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Published: Mar, 2017 | Pages:
73 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Batten Disease - Pipeline Review, H1 2017 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Batten Disease - Pipeline Review, H1 2017, provides an overview of the Batten Disease (Central Nervous System) pipeline landscape. Batten disease, a rare genetic disorder, belongs to a group of progressive degenerative neurometabolic disorders known as the neuronal ceroid lipofuscinoses. Symptoms of Batten disease usually become apparent between five and 15 years of age. Symptoms include vision loss, lack of muscle coordination, mental retardation or decreasing mental function, emotional disturbances, seizures, muscle spasms, deterioration of muscle tone and movement problems. Report Highlights Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Batten Disease - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Batten Disease (Central Nervous System), 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 Batten Disease (Central Nervous System) pipeline guide also reviews of key players involved in therapeutic development for Batten Disease 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 II, Preclinical, Discovery and Unknown stages are 1, 1, 10, 2 and 1 respectively. Similarly, the Universities portfolio in Phase II, Phase I, Preclinical and Discovery stages comprises 1, 1, 3 and 1 molecules, respectively. Batten Disease (Central Nervous System) 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 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. 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 Batten Disease (Central Nervous System). - The pipeline guide reviews pipeline therapeutics for Batten Disease (Central Nervous System) 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 Batten Disease (Central Nervous System) therapeutics and enlists all their major and minor projects. - The pipeline guide evaluates Batten Disease (Central Nervous System) 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 Batten Disease (Central Nervous System) 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 Batten Disease (Central Nervous System). - 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 Batten Disease (Central Nervous System) 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 Global Markets Direct Report Coverage Batten Disease - Overview Batten Disease - Therapeutics Development Pipeline Overview Pipeline by Companies Pipeline by Universities/Institutes Products under Development by Companies Products under Development by Universities/Institutes Batten Disease - Therapeutics Assessment Assessment by Target Assessment by Mechanism of Action Assessment by Route of Administration Assessment by Molecule Type Batten Disease - Companies Involved in Therapeutics Development Abeona Therapeutics Inc BioMarin Pharmaceutical Inc CereSpir Inc Evotec AG Ionis Pharmaceuticals Inc Mitochon Pharmaceuticals Inc RegenxBio Inc Retrotope Inc Spark Therapeutics Inc Batten Disease - Drug Profiles ABO-201 - Drug Profile Product Description Mechanism Of Action R&D Progress ABO-202 - 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 TPP1 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-001 - 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 Feb 07, 2017: Abeona Therapeutics Presents Data on ABO-202 at the 13th Annual WORLDSymposium 2017 Jan 03, 2017: Abeona Therapeutics Receives Orphan Drug Designation in The European Union for ABO-201 Gene Therapy Program in Juvenile Batten Disease Oct 07, 2016: Abeona Therapeutics Announces Publication of Preclinical Data Supporting Clinical Translation of Juvenile Batten Disease Gene Therapy Sep 15, 2016: BioMarin Announces EMA Validation of Brineura (Cerliponase Alfa) Marketing Authorization Application for Treatment of CLN2 Disease, a Form of Batten Disease Sep 06, 2016: BioMarin Announces Update to Brineura (Cerliponase Alfa) Program for Treatment of CLN2 Disease, a Form of Batten Disease 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, 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 Batten Disease - Pipeline by Abeona Therapeutics Inc, H1 2017 Batten Disease - Pipeline by BioMarin Pharmaceutical Inc, H1 2017 Batten Disease - Pipeline by CereSpir Inc, H1 2017 Batten Disease - Pipeline by Evotec AG, H1 2017 Batten Disease - Pipeline by Ionis Pharmaceuticals Inc, H1 2017 Batten Disease - Pipeline by Mitochon Pharmaceuticals Inc, H1 2017 Batten Disease - Pipeline by RegenxBio Inc, H1 2017 Batten Disease - Pipeline by Retrotope Inc, H1 2017 Batten Disease - Pipeline by Spark Therapeutics Inc, H1 2017 Batten Disease - Dormant Projects, H1 2017 Batten Disease - Discontinued Products, H1 2017
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