Epidermolysis Bullosa – Pipeline Review, H1 2017

Global Markets Direct’s, ‘Epidermolysis Bullosa – Pipeline Review, H1 2017’, provides an overview of the Epidermolysis Bullosa pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Epidermolysis Bullosa, 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 Epidermolysis Bullosa 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 Epidermolysis Bullosa

The report reviews pipeline therapeutics for Epidermolysis Bullosa 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 Epidermolysis Bullosa therapeutics and enlists all their major and minor projects

The report assesses Epidermolysis Bullosa 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 Epidermolysis Bullosa

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 Epidermolysis Bullosa

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 Epidermolysis Bullosa 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

Almirall SA

Amryt Pharma plc

Anterogen Co Ltd

Berg LLC

CSL Ltd

Exicure Inc

Fibrocell Science Inc

GlaxoSmithKline Plc

Immusoft Corp

InMed Pharmaceuticals Inc

Karus Therapeutics Ltd

Mesoblast Ltd

ProQR Therapeutics NV

RegeneRx Biopharmaceuticals Inc

Scioderm Inc

Stratatech Corp

TransDerm Inc

WAVE Life Sciences Ltd

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Epidermolysis Bullosa - Overview

Epidermolysis Bullosa - Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Epidermolysis Bullosa - Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Epidermolysis Bullosa - Companies Involved in Therapeutics Development

Abeona Therapeutics Inc

Almirall SA

Amryt Pharma plc

Anterogen Co Ltd

Berg LLC

CSL Ltd

Exicure Inc

Fibrocell Science Inc

GlaxoSmithKline Plc

Immusoft Corp

InMed Pharmaceuticals Inc

Karus Therapeutics Ltd

Mesoblast Ltd

ProQR Therapeutics NV

RegeneRx Biopharmaceuticals Inc

Scioderm Inc

Stratatech Corp

TransDerm Inc

WAVE Life Sciences Ltd

Epidermolysis Bullosa - Drug Profiles

AC-203 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ADP-31415 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

allantoin - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ALLO-ASC - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Antisense Oligonucleotide 1 to Inhibit Keratin for Epidermolysis Bullosa Simplex - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Antisense Oligonucleotide 2 to Inhibit Keratin for Epidermolysis Bullosa Simplex - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Antisense Oligonucleotide to Inhibit IL-1 Beta for Epidermolysis Bullosa - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Biologic for Epidermolysis Bullosa - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

birch bark extract - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Cell Therapy for Dermatology and Immunology - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Cell Therapy for Epidermolysis Bullosa - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Cell Therapy for Recessive Dystrophic Epidermolysis Bullosa - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

EB-101 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

EB-201 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

FCX-007 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy for Epidermolysis Bullosa - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy for Epidermolysis Bullosa - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate Lysyl Hydroxylase-3 for Recessive Dystrophic Epidermolysis Bullosa - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ICX-RHY - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

INM-750 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

KA-1463 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

M-230 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

QR-313 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

QRX-323 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

remestemcel-L - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

RGN-137 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

sirolimus - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

TXA-127 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ubidecarenone - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Epidermolysis Bullosa - Dormant Projects

Epidermolysis Bullosa - Discontinued Products

Epidermolysis Bullosa - Product Development Milestones

Featured News & Press Releases

May 02, 2017: Abeona Therapeutics Provides Update on EB-101 Gene Therapy for Severe Form of Epidermolysis Bullosa from the Society for Investigative Dermatology Conference

Apr 25, 2017: Abeona Therapeutics Announces EB-101 Clinical Update to be Presented at the Society for Investigative Dermatology Annual Meeting

Apr 24, 2017: Amryt Pharma Announces Enrollment of First Patient in Phase 3 Clinical Trial for AP101 in EB

Apr 03, 2017: Amicus Therapeutics Completes Enrollment in ESSENCE Phase 3 Epidermolysis Bullosa Clinical Study

Mar 28, 2017: Amryt Pharma: Phase 3 Clinical Trial for AP101 in EB

Mar 08, 2017: Abeona Therapeutics Receives Orphan Drug Designation in the European Union for EB-101 Gene Therapy Clinical Trial for Epidermolysis Bullosa

Mar 06, 2017: Castle Creek Pharmaceuticals Announces Presentation of Phase 2 Clinical Data for Diacerein 1% Ointment in Treatment of Epidermolysis Bullosa Simplex

Mar 06, 2017: Amryt Pharma Announces AP101 trial discussions completed with FDA & EMA

Feb 27, 2017: RegeneRx Licensee Receives FDA Permission to Conduct a Phase 3 Trial of RGN-137 for Epidermolysis Bullosa in U.S.

