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Batten Disease - Pipeline Review, H2 2016

Batten Disease - Pipeline Review, H2 2016

Summary

Global Markets Direct’s, ‘Batten Disease - Pipeline Review, H2 2016’, provides an overview of the Batten Disease pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Batten Disease, 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 Batten Disease 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 Batten Disease
    • The report reviews pipeline therapeutics for Batten Disease 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 Batten Disease therapeutics and enlists all their major and minor projects
    • The report assesses Batten Disease 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 Batten Disease
    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 Batten Disease
    • 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 Batten Disease 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


    Introduction
    Global Markets Direct Report Coverage
    Batten Disease Overview
    Therapeutics Development
    Pipeline Products for Batten Disease - Overview
    Pipeline Products for Batten Disease - Comparative Analysis
    Batten Disease - Therapeutics under Development by Companies
    Batten Disease - Therapeutics under Investigation by Universities/Institutes
    Batten Disease - Pipeline Products Glance
    Late Stage Products
    Clinical Stage Products
    Early Stage Products
    Unknown Stage Products
    Batten Disease - Products under Development by Companies
    Batten Disease - Products under Investigation by Universities/Institutes
    Batten Disease - Companies Involved in Therapeutics Development
    Abeona Therapeutics, Inc.
    BioMarin Pharmaceutical Inc.
    CereSpir Incorporated
    Evotec AG
    Ionis Pharmaceuticals, Inc.
    Mitochon Pharmaceuticals, Inc.
    RegenxBio Inc.
    Retrotope, Inc
    Spark Therapeutics, Inc.
    Batten Disease - Therapeutics Assessment
    Assessment by Monotherapy Products
    Assessment by Target
    Assessment by Mechanism of Action
    Assessment by Route of Administration
    Assessment by Molecule Type
    Drug Profiles
    ABO-201 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Antisense Oligonucleotides to Activate CLN3 for Batten Disease - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    cerliponase alfa - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    CSP-1103 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    DUOC-01 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Gene Therapy to Activate CLN1 and CLN2 for Batten Disease - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Gene Therapy to Activate CLN6 for Batten Disease - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Gene Therapy to Activate Palmitoyl Protein Thioesterase 1 for Batten Disease - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Gene Therapy to Activate Tripeptidyl Peptidase 1 for Batten Disease - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    INI-0602 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    MP-101 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    RT-002 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Small Molecule for Batten Disease - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Small Molecules for Juvenile Batten Disease - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Small Molecules to Inhibit CDC42 for Batten Disease - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    SNB-2401 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    SNB-4050 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    SPKTPP-1 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    XN-001 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Batten Disease - Dormant Projects
    Batten Disease - Discontinued Products
    Batten Disease - Product Development Milestones
    Featured News & Press Releases
    Jul 27, 2016: FDA Accepts BLA for BioMarins Cerliponase Alfa for CLN2 Disease, Form of Batten Disease
    May 03, 2016: BioMarin Announces EMA Grants Accelerated Assessment for Cerliponase Alfa, Experimental Treatment for a Form of Batten Disease
    Mar 02, 2016: Abeona Therapeutics Highlights New Preclinical Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) Data at WORLDSymposium 2016
    Mar 02, 2016: BioMarin Announces Positive Data From Cerliponase Alfa Program for Treatment of CLN2 Disease, a Form of Batten Disease, at 12th Annual WORLDSymposium 2016
    Nov 11, 2015: Spark Therapeutics Announces SPK-TPP1, Its First Program Targeting Neurodegenerative Disease
    Jan 12, 2015: BioMarin Provides Preliminary Data From Ongoing Phase 1/2 Pivotal Study of BMN 190 for Treatment of CLN2 Disorder, a Form of Batten Disease
    Sep 23, 2013: BioMarin Doses First Patient in Phase 1/2 Trial With BMN 190 for the Treatment of Neuronal Ceroid Lipofuscinosis Type 2, a Form of Batten Disease
    Apr 01, 2013: BioMarin Pharma Submits CTA For BMN-190 For Batten Disease
    Mar 15, 2012: Combination treatment in mice shows promise for fatal neurological disorder in kids
    Appendix
    Methodology
    Coverage
    Secondary Research
    Primary Research
    Expert Panel Validation
    Contact Us
    Disclaimer
    List of Tables
    Number of Products under Development for Batten Disease, H2 2016
    Number of Products under Development for Batten Disease - Comparative Analysis, H2 2016
    Number of Products under Development by Companies, H2 2016
    Number of Products under Investigation by Universities/Institutes, H2 2016
    Comparative Analysis by Late Stage Development, H2 2016
    Comparative Analysis by Clinical Stage Development, H2 2016
    Comparative Analysis by Early Stage Development, H2 2016
    Comparative Analysis by Unknown Stage Development, H2 2016
    Products under Development by Companies, H2 2016
    Products under Investigation by Universities/Institutes, H2 2016
    Batten Disease - Pipeline by Abeona Therapeutics, Inc. , H2 2016
    Batten Disease - Pipeline by BioMarin Pharmaceutical Inc., H2 2016
    Batten Disease - Pipeline by CereSpir Incorporated, H2 2016
    Batten Disease - Pipeline by Evotec AG, H2 2016
    Batten Disease - Pipeline by Ionis Pharmaceuticals, Inc., H2 2016
    Batten Disease - Pipeline by Mitochon Pharmaceuticals, Inc., H2 2016
    Batten Disease - Pipeline by RegenxBio Inc., H2 2016
    Batten Disease - Pipeline by Retrotope, Inc, H2 2016
    Batten Disease - Pipeline by Spark Therapeutics, Inc., H2 2016
    Assessment by Monotherapy Products, H2 2016
    Number of Products by Stage and Target, H2 2016
    Number of Products by Stage and Mechanism of Action, H2 2016
    Number of Products by Stage and Route of Administration, H2 2016
    Number of Products by Stage and Molecule Type, H2 2016
    Batten Disease - Dormant Projects, H2 2016
    Batten Disease - Discontinued Products, H2 2016
    List of Figures
    Number of Products under Development for Batten Disease, H2 2016
    Number of Products under Development for Batten Disease - Comparative Analysis, H2 2016
    Number of Products under Development by Companies, H2 2016
    Number of Products under Investigation by Universities/Institutes, H2 2016
    Comparative Analysis by Clinical Stage Development, H2 2016
    Comparative Analysis by Early Stage Products, H2 2016
    Assessment by Monotherapy Products, H2 2016
    Number of Products by Targets, H2 2016
    Number of Products by Stage and Targets, H2 2016
    Number of Products by Mechanism of Actions, H2 2016
    Number of Products by Stage and Mechanism of Actions, H2 2016
    Number of Products by Routes of Administration, H2 2016
    Number of Products by Stage and Routes of Administration, H2 2016
    Number of Products by Molecule Types, H2 2016
    Number of Products by Stage and Molecule Types, H2 2016

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