Epidermolysis Bullosa – Pipeline Review, H1 2018

Global Markets Direct’s, ‘Epidermolysis Bullosa – Pipeline Review, H1 2018’, 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

Berg LLC

CSL Ltd

Exicure Inc

Fibrocell Science Inc

GlaxoSmithKline Plc

Immusoft Corp

InMed Pharmaceuticals Inc

MallInckrodt Plc

Mesoblast Ltd

ProQR Therapeutics NV

RegeneRx Biopharmaceuticals Inc

Shionogi & Co Ltd

TransDerm Inc

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

Berg LLC

CSL Ltd

Exicure Inc

Fibrocell Science Inc

GlaxoSmithKline Plc

Immusoft Corp

InMed Pharmaceuticals Inc

MallInckrodt Plc

Mesoblast Ltd

ProQR Therapeutics NV

RegeneRx Biopharmaceuticals Inc

Shionogi & Co Ltd

TransDerm Inc

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

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

Product Description

Mechanism Of Action

R&D Progress

AP-103 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

AZT-03 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

CL-2020 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

CSL-730 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

diacerein 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 to Activate COL7A1 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

HOLOGENE-17 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

ISN-001 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

KB-103 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Protein to Target COL7A1 for Epidermolysis Bullosa Drug Profile

Product Description

Mechanism Of Action

R&D Progress

QRX-313 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

QRX-323 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

QRX-333 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

S-005151 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

sirolimus Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Stem Cell Therapy for Diabetic Foot Ulcers, Epidermolysis Bullosa, Liver Diseases, Kidney Disease, Peripheral Artery Occlusive Disease and Skin Ulcers Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Stem Cell Therapy for Recessive Dystrophic Epidermolysis Bullosa 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

Apr 19, 2018: Krystal Biotech's KB103 Receives Orphan Medicinal Product Designation in Europe for Dystrophic Epidermolysis Bullosa

Apr 18, 2018: Krystal Biotech Presents Their Skin TARgeted Delivery Platform at the World Orphan Drug Congress USA

Mar 27, 2018: Krystal Submits Investigational New Drug Application for KB103, Topical Gene Therapy Candidate for Dystrophic Epidermolysis Bullosa

Feb 26, 2018: TWi Biotechnology Announced Regulatory Clearance to Begin Phase 2 Proof-of-Concept Clinical trial of AC-203 for treatment of Inherited Epidermolysis Bullosa

Feb 14, 2018: RegeneRx Provides Update on RGN-137

Jan 30, 2018: Fibrocell Announces FDA Allowance to Initiate Pediatric Enrollment in Phase 1/2 Clinical Trial of FCX-007 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Jan 29, 2018: Abeona Receives FDA Regenerative Medicine Advanced Therapy Designation for EB-101 Gene Therapy in Epidermolysis Bullosa

Jan 24, 2018: Fibrocell to Present at Phacilitate Cell and Gene Therapy World 2018

Jan 16, 2018: Krystal Biotech Announces Granting of U.S. Composition of Matter Patent Covering Herpes Simplex Virus (HSV) Vectors and Methods of Using the Same to Treat Skin Diseases

Jan 15, 2018: Castle Creek Pharmaceuticals Announces Data Validating First Investigator's Global Assessment Scale for Epidermolysis Bullosa Simplex Clinical Research

Jan 03, 2018: Castle Creek Pharmaceuticals to Participate in 5th Annual Dermatology Summit

Nov 29, 2017: ProQR Receives Orphan Drug Designation from EMA for Drug Candidate QR-313 for Dystrophic Epidermolysis Bullosa

Nov 13, 2017: Krystal Biotech Reports that KB103 Received Clearance from RAC Review

Nov 07, 2017: Krystal Biotech's KB103 Granted Orphan Drug Designation by the FDA to Treat Patients With Dystrophic Epidermolysis Bullosa

Oct 27, 2017: Castle Creek Pharmaceuticals Receives Partners 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

Table 1: Number of Products under Development for Epidermolysis Bullosa, H1 2018

Table 2: Number of Products under Development by Companies, H1 2018

Table 3: Number of Products under Development by Companies, H1 2018 (Contd..1), H1 2018

Table 4: Number of Products under Development by Universities/Institutes, H1 2018

Table 5: Products under Development by Companies, H1 2018

Table 6: Products under Development by Companies, H1 2018 (Contd..1), H1 2018

Table 7: Products under Development by Companies, H1 2018 (Contd..2), H1 2018

Table 8: Products under Development by Universities/Institutes, H1 2018

Table 9: Number of Products by Stage and Target, H1 2018

Table 10: Number of Products by Stage and Mechanism of Action, H1 2018

Table 11: Number of Products by Stage and Route of Administration, H1 2018

Table 12: Number of Products by Stage and Molecule Type, H1 2018

Table 13: Epidermolysis Bullosa Pipeline by Abeona Therapeutics Inc, H1 2018

Table 14: Epidermolysis Bullosa Pipeline by Almirall SA, H1 2018

Table 15: Epidermolysis Bullosa Pipeline by Amryt Pharma plc, H1 2018

Table 16: Epidermolysis Bullosa Pipeline by Berg LLC, H1 2018

Table 17: Epidermolysis Bullosa Pipeline by CSL Ltd, H1 2018

Table 18: Epidermolysis Bullosa Pipeline by Exicure Inc, H1 2018

Table 19: Epidermolysis Bullosa Pipeline by Fibrocell Science Inc, H1 2018

Table 20: Epidermolysis Bullosa Pipeline by GlaxoSmithKline Plc, H1 2018

Table 21: Epidermolysis Bullosa Pipeline by Immusoft Corp, H1 2018

Table 22: Epidermolysis Bullosa Pipeline by InMed Pharmaceuticals Inc, H1 2018

Table 23: Epidermolysis Bullosa Pipeline by MallInckrodt Plc, H1 2018

Table 24: Epidermolysis Bullosa Pipeline by Mesoblast Ltd, H1 2018

Table 25: Epidermolysis Bullosa Pipeline by ProQR Therapeutics NV, H1 2018

Table 26: Epidermolysis Bullosa Pipeline by RegeneRx Biopharmaceuticals Inc, H1 2018

Table 27: Epidermolysis Bullosa Pipeline by Shionogi & Co Ltd, H1 2018

Table 28: Epidermolysis Bullosa Pipeline by TransDerm Inc, H1 2018

Table 29: Epidermolysis Bullosa Dormant Projects, H1 2018

Table 30: Epidermolysis Bullosa Discontinued Products, H1 2018

List of Figures

List of Figures

Figure 1: Number of Products under Development for Epidermolysis Bullosa, H1 2018

Figure 2: Number of Products under Development by Companies, H1 2018

Figure 3: Number of Products under Development by Universities/Institutes, H1 2018

Figure 4: Number of Products by Top 10 Targets, H1 2018

Figure 5: Number of Products by Stage and Top 10 Targets, H1 2018

Figure 6: Number of Products by Top 10 Mechanism of Actions, H1 2018

Figure 7: Number of Products by Stage and Top 10 Mechanism of Actions, H1 2018

Figure 8: Number of Products by Routes of Administration, H1 2018

Figure 9: Number of Products by Stage and Routes of Administration, H1 2018

Figure 10: Number of Products by Top 10 Molecule Types, H1 2018

Figure 11: Number of Products by Stage and Top 10 Molecule Types, H1 2018

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