Batten Disease - Pipeline Review, H1 2016

Published: Mar, 2016 | Pages: 61 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

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