Feb 23, 2017: Fibrocell Announces Dosing of First Patient in Phase I/II Clinical Trial of FCX-007 Gene Therapy for Treatment of Recessive Dystrophic Epidermolysis Bullosa

Feb 07, 2017: Amryt Pharma: Grant of patent in Japan for lead drug candidate, AP101 (Episalvan) for Treatment of Epidermolysis Bullosa

Jan 18, 2017: Tarix Orphan Receives Rare Pediatric Disease Designation for TXA127 for Recessive Dystrophic Epidermolysis Bullosa

Jan 05, 2017: Fibrocell Announces FDA Fast Track Designation of FCX-007 for Treatment of Recessive Dystrophic Epidermolysis Bullosa

Dec 02, 2016: Amryt Pharma: €20m facility agreed with European Investment Bank

Nov 05, 2016: Abeona Therapeutics Honored with Partner in Progress Award from DEBRA of America

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 Epidermolysis Bullosa, H1 2017

Number of Products under Development by Companies, H1 2017

Number of Products under Development by Companies, H1 2017 (Contd..1), H1 2017

Number of Products under Development by Universities/Institutes, H1 2017

Products under Development by Companies, H1 2017

Products under Development by Companies, H1 2017 (Contd..1), H1 2017

Products under Development by Universities/Institutes, H1 2017

Number of Products by Stage and Target, H1 2017

Number of Products by Stage and Mechanism of Action, H1 2017

Number of Products by Stage and Route of Administration, H1 2017

Number of Products by Stage and Molecule Type, H1 2017

Epidermolysis Bullosa – Pipeline by Abeona Therapeutics Inc, H1 2017

Epidermolysis Bullosa – Pipeline by Almirall SA, H1 2017

Epidermolysis Bullosa – Pipeline by Amryt Pharma plc, H1 2017

Epidermolysis Bullosa – Pipeline by Anterogen Co Ltd, H1 2017

Epidermolysis Bullosa – Pipeline by Berg LLC, H1 2017

Epidermolysis Bullosa – Pipeline by CSL Ltd, H1 2017

Epidermolysis Bullosa – Pipeline by Exicure Inc, H1 2017

Epidermolysis Bullosa – Pipeline by Fibrocell Science Inc, H1 2017

Epidermolysis Bullosa – Pipeline by GlaxoSmithKline Plc, H1 2017

Epidermolysis Bullosa – Pipeline by Immusoft Corp, H1 2017

Epidermolysis Bullosa – Pipeline by InMed Pharmaceuticals Inc, H1 2017

Epidermolysis Bullosa – Pipeline by Karus Therapeutics Ltd, H1 2017

Epidermolysis Bullosa – Pipeline by Mesoblast Ltd, H1 2017

Epidermolysis Bullosa – Pipeline by ProQR Therapeutics NV, H1 2017

Epidermolysis Bullosa – Pipeline by RegeneRx Biopharmaceuticals Inc, H1 2017

Epidermolysis Bullosa – Pipeline by Scioderm Inc, H1 2017

Epidermolysis Bullosa – Pipeline by Stratatech Corp, H1 2017

Epidermolysis Bullosa – Pipeline by TransDerm Inc, H1 2017

Epidermolysis Bullosa – Pipeline by WAVE Life Sciences Ltd, H1 2017

Epidermolysis Bullosa – Dormant Projects, H1 2017

Epidermolysis Bullosa – Discontinued Products, H1 2017

List of Figures

List of Figures

Number of Products under Development for Epidermolysis Bullosa, H1 2017

Number of Products under Development by Companies, H1 2017

Number of Products under Development by Universities/Institutes, H1 2017

Number of Products by Targets, H1 2017

Number of Products by Stage and Targets, H1 2017

Number of Products by Mechanism of Actions, H1 2017

Number of Products by Stage and Mechanism of Actions, H1 2017

Number of Products by Routes of Administration, H1 2017

Number of Products by Stage and Top 10 Routes of Administration, H1 2017

Number of Products by Molecule Types, H1 2017

Number of Products by Stage and Top 10 Molecule Types, H1 2017

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