Batten Disease - Pipeline Review, H2 2016

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Batten Disease - Pipeline Review, H2 2016 Summary Global Markets Directs, 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 Directs 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 Directs 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 its 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 6 Introduction 7 Global Markets Direct Report Coverage 7 Batten Disease Overview 8 Therapeutics Development 9 Pipeline Products for Batten Disease - Overview 9 Pipeline Products for Batten Disease - Comparative Analysis 10 Batten Disease - Therapeutics under Development by Companies 11 Batten Disease - Therapeutics under Investigation by Universities/Institutes 12 Batten Disease - Pipeline Products Glance 13 Late Stage Products 13 Clinical Stage Products 14 Early Stage Products 15 Unknown Stage Products 16 Batten Disease - Products under Development by Companies 17 Batten Disease - Products under Investigation by Universities/Institutes 18 Batten Disease - Companies Involved in Therapeutics Development 19 Abeona Therapeutics, Inc. 19 BioMarin Pharmaceutical Inc. 20 CereSpir Incorporated 21 Evotec AG 22 Ionis Pharmaceuticals, Inc. 23 Mitochon Pharmaceuticals, Inc. 24 RegenxBio Inc. 25 Retrotope, Inc 26 Spark Therapeutics, Inc. 27 Batten Disease - Therapeutics Assessment 28 Assessment by Monotherapy Products 28 Assessment by Target 29 Assessment by Mechanism of Action 31 Assessment by Route of Administration 33 Assessment by Molecule Type 35 Drug Profiles 37 ABO-201 - Drug Profile 37 Product Description 37 Mechanism Of Action 37 R&D Progress 37 Antisense Oligonucleotides to Activate CLN3 for Batten Disease - Drug Profile 38 Product Description 38 Mechanism Of Action 38 R&D Progress 38 cerliponase alfa - Drug Profile 39 Product Description 39 Mechanism Of Action 39 R&D Progress 39 CSP-1103 - Drug Profile 41 Product Description 41 Mechanism Of Action 41 R&D Progress 41 DUOC-01 - Drug Profile 43 Product Description 43 Mechanism Of Action 43 R&D Progress 43 Gene Therapy to Activate CLN1 and CLN2 for Batten Disease - Drug Profile 44 Product Description 44 Mechanism Of Action 44 R&D Progress 44 Gene Therapy to Activate CLN6 for Batten Disease - Drug Profile 45 Product Description 45 Mechanism Of Action 45 R&D Progress 45 Gene Therapy to Activate Palmitoyl Protein Thioesterase 1 for Batten Disease - Drug Profile 46 Product Description 46 Mechanism Of Action 46 R&D Progress 46 Gene Therapy to Activate Tripeptidyl Peptidase 1 for Batten Disease - Drug Profile 47 Product Description 47 Mechanism Of Action 47 R&D Progress 47 INI-0602 - Drug Profile 49 Product Description 49 Mechanism Of Action 49 R&D Progress 49 MP-101 - Drug Profile 50 Product Description 50 Mechanism Of Action 50 R&D Progress 50 RT-002 - Drug Profile 51 Product Description 51 Mechanism Of Action 51 R&D Progress 51 Small Molecule for Batten Disease - Drug Profile 52 Product Description 52 Mechanism Of Action 52 R&D Progress 52 Small Molecules for Juvenile Batten Disease - Drug Profile 53 Product Description 53 Mechanism Of Action 53 R&D Progress 53 Small Molecules to Inhibit CDC42 for Batten Disease - Drug Profile 54 Product Description 54 Mechanism Of Action 54 R&D Progress 54 SNB-2401 - Drug Profile 55 Product Description 55 Mechanism Of Action 55 R&D Progress 55 SNB-4050 - Drug Profile 56 Product Description 56 Mechanism Of Action 56 R&D Progress 56 SPKTPP-1 - Drug Profile 57 Product Description 57 Mechanism Of Action 57 R&D Progress 57 XN-001 - Drug Profile 58 Product Description 58 Mechanism Of Action 58 R&D Progress 58 Batten Disease - Dormant Projects 59 Batten Disease - Discontinued Products 60 Batten Disease - Product Development Milestones 61 Featured News & Press Releases 61 Jul 27, 2016: FDA Accepts BLA for BioMarins Cerliponase Alfa for CLN2 Disease, Form of Batten Disease 61 May 03, 2016: BioMarin Announces EMA Grants Accelerated Assessment for Cerliponase Alfa, Experimental Treatment for a Form of Batten Disease 61 Mar 02, 2016: Abeona Therapeutics Highlights New Preclinical Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) Data at WORLDSymposium 2016 62 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 63 Nov 11, 2015: Spark Therapeutics Announces SPK-TPP1, Its First Program Targeting Neurodegenerative Disease 66 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 66 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 68 Apr 01, 2013: BioMarin Pharma Submits CTA For BMN-190 For Batten Disease 68 Mar 15, 2012: Combination treatment in mice shows promise for fatal neurological disorder in kids 69 Appendix 71 Methodology 71 Coverage 71 Secondary Research 71 Primary Research 71 Expert Panel Validation 71 Contact Us 71 Disclaimer 72

List of Tables Number of Products under Development for Batten Disease, H2 2016 9 Number of Products under Development for Batten Disease - Comparative Analysis, H2 2016 10 Number of Products under Development by Companies, H2 2016 11 Number of Products under Investigation by Universities/Institutes, H2 2016 12 Comparative Analysis by Late Stage Development, H2 2016 13 Comparative Analysis by Clinical Stage Development, H2 2016 14 Comparative Analysis by Early Stage Development, H2 2016 15 Comparative Analysis by Unknown Stage Development, H2 2016 16 Products under Development by Companies, H2 2016 17 Products under Investigation by Universities/Institutes, H2 2016 18 Batten Disease - Pipeline by Abeona Therapeutics, Inc. , H2 2016 19 Batten Disease - Pipeline by BioMarin Pharmaceutical Inc., H2 2016 20 Batten Disease - Pipeline by CereSpir Incorporated, H2 2016 21 Batten Disease - Pipeline by Evotec AG, H2 2016 22 Batten Disease - Pipeline by Ionis Pharmaceuticals, Inc., H2 2016 23 Batten Disease - Pipeline by Mitochon Pharmaceuticals, Inc., H2 2016 24 Batten Disease - Pipeline by RegenxBio Inc., H2 2016 25 Batten Disease - Pipeline by Retrotope, Inc, H2 2016 26 Batten Disease - Pipeline by Spark Therapeutics, Inc., H2 2016 27 Assessment by Monotherapy Products, H2 2016 28 Number of Products by Stage and Target, H2 2016 30 Number of Products by Stage and Mechanism of Action, H2 2016 32 Number of Products by Stage and Route of Administration, H2 2016 34 Number of Products by Stage and Molecule Type, H2 2016 36 Batten Disease - Dormant Projects, H2 2016 59 Batten Disease - Discontinued Products, H2 2016 60


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