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Published: Oct, 2016 | Pages:
90 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)
Epidermolysis Bullosa - Pipeline Review, H2 2016 Summary Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Epidermolysis Bullosa - Pipeline Review, H2 2016, provides an overview of the Epidermolysis Bullosa (Dermatology) pipeline landscape. Epidermolysis bullosa (EB) is a group of inherited bullous disorders characterized by blister formation in response to mechanical trauma. Symptoms include blistering of skin, deformity or loss of fingernails and toenails, internal blistering, including on the throat, esophagus, upper airway, stomach, intestines and urinary tract, skin thickening on palms and soles of the feet (hyperkeratosis) and scalp blistering, scarring and hair loss (scarring alopecia). Treatment includes antibiotics, antibiotic and surgery. Report Highlights Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Epidermolysis Bullosa - Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Epidermolysis Bullosa (Dermatology), 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 Epidermolysis Bullosa (Dermatology) pipeline guide also reviews of key players involved in therapeutic development for Epidermolysis Bullosa and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Phase II, Phase I, Preclinical and Discovery stages are 1, 7, 1, 8 and 4 respectively for Similarly, the Universities portfolio in Phase I and Preclinical stages comprises 1 and 1 molecules, respectively for Epidermolysis Bullosa. Epidermolysis Bullosa (Dermatology) 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 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. 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 Epidermolysis Bullosa (Dermatology). - The pipeline guide reviews pipeline therapeutics for Epidermolysis Bullosa (Dermatology) 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 Epidermolysis Bullosa (Dermatology) therapeutics and enlists all their major and minor projects. - The pipeline guide evaluates Epidermolysis Bullosa (Dermatology) 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 Epidermolysis Bullosa (Dermatology) Reasons To Buy - 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 Epidermolysis Bullosa (Dermatology). - 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 Epidermolysis Bullosa (Dermatology) 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.
Table of Contents
Table of Contents 2 List of Tables 5 List of Figures 6 Introduction 7 Global Markets Direct Report Coverage 7 Epidermolysis Bullosa Overview 8 Therapeutics Development 9 Pipeline Products for Epidermolysis Bullosa - Overview 9 Pipeline Products for Epidermolysis Bullosa - Comparative Analysis 10 Epidermolysis Bullosa - Therapeutics under Development by Companies 11 Epidermolysis Bullosa - Therapeutics under Investigation by Universities/Institutes 13 Epidermolysis Bullosa - Pipeline Products Glance 14 Late Stage Products 14 Clinical Stage Products 15 Early Stage Products 16 Epidermolysis Bullosa - Products under Development by Companies 17 Epidermolysis Bullosa - Products under Investigation by Universities/Institutes 18 Epidermolysis Bullosa - Companies Involved in Therapeutics Development 19 Abeona Therapeutics, Inc. 19 Amryt Pharma plc 20 Berg LLC 21 Fibrocell Science, Inc. 22 GlaxoSmithKline Plc 23 InMed Pharmaceuticals Inc. 24 Karus Therapeutics Limited 25 ProQR Therapeutics N.V. 26 RegeneRx Biopharmaceuticals, Inc. 27 Scioderm, Inc. 