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

Published: Sep, 2016 | Pages: 61 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

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 2
List of Tables 4
List of Figures 5
Introduction 6
Global Markets Direct Report Coverage 6
Leber Congenital Amaurosis (LCA) Overview 7
Therapeutics Development 8
Pipeline Products for Leber Congenital Amaurosis (LCA) - Overview 8
Pipeline Products for Leber Congenital Amaurosis (LCA) - Comparative Analysis 9
Leber Congenital Amaurosis (LCA) - Therapeutics under Development by Companies 10
Leber Congenital Amaurosis (LCA) - Therapeutics under Investigation by Universities/Institutes 11
Leber Congenital Amaurosis (LCA) - Pipeline Products Glance 12
Late Stage Products 12
Clinical Stage Products 13
Early Stage Products 14
Leber Congenital Amaurosis (LCA) - Products under Development by Companies 15
Leber Congenital Amaurosis (LCA) - Products under Investigation by Universities/Institutes 16
Leber Congenital Amaurosis (LCA) - Companies Involved in Therapeutics Development 17
AmpliPhi Biosciences Corporation 17
Applied Genetic Technologies Corporation 18
Editas Medicine, Inc. 19
ProQR Therapeutics N.V. 20
QLT Inc. 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 26
Assessment by Route of Administration 28
Assessment by Molecule Type 30
Drug Profiles 32
A-001 - Drug Profile 32
Product Description 32
Mechanism Of Action 32
R&D Progress 32
Gene Therapy for Leber Congenital Amaurosis 10 - Drug Profile 34
Product Description 34
Mechanism Of Action 34
R&D Progress 34
Gene Therapy to Activate Retinal Guanylyl Cyclase-1 for Leber Congenital Amaurosis-1 - Drug Profile 35
Product Description 35
Mechanism Of Action 35
R&D Progress 35
Gene Therapy to Activate Retinal Pigment Epithelium 65 for Leber Congenital Amaurosis - Drug Profile 36
Product Description 36
Mechanism Of Action 36
R&D Progress 36
HORARPE-65 - Drug Profile 38
Product Description 38
Mechanism Of Action 38
R&D Progress 38
QR-110 - Drug Profile 39
Product Description 39
Mechanism Of Action 39
R&D Progress 39
voretigene neparvovec - Drug Profile 40
Product Description 40
Mechanism Of Action 40
R&D Progress 40
zuretinol acetate - Drug Profile 44
Product Description 44
Mechanism Of Action 44
R&D Progress 44
Leber Congenital Amaurosis (LCA) - Dormant Projects 49
Leber Congenital Amaurosis (LCA) - Product Development Milestones 50
Featured News & Press Releases 50
Jun 01, 2016: ProQR's Drug Candidate QR-110 for Leber's Congenital Amaurosis Type 10 Receives EMA and FDA Orphan Drug Designation 50
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 50
May 04, 2015: Penn Study Indicates that Gene Therapy Efficacy for LCA is Dynamic: Improvement is Followed by Decline in Vision 50
May 01, 2015: Spark Therapeutics to Have Multiple Presentations at 2015 ARVO Meeting 52
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 52
Nov 06, 2014: Spark Therapeutics Receives FDA Breakthrough Therapy Designation for Its Lead Product Candidate, SPK-RPE65 54
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 55
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 56
May 08, 2014: Spark Therapeutics Scientific Advisor to Deliver Keynote at the Association for Research in Vision and Ophthalmology's 2014 Annual Meeting 57
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 57
Jan 14, 2014: Spark Therapeutics Achieves Recruitment Goal in Phase 3 Gene Therapy Clinical Study for Inherited Blindness 58
Appendix 60
Methodology 60
Coverage 60
Secondary Research 60
Primary Research 60
Expert Panel Validation 60
Contact Us 60
Disclaimer 61

List of Tables

Number of Products under Development for Leber Congenital Amaurosis (LCA), H2 2016 8
Number of Products under Development for Leber Congenital Amaurosis (LCA) - Comparative Analysis, H2 2016 9
Number of Products under Development by Companies, H2 2016 10
Number of Products under Investigation by Universities/Institutes, H2 2016 11
Comparative Analysis by Late Stage Development, H2 2016 12
Comparative Analysis by Clinical Stage Development, H2 2016 13
Comparative Analysis by Early Stage Development, H2 2016 14
Products under Development by Companies, H2 2016 15
Products under Investigation by Universities/Institutes, H2 2016 16
Leber Congenital Amaurosis (LCA) - Pipeline by AmpliPhi Biosciences Corporation, H2 2016 17
Leber Congenital Amaurosis (LCA) - Pipeline by Applied Genetic Technologies Corporation, H2 2016 18
Leber Congenital Amaurosis (LCA) - Pipeline by Editas Medicine, Inc., H2 2016 19
Leber Congenital Amaurosis (LCA) - Pipeline by ProQR Therapeutics N.V., H2 2016 20
Leber Congenital Amaurosis (LCA) - Pipeline by QLT Inc., H2 2016 21
Leber Congenital Amaurosis (LCA) - Pipeline by Spark Therapeutics, Inc., H2 2016 22
Assessment by Monotherapy Products, H2 2016 23
Number of Products by Stage and Target, H2 2016 25
Number of Products by Stage and Mechanism of Action, H2 2016 27
Number of Products by Stage and Route of Administration, H2 2016 29
Number of Products by Stage and Molecule Type, H2 2016 31
Leber Congenital Amaurosis (LCA) - Dormant Projects, H2 2016 49