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Published: Mar, 2015 | Pages:
81 | Publisher: Global Markets Direct
Industry: Healthcare | Report Format: Electronic (PDF)
Alpha- Antitrypsin Deficiency - Pipeline Review, H1 2015 Summary Global Markets Direct's, 'Alpha- Antitrypsin Deficiency - Pipeline Review, H1 2015', provides an overview of the Alpha- Antitrypsin Deficiency's therapeutic pipeline. This report provides comprehensive information on the therapeutic development for Alpha- Antitrypsin Deficiency, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Alpha- Antitrypsin Deficiency and special features on late-stage 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, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct's team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis. The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products. Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data for the indicated disease. Scope - The report provides a snapshot of the global therapeutic landscape of AlphaAntitrypsin Deficiency - The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities - The report reviews key players involved in the therapeutics development for AlphaAntitrypsin Deficiency and enlists all their major and minor projects - The report summarizes all the dormant and discontinued pipeline projects - A review of the AlphaAntitrypsin Deficiency products under development by companies and universities/research institutes based on information derived from company and industry-specific sources - Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages - A detailed assessment of monotherapy and combination therapy pipeline projects - Coverage of the AlphaAntitrypsin Deficiency pipeline on the basis of target, MoA, route of administration and molecule type - Latest news and deals relating related to pipeline products Reasons to buy - Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies - Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage - Develop strategic initiatives by understanding the focus areas of leading companies - Identify and understand important and diverse types of therapeutics under development for AlphaAntitrypsin Deficiency - Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline - Devise corrective measures for pipeline projects by understanding AlphaAntitrypsin Deficiency 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
Table of Contents Table of Contents 2 List of table 5 List of Figures 6 Introduction 7 Global Markets Direct Report Coverage 7 Alpha- Antitrypsin Deficiency Overview 8 Therapeutics Development 9 Pipeline Products for Alpha- Antitrypsin Deficiency - Overview 9 Pipeline Products for Alpha- Antitrypsin Deficiency - Comparative Analysis 10 Alpha- Antitrypsin Deficiency - Therapeutics under Development by Companies 11 Alpha- Antitrypsin Deficiency - Therapeutics under Investigation by Universities/Institutes 13 Alpha- Antitrypsin Deficiency - Pipeline Products Glance 14 Late Stage Products 14 Clinical Stage Products 15 Early Stage Products 16 Alpha- Antitrypsin Deficiency - Products under Development by Companies 17 Alpha- Antitrypsin Deficiency - Products under Investigation by Universities/Institutes 19 Alpha- Antitrypsin Deficiency - Companies Involved in Therapeutics Development 20 Applied Genetic Technologies Corporation 20 Arrowhead Research Corporation 21 Carolus Therapeutics, Inc. 22 Cevec Pharmaceuticals GmbH 23 Dicerna Pharmaceuticals, Inc. 24 Grifols, S.A. 25 iBio, Inc. 26 Inhibrx 27 International Stem Cell Corporation 28 Intrexon Corporation 29 Kamada Ltd. 30 Polyphor Ltd. 31 Promethera Biosciences S.A. 32 Sangamo BioSciences, Inc. 33 Alpha- Antitrypsin Deficiency - Therapeutics Assessment 34 Assessment by Monotherapy Products 34 Assessment by Target 35 Assessment by Mechanism of Action 37 Assessment by Route of Administration 39 Assessment by Molecule