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Published: Jul, 2015 | Pages:
52 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Batten Disease - Pipeline Review, H2 2015 Summary Global Markets Direct's, 'Batten disease - Pipeline Review, H2 2015', provides an overview of the Batten disease's therapeutic pipeline. This report provides comprehensive information on the therapeutic development for Batten disease, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Batten disease and special features on late-stage 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, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct's team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis. The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products. Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease. Scope - The report provides a snapshot of the global therapeutic landscape of Batten disease - The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities - The report reviews key players involved in the therapeutics development for Batten disease and enlists all their major and minor projects - The report summarizes all the dormant and discontinued pipeline projects - A review of the Batten disease products under development by companies and universities/research institutes based on information derived from company and industry-specific sources - Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages - A detailed assessment of monotherapy and combination therapy pipeline projects - Coverage of the Batten disease pipeline on the basis of target, MoA, route of administration and molecule type - Latest news and deals relating related to pipeline products Reasons to buy - Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies - Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage - Develop strategic initiatives by understanding the focus areas of leading companies - Identify and understand important and diverse types of therapeutics under development for Batten disease - Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline - 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
Table of Contents
Table of Contents 2 List of Tables 4 List of Figures 5 Introduction 6 Global Markets Direct Report Coverage 6 Batten Disease Overview 7 Therapeutics Development 8 Pipeline Products for Batten Disease - Overview 8 Pipeline Products for Batten Disease - Comparative Analysis 9 Batten Disease - Therapeutics under Development by Companies 10 Batten Disease - Therapeutics under Investigation by Universities/Institutes 11 Batten Disease - Pipeline Products Glance 12 Clinical Stage Products 12 Early Stage Products 13 Unknown Stage Products 14 Batten Disease - Products under Development by Companies 15 Batten Disease - Products under Investigation by Universities/Institutes 16 Batten Disease - Companies Involved in Therapeutics Development 17 Abeona Therapeutics, Inc. 17 BioMarin Pharmaceutical Inc. 18 CereSpir Incorporated 19 Xonovo Inc 20 Batten Disease - 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 ABO-201 - Drug Profile 30 Product Description 30 Mechanism of Action 30 R&D Progress 30 cerliponase alfa - Drug Profile 31 Product Description 31 Mechanism of Action 31 R&D Progress 31 CSP-1103 - Drug Profile 32 Product Description 32 Mechanism of Action 32 R&D Progress 32 Gene Therapy to Activate Palmitoyl Protein Thioesterase 1 for Batten Disease - Drug Profile 34 Product Description 34 Mechanism of Action 34 R&D Progress 34 Gene Therapy to Activate Tripeptidyl Peptidase 1 for Batten Disease - Drug Profile 35 Product Description 35 Mechanism of Action 35 R&D Progress 35 INI-0602 - Drug Profile 36 Product Description 36 Mechanism of Action 36 R&D Progress 36 MP-101 - Drug Profile 37 Product Description 37 Mechanism of Action 37 R&D Progress 37 Small Molecules to Inhibit CDC42 for Batten Disease - Drug Profile 38 Product Description 38 Mechanism of Action 38 R&D Progress 38 SNB-2401 - Drug Profile 39 Product Description 39 Mechanism of Action 39 R&D Progress 39 SNB-4050 - Drug Profile 40 Product Description 40 Mechanism of Action 40 R&D Progress 40 XN-001 - Drug Profile 41 Product Description 41 Mechanism of Action 41 R&D Progress 41 Batten Disease - Recent Pipeline Updates 42 Batten Disease - Dormant Projects 45 Batten Disease - Discontinued Products 46 Batten Disease - Product Development Milestones 47 Featured News & Press Releases 47 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 47 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 48 Apr 01, 2013: BioMarin Pharma Submits CTA For BMN-190 For Batten Disease 48 Mar 15, 2012: Combination treatment in mice shows promise for fatal neurological disorder in kids 49 Appendix 51 Methodology 51 Coverage 51 Secondary Research 51 Primary Research 51 Expert Panel Validation 51 Contact Us 51 Disclaimer 52
List of Tables
Number of Products under Development for Batten Disease, H2 2015 8 Number of Products under Development for Batten Disease - Comparative Analysis, H2 2015 9 Number of Products under Development by Companies, H2 2015 10 Number of Products under Investigation by Universities/Institutes, H2 2015 11 Comparative Analysis by Clinical Stage Development, H2 2015 12 Comparative Analysis by Early Stage Development, H2 2015 13 Comparative Analysis by Unknown Stage Development, H2 2015 14 Products under Development by Companies, H2 2015 15 Products under Investigation by Universities/Institutes, H2 2015 16 Batten Disease - Pipeline by Abeona Therapeutics, Inc. , H2 2015 17 Batten Disease - Pipeline by BioMarin Pharmaceutical Inc., H2 2015 18 Batten Disease - Pipeline by CereSpir Incorporated, H2 2015 19 Batten Disease - Pipeline by Xonovo Inc, H2 2015 20 Assessment by Monotherapy Products, H2 2015 21 Number of Products by Stage and Target, H2 2015 23 Number of Products by Stage and Mechanism of Action, H2 2015 25 Number of Products by Stage and Route of Administration, H2 2015 27 Number of Products by Stage and Molecule Type, H2 2015 29 Batten Disease Therapeutics - Recent Pipeline Updates, H2 2015 42 Batten Disease - Dormant Projects, H2 2015 45 Batten Disease - Discontinued Products, H2 2015 46
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