Epidermolysis Bullosa – Pipeline Review, H2 2017

Global Markets Direct’s, ‘Epidermolysis Bullosa – Pipeline Review, H2 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

Mesoblast Ltd

ProQR Therapeutics NV

RegeneRx Biopharmaceuticals Inc

Stratatech Corp

TransDerm Inc

WAVE Life Sciences Ltd

Table of Contents

Table of Contents

Table of Contents 2

List of Tables 4

List of Figures 4

Introduction 5

Global Markets Direct Report Coverage 5

Epidermolysis Bullosa - Overview 6

Epidermolysis Bullosa - Therapeutics Development 7

Pipeline Overview 7

Pipeline by Companies 8

Pipeline by Universities/Institutes 11

Products under Development by Companies 12

Products under Development by Universities/Institutes 14

Epidermolysis Bullosa - Therapeutics Assessment 15

Assessment by Target 15

Assessment by Mechanism of Action 17

Assessment by Route of Administration 19

Assessment by Molecule Type 21

Epidermolysis Bullosa - Companies Involved in Therapeutics Development 23

Abeona Therapeutics Inc 23

Almirall SA 23

Amryt Pharma plc 24

Anterogen Co Ltd 24

Berg LLC 25

CSL Ltd 25

Exicure Inc 25

Fibrocell Science Inc 26

GlaxoSmithKline Plc 26

Immusoft Corp 27

InMed Pharmaceuticals Inc 27

Mesoblast Ltd 28

ProQR Therapeutics NV 28

RegeneRx Biopharmaceuticals Inc 29

Stratatech Corp 29

TransDerm Inc 29

WAVE Life Sciences Ltd 30

Epidermolysis Bullosa - Drug Profiles 31

AC-203 - Drug Profile 31

ADP-31415 - Drug Profile 33

Antisense Oligonucleotide 1 to Inhibit Keratin for Epidermolysis Bullosa Simplex - Drug Profile 34

Antisense Oligonucleotide 2 to Inhibit Keratin for Epidermolysis Bullosa Simplex - Drug Profile 35

Antisense Oligonucleotide to Inhibit IL-1 Beta for Epidermolysis Bullosa - Drug Profile 36

Biologic for Epidermolysis Bullosa - Drug Profile 37

birch bark extract - Drug Profile 38

Cell Therapy for Dermatology and Immunology - Drug Profile 41

Cell Therapy for Epidermolysis Bullosa - Drug Profile 43

diacerein - Drug Profile 44

EB-101 - Drug Profile 46

EB-201 - Drug Profile 49

FCX-007 - Drug Profile 50

Gene Therapy for Epidermolysis Bullosa - Drug Profile 54

Gene Therapy to Activate COL7A1 for Epidermolysis Bullosa - Drug Profile 55

Gene Therapy to Activate Lysyl Hydroxylase-3 for Recessive Dystrophic Epidermolysis Bullosa - Drug Profile 56

HOLOGENE-17 - Drug Profile 57

ICX-RHY - Drug Profile 58

INM-750 - Drug Profile 60

ISN-001 - Drug Profile 62

KB-103 - Drug Profile 64

M-230 - Drug Profile 65

Protein to Target COL7A1 for Epidermolysis Bullosa - Drug Profile 66

QRX-313 - Drug Profile 67

QRX-323 - Drug Profile 68

remestemcel-L - Drug Profile 69

RGN-137 - Drug Profile 75

sirolimus - Drug Profile 78

Stem Cell Therapy for Recessive Dystrophic Epidermolysis Bullosa - Drug Profile 80

TXA-127 - Drug Profile 81

ubidecarenone - Drug Profile 85

Epidermolysis Bullosa - Dormant Projects 88

Epidermolysis Bullosa - Discontinued Products 89

Epidermolysis Bullosa - Product Development Milestones 90

Featured News & Press Releases 90

Appendix 102

Methodology 102

Coverage 102

Secondary Research 102

Primary Research 102

Expert Panel Validation 102

Contact Us 102

Disclaimer 103

List of Tables

List of Tables

Number of Products under Development for Epidermolysis Bullosa, H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products under Development by Companies, H2 2017 (Contd..1), H2 2017

Number of Products under Development by Universities/Institutes, H2 2017

Products under Development by Companies, H2 2017

Products under Development by Companies, H2 2017 (Contd..1), H2 2017

Products under Development by Universities/Institutes, H2 2017

Number of Products by Stage and Target, H2 2017

Number of Products by Stage and Mechanism of Action, H2 2017

Number of Products by Stage and Route of Administration, H2 2017

Number of Products by Stage and Molecule Type, H2 2017

Epidermolysis Bullosa – Pipeline by Abeona Therapeutics Inc, H2 2017

Epidermolysis Bullosa – Pipeline by Almirall SA, H2 2017

Epidermolysis Bullosa – Pipeline by Amryt Pharma plc, H2 2017

Epidermolysis Bullosa – Pipeline by Anterogen Co Ltd, H2 2017

Epidermolysis Bullosa – Pipeline by Berg LLC, H2 2017

Epidermolysis Bullosa – Pipeline by CSL Ltd, H2 2017

Epidermolysis Bullosa – Pipeline by Exicure Inc, H2 2017

Epidermolysis Bullosa – Pipeline by Fibrocell Science Inc, H2 2017

Epidermolysis Bullosa – Pipeline by GlaxoSmithKline Plc, H2 2017

Epidermolysis Bullosa – Pipeline by Immusoft Corp, H2 2017

Epidermolysis Bullosa – Pipeline by InMed Pharmaceuticals Inc, H2 2017

Epidermolysis Bullosa – Pipeline by Mesoblast Ltd, H2 2017

Epidermolysis Bullosa – Pipeline by ProQR Therapeutics NV, H2 2017

Epidermolysis Bullosa – Pipeline by RegeneRx Biopharmaceuticals Inc, H2 2017

Epidermolysis Bullosa – Pipeline by Stratatech Corp, H2 2017

Epidermolysis Bullosa – Pipeline by TransDerm Inc, H2 2017

Epidermolysis Bullosa – Pipeline by WAVE Life Sciences Ltd, H2 2017

Epidermolysis Bullosa – Dormant Projects, H2 2017

Epidermolysis Bullosa – Discontinued Products, H2 2017

List of Figures

List of Figures

Number of Products under Development for Epidermolysis Bullosa, H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products under Development by Universities/Institutes, H2 2017

Number of Products by Targets, H2 2017

Number of Products by Stage and Targets, H2 2017

Number of Products by Mechanism of Actions, H2 2017

Number of Products by Stage and Mechanism of Actions, H2 2017

Number of Products by Routes of Administration, H2 2017

Number of Products by Stage and Top 10 Routes of Administration, H2 2017

Number of Products by Molecule Types, H2 2017

Number of Products by Stage and Molecule Types, H2 2017

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