31
Mechanism of Action 31
R&D Progress 31
Gene Therapy to Activate RPE65 for Leber Congenital Amaurosis - Drug Profile 32
Product Description 32
Mechanism of Action 32
R&D Progress 32
QR-110 - Drug Profile 33
Product Description 33
Mechanism of Action 33
R&D Progress 33
tgAAG-76 - Drug Profile 34
Product Description 34
Mechanism of Action 34
R&D Progress 34
voretigene neparvovec - Drug Profile 36
Product Description 36
Mechanism of Action 36
R&D Progress 36
zuretinol acetate - Drug Profile 39
Product Description 39
Mechanism of Action 39
R&D Progress 39
Leber Congenital Amaurosis (LCA) - Recent Pipeline Updates 41
Leber Congenital Amaurosis (LCA) - Dormant Projects 45
Leber Congenital Amaurosis (LCA) - Discontinued Products 46
Leber Congenital Amaurosis (LCA) - Product Development Milestones 47
Featured News & Press Releases 47
May 04, 2015: Penn Study Indicates that Gene Therapy Efficacy for LCA is Dynamic: Improvement is Followed by Decline in Vision 47
May 01, 2015: Spark Therapeutics to Have Multiple Presentations at 2015 ARVO Meeting 48
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 49
Nov 06, 2014: Spark Therapeutics Receives FDA Breakthrough Therapy Designation for Its Lead Product Candidate, SPK-RPE65 50
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 51
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 52
May 08, 2014: Spark Therapeutics Scientific Advisor to Deliver Keynote at the Association for Research in Vision and Ophthalmology's 2014 Annual Meeting 53
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 53
Jan 14, 2014: Spark Therapeutics Achieves Recruitment Goal in Phase 3 Gene Therapy Clinical Study for Inherited Blindness 54
Appendix 56
Methodology 56
Coverage 56
Secondary Research 56
Primary Research 56
Expert Panel Validation 56
Contact Us 56
Disclaimer 57

List of Tables

Number of Products under Development for Leber Congenital Amaurosis (LCA), H1 2016 7
Number of Products under Development for Leber Congenital Amaurosis (LCA) - Comparative Analysis, H1 2016 8
Number of Products under Development by Companies, H1 2016 9
Number of Products under Investigation by Universities/Institutes, H1 2016 10
Comparative Analysis by Late Stage Development, H1 2016 11
Comparative Analysis by Clinical Stage Development, H1 2016 12
Comparative Analysis by Early Stage Development, H1 2016 13
Products under Development by Companies, H1 2016 14
Products under Investigation by Universities/Institutes, H1 2016 15
Leber Congenital Amaurosis (LCA) - Pipeline by AmpliPhi Biosciences Corporation, H1 2016 16
Leber Congenital Amaurosis (LCA) - Pipeline by Editas Medicine, Inc., H1 2016 17
Leber Congenital Amaurosis (LCA) - Pipeline by ProQR Therapeutics N.V., H1 2016 18
Leber Congenital Amaurosis (LCA) - Pipeline by QLT Inc., H1 2016 19
Leber Congenital Amaurosis (LCA) - Pipeline by Spark Therapeutics, Inc., H1 2016 20
Assessment by Monotherapy Products, H1 2016 21
Number of Products by Stage and Target, H1 2016 23
Number of Products by Stage and Mechanism of Action, H1 2016 25
Number of Products by Stage and Route of Administration, H1 2016 27
Number of Products by Stage and Molecule Type, H1 2016 29
Leber Congenital Amaurosis (LCA) Therapeutics - Recent Pipeline Updates, H1 2016 41
Leber Congenital Amaurosis (LCA) - Dormant Projects, H1 2016 45
Leber Congenital Amaurosis (LCA) - Discontinued Products, H1 2016 46