Batten Disease - Pipeline Review, H1 2019

Global Markets Direct’s, ‘Batten Disease - Pipeline Review, H1 2019’, 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

Companies mentioned

Abeona Therapeutics Inc

Amicus Therapeutics Inc

Exicure Inc

Recursion Pharmaceuticals Inc

RegenxBio Inc

Spark Therapeutics Inc

Abeona Therapeutics Inc

Amicus Therapeutics Inc

Exicure Inc

Recursion Pharmaceuticals Inc

RegenxBio Inc

Spark Therapeutics Inc

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Batten Disease – Overview

Batten Disease – Therapeutics Development

Pipeline Overview

Pipeline ...

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct 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

Amicus Therapeutics Inc

Exicure Inc

Recursion Pharmaceuticals Inc

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 for Batten Disease – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate CLN3 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 CLN8 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 TPP1 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

Oligonucleotides for Neurology – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

PLX-300 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Recombinant Palmitoyl-Protein Thioesterase-1 Replacement for Batten Disease – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

RGX-181 – 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 Batten Disease – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules for 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

Jan 31, 2019: Spark Therapeutics announces presentation of preclinical data in CLN2 disease at 15th annual WORLDSymposium

Jan 31, 2019: REGENXBIO receives Rare Pediatric Disease Designation for RGX-181 Gene Therapy for the Treatment of CLN2 Form of Batten Disease

Dec 06, 2018: Abeona Therapeutics provides update on Infantile Batten Disease drug candidate ABO-202 at 2018 R&D Day

Nov 14, 2018: FDA grants Orphan Drug Designation to RGX-181 gene therapy for the treatment of CLN2 form of Batten disease

Aug 30, 2018: REGENXBIO Announces Pipeline Expansion With RGX-181 Gene Therapy For CLN2 Form Of Batten Disease

Apr 20, 2018: Abeona Therapeutics Receives Orphan Drug Designation in the European Union for ABO-202 Gene Therapy Program in Batten Disease

Mar 15, 2018: Abeona Therapeutics Receives FDA Rare Pediatric Disease Designation for ABO-202 Gene Therapy Program in CLN1 Disease

Feb 12, 2018: Abeona Therapeutics Receives FDA Orphan Drug Designation for ABO-202 Gene Therapy Program in Infantile Batten Disease

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

Aug 07, 2017: Polaryx Therapeutics Receives Orphan Drug Designation From the U.S. FDA for PLX-200

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

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

List of Tables

Number of Products under Development for Batten Disease, H1 2019

Number of Products under Development by Companies, H1 2019

Number of Products ...

List of Tables

Number of Products under Development for Batten Disease, H1 2019

Number of Products under Development by Companies, H1 2019

Number of Products under Development by Universities/Institutes, H1 2019

Products under Development by Companies, H1 2019

Products under Development by Companies, H1 2019 (Contd..1), H1 2019

Products under Development by Universities/Institutes, H1 2019

Number of Products by Stage and Target, H1 2019

Number of Products by Stage and Mechanism of Action, H1 2019

Number of Products by Stage and Route of Administration, H1 2019

Number of Products by Stage and Molecule Type, H1 2019

Batten Disease – Pipeline by Abeona Therapeutics Inc, H1 2019

Batten Disease – Pipeline by Amicus Therapeutics Inc, H1 2019

Batten Disease – Pipeline by Exicure Inc, H1 2019

Batten Disease – Pipeline by Recursion Pharmaceuticals Inc, H1 2019

Batten Disease – Pipeline by RegenxBio Inc, H1 2019

Batten Disease – Pipeline by Spark Therapeutics Inc, H1 2019

Batten Disease – Dormant Projects, H1 2019

Batten Disease – Discontinued Products, H1 2019

List of Figures

List of Figures

Number of Products under Development for Batten Disease, H1 2019

Number of Products under Development by Companies, H1 2019

Number of Products ...

List of Figures

Number of Products under Development for Batten Disease, H1 2019

Number of Products under Development by Companies, H1 2019

Number of Products under Development by Universities/Institutes, H1 2019

Number of Products by Top 10 Targets, H1 2019

Number of Products by Stage and Top 10 Targets, H1 2019

Number of Products by Top 10 Mechanism of Actions, H1 2019

Number of Products by Stage and Top 10 Mechanism of Actions, H1 2019

Number of Products by Top 10 Routes of Administration, H1 2019

Number of Products by Stage and Top 10 Routes of Administration, H1 2019

Number of Products by Molecule Types, H1 2019

Number of Products by Stage and Molecule Types, H1 2019

    Pricing

Discounts available for multiple report purchases.

reportstore@globalmarketsdirect.com
+44 (0) 161 359 5414

Saved reports