28 Stratatech Corp 29 TWi Pharmaceuticals, Inc. 30 WAVE Life Sciences Ltd. 31 Epidermolysis Bullosa - Therapeutics Assessment 32 Assessment by Monotherapy Products 32 Assessment by Target 33 Assessment by Mechanism of Action 35 Assessment by Route of Administration 37 Assessment by Molecule Type 39 Drug Profiles 41 allantoin - Drug Profile 41 Product Description 41 Mechanism Of Action 41 R&D Progress 41 ALLO-ASC - Drug Profile 44 Product Description 44 Mechanism Of Action 44 R&D Progress 44 Antisense Oligonucleotide 1 to Inhibit Keratin for Epidermolysis Bullosa Simplex - Drug Profile 45 Product Description 45 Mechanism Of Action 45 R&D Progress 45 Antisense Oligonucleotide 2 to Inhibit Keratin for Epidermolysis Bullosa Simplex - Drug Profile 46 Product Description 46 Mechanism Of Action 46 R&D Progress 46 Biologic for Epidermolysis Bullosa - Drug Profile 47 Product Description 47 Mechanism Of Action 47 R&D Progress 47 birch bark extract - Drug Profile 48 Product Description 48 Mechanism Of Action 48 R&D Progress 48 Cell Therapy for Dermatology and Immunology - Drug Profile 50 Product Description 50 Mechanism Of Action 50 R&D Progress 50 Cell Therapy for Epidermolysis Bullosa - Drug Profile 52 Product Description 52 Mechanism Of Action 52 R&D Progress 52 Cell Therapy for Recessive Dystrophic Epidermolysis Bullosa - Drug Profile 53 Product Description 53 Mechanism Of Action 53 R&D Progress 53 diacerein - Drug Profile 54 Product Description 54 Mechanism Of Action 54 R&D Progress 54 EB-101 - Drug Profile 57 Product Description 57 Mechanism Of Action 57 R&D Progress 57 EB-201 - Drug Profile 58 Product Description 58 Mechanism Of Action 58 R&D Progress 58 FCX-007 - Drug Profile 59 Product Description 59 Mechanism Of Action 59 R&D Progress 59 Gene Therapy for Epidermolysis Bullosa - Drug Profile 62 Product Description 62 Mechanism Of Action 62 R&D Progress 62 Gene Therapy for Epidermolysis Bullosa - Drug Profile 63 Product Description 63 Mechanism Of Action 63 R&D Progress 63 Gene Therapy to Activate Lysyl Hydroxylase-3 for Recessive Dystrophic Epidermolysis Bullosa - Drug Profile 64 Product Description 64 Mechanism Of Action 64 R&D Progress 64 ICX-RHY - Drug Profile 65 Product Description 65 Mechanism Of Action 65 R&D Progress 65 INM-750 - Drug Profile 67 Product Description 67 Mechanism Of Action 67 R&D Progress 67 KA-1463 - Drug Profile 68 Product Description 68 Mechanism Of Action 68 R&D Progress 68 QR-313 - Drug Profile 69 Product Description 69 Mechanism Of Action 69 R&D Progress 69 RGN-137 - Drug Profile 70 Product Description 70 Mechanism Of Action 70 R&D Progress 70 TXA-127 - Drug Profile 72 Product Description 72 Mechanism Of Action 72 R&D Progress 72 ubidecarenone - Drug Profile 75 Product Description 75 Mechanism Of Action 75 R&D Progress 75 Epidermolysis Bullosa - Dormant Projects 77 Epidermolysis Bullosa - Discontinued Products 78 Epidermolysis Bullosa - Product Development Milestones 79 Featured News & Press Releases 79 Oct 01, 2016: Castle Creek Pharmaceuticals Announces Late-Breaking Results at EADV of Phase 2 Topical Diacerein 1% Data in Patients with Epidermolysis Bullosa Simplex 79 Sep 26, 2016: Abeona Therapeutics Enrolls First Patient in Phase 2 for EB-101 Gene Therapy Clinical Trial for Epidermolysis Bullosa 80 Sep 19, 2016: Fibrocell Completes Enrollment in NC1+ Cohort of Phase I/II Clinical Trial of FCX-007 for Treatment of Recessive Dystrophic Epidermolysis Bullosa 80 Sep 13, 2016: Amryt Pharma announces US patent for Lead Drug, Episalvan 81 Sep 