Batten Disease - Pipeline Review, H1 2018

  • ID: 4464788
  • Report
  • 56 pages
  • Global Markets Direct
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Batten Disease - Pipeline Review, H1 2018

Summary:

This latest Pharmaceutical and Healthcare disease pipeline guide Batten Disease - Pipeline Review, H1 2018, 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:

This Pharmaceutical and Healthcare latest pipeline guide Batten Disease - Pipeline Review, H1 2018, 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 Phase II, Preclinical and Discovery stages are 1, 10 and 2 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 sourcedfrom the publisher'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 for Purchase:
  • 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.
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Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • Abeona Therapeutics Inc
  • Evotec AG
  • RegenxBio Inc
  • Spark Therapeutics Inc
  • MORE
Introduction

The Publisher's 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

Evotec AG

RegenxBio Inc

Spark Therapeutics Inc

Batten Disease - Drug Profiles

(gemfibrozil + tretinoin) - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ABO-201 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ABO-202 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Biologic to Activate CLN6 for Batten Disease - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

DUOC-01 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

gemfibrozil - 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

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-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

Dec 11, 2017: Polaryx Therapeutics Receives Orphan Drug Designation From the US FDA for the Treatment of Neuronal Ceroid Lipofuscinoses With PLX-100

Feb 07, 2017: Abeona Therapeutics Presents Data on ABO-201 at the 13th Annual WORLDSymposium 2017

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

Mar 02, 2016: Abeona Therapeutics Highlights New Preclinical Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) Data at WORLDSymposium 2016

Nov 11, 2015: Spark Therapeutics Announces SPK-TPP1, Its First Program Targeting Neurodegenerative 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 2018

Number of Products under Development by Companies, H1 2018

Number of Products under Development by Universities/Institutes, H1 2018

Products under Development by Companies, H1 2018

Products under Development by Universities/Institutes, H1 2018

Number of Products by Stage and Target, H1 2018

Number of Products by Stage and Mechanism of Action, H1 2018

Number of Products by Stage and Route of Administration, H1 2018

Number of Products by Stage and Molecule Type, H1 2018

Batten Disease - Pipeline by Abeona Therapeutics Inc, H1 2018

Batten Disease - Pipeline by Evotec AG, H1 2018

Batten Disease - Pipeline by RegenxBio Inc, H1 2018

Batten Disease - Pipeline by Spark Therapeutics Inc, H1 2018

Batten Disease - Dormant Projects, H1 2018

Batten Disease - Discontinued Products, H1 2018

List of Figures

Number of Products under Development for Batten Disease, H1 2018

Number of Products under Development by Companies, H1 2018

Number of Products under Development by Universities/Institutes, H1 2018

Number of Products by Top 10 Targets, H1 2018

Number of Products by Stage and Top 10 Targets, H1 2018

Number of Products by Top 10 Mechanism of Actions, H1 2018

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2018

Number of Products by Routes of Administration, H1 2018

Number of Products by Stage and Routes of Administration, H1 2018

Number of Products by Molecule Types, H1 2018

Number of Products by Stage and Molecule Types, H1
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  • Abeona Therapeutics Inc
  • Evotec AG
  • RegenxBio Inc
  • Spark Therapeutics Inc
Note: Product cover images may vary from those shown
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