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Batten Disease-Pipeline Review, H2 2016

Batten Disease-Pipeline Review, H2 2016


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Executive Summary

Batten Disease-Pipeline Review, H2 2016

Summary

Global Markets Direct's, '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 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 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 Figures

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 Clinical Stage Development, H2 2016 14

Comparative Analysis by Early Stage Products, H2 2016 15

Assessment by Monotherapy Products, H2 2016 28

Number of Products by Targets, H2 2016 29

Number of Products by Stage and Targets, H2 2016 29

Number of Products by Mechanism of Actions, H2 2016 31

Number of Products by Stage and Mechanism of Actions, H2 2016 31

Number of Products by Routes of Administration, H2 2016 33

Number of Products by Stage and Routes of Administration, H2 2016 33

Number of Products by Molecule Types, H2 2016 35

Number of Products by Stage and Molecule Types, H2 2016 35

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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Abeona Therapeutics, Inc.

BioMarin Pharmaceutical Inc.

CereSpir Incorporated

Evotec AG

Ionis Pharmaceuticals, Inc.

Mitochon Pharmaceuticals, Inc.

RegenxBio Inc.

Retrotope, Inc

Spark Therapeutics, Inc.

Batten Disease Therapeutic Products under Development, Key Players in Batten Disease Therapeutics, Batten Disease Pipeline Overview, Batten Disease Pipeline, Batten Disease Pipeline Assessment


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