08, 2016: Abeona Therapeutics Enrolls 5th Patient in Phase 1/2 Gene Therapy Clinical Trial for Epidermolysis Bullosa 81 Sep 07, 2016: Tarix Orphan Receives Orphan Drug Status for TXA127 for Genetic Skin Disorder Recessive Dystrophic Epidermolysis Bullosa 82 Jul 27, 2016: Fibrocell Announces First Subjects Enrolled in Phase I/II Clinical Trial of FCX-007 for Treatment of Recessive Dystrophic Epidermolysis Bullosa 83 Jun 29, 2016: Fibrocell Announces Update on FCX-007 83 Jun 27, 2016: Tarix Orphan Announces Positive Results with TXA127 in Animal Model of Dystrophic Epidermolysis Bullosa 83 Jun 13, 2016: Fibrocell Announces Initiation of Patient Recruitment in Phase I/II Clinical Trial of FCX-007 for Treatment of Recessive Dystrophic Epidermolysis Bullosa 84 Apr 18, 2016: Fibrocell and Intrexon Announce Allowance to Commence Phase I/II Trial for FCX-007 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa 85 Dec 04, 2015: RegeneRx Receives Chinese Patent for Key TB4 Peptide Fragment 85 Oct 08, 2015: Fibrocell and Intrexon Announce Poster Presentation on FCX-007 for the Treatment of RDEB at American Society of Human Genetics Annual Meeting 86 Sep 25, 2015: Fibrocell and Intrexon Provide Regulatory Update for FCX-007 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa 86 Jul 22, 2015: RegeneRx Receives Notice of Acceptance for Australian Patent for Key TB4 Peptide Fragment 87 Appendix 88 Methodology 88 Coverage 88 Secondary Research 88 Primary Research 88 Expert Panel Validation 88 Contact Us 88 Disclaimer 89
List of Tables
Number of Products under Development for Epidermolysis Bullosa, H2 2016 9 Number of Products under Development for Epidermolysis Bullosa - Comparative Analysis, H2 2016 10 Number of Products under Development by Companies, H2 2016 12 Number of Products under Investigation by Universities/Institutes, H2 2016 13 Comparative Analysis by Late Stage Development, H2 2016 14 Comparative Analysis by Clinical Stage Development, H2 2016 15 Comparative Analysis by Early Stage Development, H2 2016 16 Products under Development by Companies, H2 2016 17 Products under Investigation by Universities/Institutes, H2 2016 18 Epidermolysis Bullosa - Pipeline by Abeona Therapeutics, Inc. , H2 2016 19 Epidermolysis Bullosa - Pipeline by Amryt Pharma plc, H2 2016 20 Epidermolysis Bullosa - Pipeline by Berg LLC, H2 2016 21 Epidermolysis Bullosa - Pipeline by Fibrocell Science, Inc., H2 2016 22 Epidermolysis Bullosa - Pipeline by GlaxoSmithKline Plc, H2 2016 23 Epidermolysis Bullosa - Pipeline by InMed Pharmaceuticals Inc., H2 2016 24 Epidermolysis Bullosa - Pipeline by Karus Therapeutics Limited, H2 2016 25 Epidermolysis Bullosa - Pipeline by ProQR Therapeutics N.V., H2 2016 26 Epidermolysis Bullosa - Pipeline by RegeneRx Biopharmaceuticals, Inc., H2 2016 27 Epidermolysis Bullosa - Pipeline by Scioderm, Inc., H2 2016 28 Epidermolysis Bullosa - Pipeline by Stratatech Corp, H2 2016 29 Epidermolysis Bullosa - Pipeline by TWi Pharmaceuticals, Inc., H2 2016 30 Epidermolysis Bullosa - Pipeline by WAVE Life Sciences Ltd., H2 2016 31 Assessment by Monotherapy Products, H2 2016 32 Number of Products by Stage and Target, H2 2016 34 Number of Products by Stage and Mechanism of Action, H2 2016 36 Number of Products by Stage and Route of Administration, H2 2016 38 Number of Products by Stage and Molecule Type, H2 2016 40 Epidermolysis Bullosa - Dormant Projects, H2 2016 77 Epidermolysis Bullosa - Discontinued Products, H2 2016 78
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