• +1-646-491-9876
    • +91-20-67278686

    Search

    Leber Congenital Amaurosis - Pipeline Review, H1 2017

    Leber Congenital Amaurosis - Pipeline Review, H1 2017

    • Report Code ID: RW0001767482
    • Category Pharmaceuticals
    • No. of Pages 51
    • Publication Month Apr-17
    • Publisher Name Global Markets Direct
    Leber Congenital Amaurosis - Pipeline Review, H1 2017

    Summary

    Publisher's latest Pharmaceutical and Healthcare disease pipeline guide Leber Congenital Amaurosis - Pipeline Review, H1 2017, provides an overview of the Leber Congenital Amaurosis (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

    Publisher's Pharmaceutical and Healthcare latest pipeline guide Leber Congenital Amaurosis - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Leber Congenital Amaurosis (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 (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 1 molecules, respectively.

    Leber Congenital Amaurosis (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 Publisher'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 (Genetic Disorders) .
    - The pipeline guide reviews pipeline therapeutics for Leber Congenital Amaurosis (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 (Genetic Disorders) therapeutics and enlists all their major and minor projects.
    - The pipeline guide evaluates Leber Congenital Amaurosis (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 (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 (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 (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
    List of Tables
    List of Figures
    Introduction
    Publisher 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
    AmpliPhi Biosciences Corp
    Editas Medicine Inc
    Novelion Therapeutics Inc
    ProQR Therapeutics NV
    Spark Therapeutics Inc
    Leber Congenital Amaurosis (LCA) - Drug Profiles
    Gene Therapy for Leber Congenital Amaurosis 10 - 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 Retinoid Isomerohydrolase 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
    HORARPE-65 - 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 09, 2017: Editas Medicine Reports on Recent Progress and 2017 Goals at J.P. Morgan Healthcare Conference
    Jun 01, 2016: ProQR's Drug Candidate QR-110 for Leber's Congenital Amaurosis Type 10 Receives EMA and FDA Orphan Drug Designation
    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
    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) , H1 2017
    Number of Products under Development by Companies, H1 2017
    Number of Products under Development by Universities/Institutes, H1 2017
    Products under Development by Companies, H1 2017
    Products under Development by Universities/Institutes, H1 2017
    Number of Products by Stage and Target, H1 2017
    Number of Products by Stage and Mechanism of Action, H1 2017
    Number of Products by Stage and Route of Administration, H1 2017
    Number of Products by Stage and Molecule Type, H1 2017
    Leber Congenital Amaurosis (LCA) - Pipeline by AmpliPhi Biosciences Corp, H1 2017
    Leber Congenital Amaurosis (LCA) - Pipeline by Editas Medicine Inc, H1 2017
    Leber Congenital Amaurosis (LCA) - Pipeline by Novelion Therapeutics Inc, H1 2017
    Leber Congenital Amaurosis (LCA) - Pipeline by ProQR Therapeutics NV, H1 2017
    Leber Congenital Amaurosis (LCA) - Pipeline by Spark Therapeutics Inc, H1 2017
    Leber Congenital Amaurosis (LCA) - Dormant Projects, H1 2017

    List of Figures

    Number of Products under Development for Leber Congenital Amaurosis (LCA) , H1 2017
    Number of Products under Development by Companies, H1 2017
    Number of Products under Development by Universities/Institutes, H1 2017
    Number of Products by Targets, H1 2017
    Number of Products by Stage and Targets, H1 2017
    Number of Products by Mechanism of Actions, H1 2017
    Number of Products by Stage and Mechanism of Actions, H1 2017
    Number of Products by Routes of Administration, H1 2017
    Number of Products by Stage and Routes of Administration, H1 2017
    Number of Products by Molecule Types, H1 2017
    Number of Products by Stage and Molecule Types, H1 2017
    AmpliPhi Biosciences Corp
    Editas Medicine Inc
    Novelion Therapeutics Inc
    ProQR Therapeutics NV
    Spark Therapeutics Inc

    Request for Sample

    Report Url http://www.reportsweb.com//leber-congenital-amaurosis-pipeline-review-h1-2017&type=sample
    Report Name
    First Name
    Last Name
    Email
    Phone
    Company
    Job Title
    Country
    Publisher
    Report Code
    Comments
     

    Inquire before Buying

    Report Url http://www.reportsweb.com//leber-congenital-amaurosis-pipeline-review-h1-2017&type=buying
    Report Name
    First Name
    Last Name
    Email
    Phone
    Company
    Job Title
    Country
    Publisher
    Report Code
    Comments
     

    Inquire for Discount

    Report Url http://www.reportsweb.com//leber-congenital-amaurosis-pipeline-review-h1-2017&type=discount
    Report Name
    First Name
    Last Name
    Email
    Phone
    Company
    Job Title
    Country
    Publisher
    Report Code
    Comments