Summary
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Leber Congenital Amaurosis (LCA) - Pipeline Review, H1 2018, 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, H1 2018, 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 III, Phase II and Preclinical stages are 1, 1, 2 and 2 respectively. Similarly, the Universities portfolio in Phase I and Preclinical stages comprises 1 and 3 molecules, respectively.
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.
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Table of Contents
List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Leber Congenital Amaurosis (LCA) - Overview
Leber Congenital Amaurosis (LCA) - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Leber Congenital Amaurosis (LCA) - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Leber Congenital Amaurosis (LCA) - Companies Involved in Therapeutics Development
Editas Medicine Inc
Novelion Therapeutics Inc
ProQR Therapeutics NV
Spark Therapeutics Inc
Leber Congenital Amaurosis (LCA) - Drug Profiles
A-001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
EDIT-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate AIPL1 for Leber’s Congenital Amaurosis - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate AIPL1 for Ophthalmology - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
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
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
voretigene neparvovec - 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) - Dormant Projects
Leber Congenital Amaurosis (LCA) - Product Development Milestones
Featured News & Press Releases
Jan 03, 2018: Spark's Gene Therapy Treatment for Blindness LUXTURNA to Cost $850,000
Oct 19, 2017: Editas Medicine Demonstrates Dose-Dependent, In Vivo Editing with EDIT-101 in CEP290 Transgenic Mice
Sep 26, 2017: Editas Medicine Receives EMA's Orphan Medicinal Product Designation for EDIT-101 for the Treatment of LCA10
Sep 12, 2017: Editas Medicine Initiates Clinical Natural History Study to Evaluate Patients with Leber Congenital Amaurosis Type 10
Jul 31, 2017: Spark Therapeutics Submits Marketing Authorization Application to European Medicines Agency for Investigational LUXTURNA (voretigene neparvovec)
Jan 09, 2017: Editas Medicine Reports on Recent Progress and 2017 Goals at J.P. Morgan Healthcare Conference
Apr 27, 2016: Editas Medicine Announces Presentations at Upcoming Scientific Conferences
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
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), H1 2018
Number of Products under Development by Companies, H1 2018
Number of Products under Development by Universities/Institutes, H1 2018
Products under Development by Companies, H1 2018
Products under Development by Universities/Institutes, H1 2018
Number of Products by Stage and Target, H1 2018
Number of Products by Stage and Mechanism of Action, H1 2018
Number of Products by Stage and Route of Administration, H1 2018
Number of Products by Stage and Molecule Type, H1 2018
Leber Congenital Amaurosis (LCA) - Pipeline by Editas Medicine Inc, H1 2018
Leber Congenital Amaurosis (LCA) - Pipeline by Novelion Therapeutics Inc, H1 2018
Leber Congenital Amaurosis (LCA) - Pipeline by ProQR Therapeutics NV, H1 2018
Leber Congenital Amaurosis (LCA) - Pipeline by Spark Therapeutics Inc, H1 2018
Leber Congenital Amaurosis (LCA) - Dormant Projects, H1 2018
List of Figures
Number of Products under Development for Leber Congenital Amaurosis (LCA), H1 2018
Number of Products under Development by Companies, H1 2018
Number of Products under Development by Universities/Institutes, H1 2018
Number of Products by Targets, H1 2018
Number of Products by Stage and Targets, H1 2018
Number of Products by Mechanism of Actions, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Routes of Administration, H1 2018
Number of Products by Stage and Routes of Administration, H1 2018
Number of Products by Molecule Types, H1 2018
Number of Products by Stage and Molecule Types, H1 2018
