Batten Disease - Pipeline Review, H1 2015

Published: Feb, 2015 | Pages: 40 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Batten Disease - Pipeline Review, H1 2015

Summary

Global Markets Direct's, 'Batten Disease - Pipeline Review, H1 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 4
Introduction 5
Global Markets Direct Report Coverage 5
Batten Disease Overview 6
Therapeutics Development 7
Pipeline Products for Batten Disease - Overview 7
Pipeline Products for Batten Disease - Comparative Analysis 8
Batten Disease - Therapeutics under Development by Companies 9
Batten Disease - Therapeutics under Investigation by Universities/Institutes 10
Batten Disease - Pipeline Products Glance 11
Clinical Stage Products 11
Early Stage Products 12
Batten Disease - Products under Development by Companies 13
Batten Disease - Products under Investigation by Universities/Institutes 14
Batten Disease - Companies Involved in Therapeutics Development 15
BioMarin Pharmaceutical Inc. 15
ReGenX Biosciences, LLC 16
Batten Disease - Therapeutics Assessment 17
Assessment by Monotherapy Products 17
Assessment by Combination Products 18
Assessment by Target 19
Assessment by Mechanism of Action 21
Assessment by Route of Administration 23
Assessment by Molecule Type 25
Drug Profiles 27
cerliponase alfa - Drug Profile 27
Product Description 27
Mechanism of Action 27
R&D Progress 27
Gene Therapy + Bone Marrow Transplantation - Drug Profile 28
Product Description 28
Mechanism of Action 28
R&D Progress 28
Gene Therapy to Activate Tripeptidyl Peptidase 1 for Batten Disease - Drug Profile 29
Product Description 29
Mechanism of Action 29
R&D Progress 29
Small Molecules for Batten Disease - Drug Profile 30
Product Description 30
Mechanism of Action 30
R&D Progress 30
Small Molecules to Inhibit CDC42 for Juvenile Neuronal Ceroid Lipofuscinosis - Drug Profile 31
Product Description 31
Mechanism of Action 31
R&D Progress 31
XN-001 - Drug Profile 32
Product Description 32
Mechanism of Action 32
R&D Progress 32
Batten Disease - Recent Pipeline Updates 33
Batten Disease - Discontinued Products 34
Batten Disease - Product Development Milestones 35
Featured News & Press Releases 35
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 35
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 36
Apr 01, 2013: BioMarin Pharma Submits CTA For BMN-190 For Batten Disease 36
Mar 15, 2012: Combination treatment in mice shows promise for fatal neurological disorder in kids 37
Appendix 39
Methodology 39
Coverage 39
Secondary Research 39
Primary Research 39
Expert Panel Validation 39
Contact Us 39
Disclaimer 40

List of Tables
Number of Products under Development for Batten Disease, H1 2015 7
Number of Products under Development for Batten Disease - Comparative Analysis, H1 2015 8
Number of Products under Development by Companies, H1 2015 9
Number of Products under Investigation by Universities/Institutes, H1 2015 10
Comparative Analysis by Clinical Stage Development, H1 2015 11
Comparative Analysis by Early Stage Development, H1 2015 12
Products under Development by Companies, H1 2015 13
Products under Investigation by Universities/Institutes, H1 2015 14
Batten Disease - Pipeline by BioMarin Pharmaceutical Inc., H1 2015 15
Batten Disease - Pipeline by ReGenX Biosciences, LLC, H1 2015 16
Assessment by Monotherapy Products, H1 2015 17
Assessment by Combination Products, H1 2015 18
Number of Products by Stage and Target, H1 2015 20
Number of Products by Stage and Mechanism of Action, H1 2015 22
Number of Products by Stage and Route of Administration, H1 2015 24
Number of Products by Stage and Molecule Type, H1 2015 26
Batten Disease Therapeutics - Recent Pipeline Updates, H1 2015 33
Batten Disease - Discontinued Products, H1 2015 34