Batten Disease - Pipeline Review, H2 2016 - Global Markets Direct - Market Research

Batten Disease - Pipeline Review, H2 2016

Batten Disease - Pipeline Review, H2 2016 - Global Markets Direct - Market Research
Batten Disease - Pipeline Review, H2 2016
Published Aug 31, 2016
72 pages — Published Aug 31, 2016
Price US$ 2,000.00  |  Buy this Report Now

About This Report

  
Abstract:

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 an

  
Source:
Document ID
GMDHC8413IDB
Industry
Format:
PDF Adobe Acrobat
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Sections

TitleStarting PageNumber of Pages
Table of Contents25
  List of Tables51
  List of Figures61
Introduction71
  Global Markets Direct Report Coverage71
Batten Disease Overview81
Therapeutics Development92
  Pipeline Products for Batten Disease Overview91
  Pipeline Products for Batten Disease Comparative Analysis101
Batten Disease Therapeutics under Development by Companies111
Batten Disease Therapeutics under Investigation by Universities/Institutes121
Batten Disease Pipeline Products Glance134
  Late Stage Products131
  Clinical Stage Products141
  Early Stage Products151
  Unknown Stage Products161
Batten Disease Products under Development by Companies171
Batten Disease Products under Investigation by Universities/Institutes181
Batten Disease Companies Involved in Therapeutics Development199
  Abeona Therapeutics, Inc.191
  BioMarin Pharmaceutical Inc.201
  CereSpir Incorporated211
  Evotec AG221
  Ionis Pharmaceuticals, Inc.231
  Mitochon Pharmaceuticals, Inc.241
  RegenxBio Inc.251
  Retrotope, Inc261
  Spark Therapeutics, Inc.271
Batten Disease Therapeutics Assessment289
  Assessment by Monotherapy Products281
  Assessment by Target292
  Assessment by Mechanism of Action312
  Assessment by Route of Administration332
  Assessment by Molecule Type352
Drug Profiles3722
  ABO-201 Drug Profile371
  Antisense Oligonucleotides to Activate CLN3 for Batten Disease Drug Profile381
  cerliponase alfa Drug Profile392
  CSP-1103 Drug Profile412
  DUOC-01 Drug Profile431
  Gene Therapy to Activate CLN1 and CLN2 for Batten Disease Drug Profile441
  Gene Therapy to Activate CLN6 for Batten Disease Drug Profile451
  Gene Therapy to Activate Palmitoyl Protein Thioesterase 1 for Batten Disease Drug Profile461
  Gene Therapy to Activate Tripeptidyl Peptidase 1 for Batten Disease Drug Profile472
  INI-0602 Drug Profile491
  MP-101 Drug Profile501
  RT-002 Drug Profile511
  Small Molecule for Batten Disease Drug Profile521
  Small Molecules for Juvenile Batten Disease Drug Profile531
  Small Molecules to Inhibit CDC42 for Batten Disease Drug Profile541
  SNB-2401 Drug Profile551
  SNB-4050 Drug Profile561
  SPKTPP-1 Drug Profile571
  XN-001 Drug Profile581
Batten Disease Dormant Projects591
Batten Disease Discontinued Products601
Batten Disease Product Development Milestones6110
  Featured News &Press Releases611
    Jul 27, 2016: FDA Accepts BLA for BioMarins Cerliponase Alfa for CLN2 Disease, Form of Batten Disease611
    May 03, 2016: BioMarin Announces EMA Grants Accelerated Assessment for Cerliponase Alfa, Experimental Treatment for a Form of Batten Disease611
    Mar 02, 2016: Abeona Therapeutics Highlights New Preclinical Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) Data at WORLDSymposium 2016621
    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 2016633
    Nov 11, 2015: Spark Therapeutics Announces SPK-TPP1, Its First Program Targeting Neurodegenerative Disease661
    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 Disease662
    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 Disease681
    Apr 01, 2013: BioMarin Pharma Submits CTA For BMN-190 For Batten Disease681
    Mar 15, 2012: Combination treatment in mice shows promise for fatal neurological disorder in kids692
Appendix712
  Methodology711
  Coverage711
  Secondary Research711
  Primary Research711
  Expert Panel Validation711
  Contact Us711
  Disclaimer721

Table Of Contents

Global Markets Direct - Market Research—Global Markets Direct is a leading provider of global business intelligence and market analysis. It publishes a range of high quality market reports created by its large research and analysis capability and drawing from its extensive unique databases of industry-specific information.

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Cite this Report

  
MLA:
Global Markets Direct - Market Research. "Batten Disease - Pipeline Review, H2 2016" Aug 31, 2016. Alacra Store. May 14, 2025. <http://www.alacrastore.com/storecontent/Global-Markets-Direct-Market-Research/Batten-Disease-Pipeline-Review-H2-2016-2088-16482>
  
APA:
Global Markets Direct - Market Research. (2016). Batten Disease - Pipeline Review, H2 2016 Aug 31, 2016. New York, NY: Alacra Store. Retrieved May 14, 2025 from <http://www.alacrastore.com/storecontent/Global-Markets-Direct-Market-Research/Batten-Disease-Pipeline-Review-H2-2016-2088-16482>
  
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