Batten Disease - Pipeline Review, H1 2016 Summary Global Markets Direct's, 'Batten Disease - Pipeline Review, H1 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
Table of Contents
Table of Contents 2 List of Tables 5 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 RegenxBio Inc. 20 Spark Therapeutics, Inc. 21 Batten Disease - Therapeutics Assessment 22 Assessment by Monotherapy Products 22 Assessment by Target 23 Assessment by Mechanism of Action 25 Assessment by Route of Administration 27 Assessment by Molecule Type 29 Drug Profiles 31 ABO-201 - Drug Profile 31 Product Description 31 Mechanism of Action 31 R&D Progress 31 cerliponase alfa - Drug Profile 32 Product Description 32 Mechanism of Action 32 R&D Progress 32 CSP-1103 - Drug Profile 33 Product Description 33 Mechanism of Action 33 R&D Progress 33 Gene Therapy to Activate Palmitoyl Protein Thioesterase 1 for Batten Disease - Drug Profile 35 Product Description 35 Mechanism of Action 35 R&D Progress 35 Gene Therapy to Activate Tripeptidyl Peptidase 1 for Batten Disease - Drug Profile 36 Product Description 36 Mechanism of Action 36 R&D Progress 36 INI-0602 - Drug Profile 38 Product Description 38 Mechanism of Action 38 R&D Progress 38 MP-101 - Drug Profile 39 Product Description 39 Mechanism of Action 39 R&D Progress 39 Small Molecules to Inhibit CDC42 for Batten Disease - Drug Profile 40 Product Description 40 Mechanism of Action 40 R&D Progress 40 SNB-2401 - Drug Profile 41 Product Description 41 Mechanism of Action 41 R&D Progress 41 SNB-4050 - Drug Profile 42 Product Description 42 Mechanism of Action 42 R&D Progress 42 SPKTPP-1 - Drug Profile 43 Product Description 43 Mechanism of Action 43 R&D Progress 43 XN-001 - Drug Profile 45 Product Description 45 Mechanism of Action 45 R&D Progress 45 Batten Disease - Recent Pipeline Updates 46 Batten Disease - Dormant Projects 50 Batten Disease - Discontinued Products 51 Batten Disease - Product Development Milestones 52 Featured News & Press Releases 52 Mar 02, 2016: Abeona Therapeutics Highlights New Preclinical Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) Data at WORLDSymposium 2016 52 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 53 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 55 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 56 Apr 01, 2013: BioMarin Pharma Submits CTA For BMN-190 For Batten Disease 57 Mar 15, 2012: Combination treatment in mice shows promise for fatal neurological disorder in kids 57 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 Batten Disease, H1 2016 8 Number of Products under Development for Batten Disease - Comparative Analysis, H1 2016 9 Number of Products under Development by Companies, H1 2016 10 Number of Products under Investigation by Universities/Institutes, H1 2016 11 Comparative Analysis by Clinical Stage Development, H1 2016 12 Comparative Analysis by Early Stage Development, H1 2016 13 Comparative Analysis by Unknown Stage Development, H1 2016 14 Products under Development by Companies, H1 2016 15 Products under Investigation by Universities/Institutes, H1 2016 16 Batten Disease - Pipeline by Abeona Therapeutics, Inc. , H1 2016 17 Batten Disease - Pipeline by BioMarin Pharmaceutical Inc., H1 2016 18 Batten Disease - Pipeline by CereSpir Incorporated, H1 2016 19 Batten Disease - Pipeline by RegenxBio Inc., H1 2016 20 Batten Disease - Pipeline by Spark Therapeutics, Inc., H1 2016 21 Assessment by Monotherapy Products, H1 2016 22 Number of Products by Stage and Target, H1 2016 24 Number of Products by Stage and Mechanism of Action, H1 2016 26 Number of Products by Stage and Route of Administration, H1 2016 28 Number of Products by Stage and Molecule Type, H1 2016 30 Batten Disease Therapeutics - Recent Pipeline Updates, H1 2016 46 Batten Disease - Dormant Projects, H1 2016 50 Batten Disease - Discontinued Products, H1 2016 51
List of Figures
Number of Products under Development for Batten Disease, H1 2016 8 Number of Products under Development for Batten Disease - Comparative Analysis, H1 2016 9 Number of Products under Development by Companies, H1 2016 10 Number of Products under Investigation by Universities/Institutes, H1 2016 11 Comparative Analysis by Clinical Stage Development, H1 2016 12 Comparative Analysis by Early Stage Products, H1 2016 13 Assessment by Monotherapy Products, H1 2016 22 Number of Products by Targets, H1 2016 23 Number of Products by Stage and Targets, H1 2016 23 Number of Products by Mechanism of Actions, H1 2016 25 Number of Products by Stage and Mechanism of Actions, H1 2016 25 Number of Products by Routes of Administration, H1 2016 27 Number of Products by Stage and Routes of Administration, H1 2016 27 Number of Products by Molecule Types, H1 2016 29 Number of Products by Stage and Molecule Types, H1 2016 29
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