Type 41 Drug Profiles 43 Alpha-1 Antitrypsin With rHuPH20 - Drug Profile 43 Product Description 43 Mechanism of Action 43 R&D Progress 43 alpha-1 proteinase inhibitor (human) second generation - Drug Profile 44 Product Description 44 Mechanism of Action 44 R&D Progress 44 ARC-AAT - Drug Profile 47 Product Description 47 Mechanism of Action 47 R&D Progress 47 CT-2009 - Drug Profile 48 Product Description 48 Mechanism of Action 48 R&D Progress 48 DC-AIM - Drug Profile 49 Product Description 49 Mechanism of Action 49 R&D Progress 49 Gene Therapy to Activate A1AT for Alpha 1-Antitrypsin Deficiency - Drug Profile 50 Product Description 50 Mechanism of Action 50 R&D Progress 50 Gene Therapy to Activate Alpha-1 Antitrypsin - Drug Profile 51 Product Description 51 Mechanism of Action 51 R&D Progress 51 HepaStem - Drug Profile 52 Product Description 52 Mechanism of Action 52 R&D Progress 52 POL-6014 - Drug Profile 54 Product Description 54 Mechanism of Action 54 R&D Progress 54 rAAV1-CB-hAAT - Drug Profile 56 Product Description 56 Mechanism of Action 56 R&D Progress 56 rAAV2-CB-hAAT - Drug Profile 57 Product Description 57 Mechanism of Action 57 R&D Progress 57 Recombinant A1PI - Drug Profile 58 Product Description 58 Mechanism of Action 58 R&D Progress 58 Recombinant Human Alpha-1 Antitrypsin - Drug Profile 60 Product Description 60 Mechanism of Action 60 R&D Progress 60 Recombinant Protein to Replace Alpha-1 Antitrypsin for Alpha Antitrypsin Deficiency - Drug Profile 61 Product Description 61 Mechanism of Action 61 R&D Progress 61 Recombinant Protein to Replace Alpha-1 Antitrypsin for Alpha-Antitrypsin Deficiency - Drug Profile 62 Product Description 62 Mechanism of Action 62 R&D Progress 62 RNAi Oligonucleotide for Alpha- Antitrypsin Deficiency - Drug Profile 63 Product Description 63 Mechanism of Action 63 R&D Progress 63 Stem Cell Therapy for Alpha-Antitrypsin Deficiency and Crigler-Najjar Syndrome - Drug Profile 65 Product Description 65 Mechanism of Action 65 R&D Progress 65 Alpha- Antitrypsin Deficiency - Recent Pipeline Updates 66 Alpha- Antitrypsin Deficiency - Dormant Projects 70 Alpha- Antitrypsin Deficiency - Discontinued Products 71 Alpha- Antitrypsin Deficiency - Product Development Milestones 72 Featured News & Press Releases 72 Jan 07, 2015: Kamada Reports Encouraging Discussions with European Co-Rapporteurs Regarding European Filing for Inhaled Alpha-1 Antitrypsin to Treat AATD 72 Nov 18, 2014: Arrowhead Files For Regulatory Permission To Begin Phase 1 Trial Of RNAi Therapeutic ARC-AAT 72 Nov 10, 2014: Arrowhead Presents Data on ARC-AAT at AASLD The Liver Meeting 2014 73 Sep 04, 2014: Kamada Reports Final Results from Phase 2/3 Clinical Trial of Inhaled Alpha-1 Antitrypsin to Treat Alpha-1 Antitrypsin Deficiency 74 Sep 03, 2014: Kamada to Announce Final Results from Phase 2/3 Clinical Trial of Inhaled AAT to Treat Alpha-1 Antitrypsin Deficiency on September 4, 2014 74 Jun 19, 2014: Arrowhead Announces New Clinical Candidate ARC-AAT for Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease 75 May 16, 2014: Kamada Announces Preliminary Results from Phase II/III Pivotal Trial in Europe and Canada of Inhaled AAT to Treat Alpha-1 Antitrypsin Deficiency 76 May 15, 2014: Kamada to Announce Results from Phase 2/3 Clinical Trial of Inhaled AAT to Treat Alpha-1 Antitrypsin Deficiency on May 16 77 Mar 24, 2014: Kamada Announces Initiation of a Phase 2 U.S. Clinical Trial of Inhaled AAT to Treat Alpha-1 Antitrypsin Deficiency 78 Mar 21, 2014: Kamada to Report Top-Line Data from Phase 2/3 Clinical Trial of Inhaled AAT to Treat Alpha-1 Antitrypsin Deficiency by Late April or Early May 78 Appendix 80 Methodology 80 Coverage 80 Secondary Research 80 Primary Research 80 Expert Panel Validation 80 Contact Us 80 Disclaimer 81
List of Tables Number of Products under Development for Alpha- Antitrypsin Deficiency, H1 2015 9 Number of Products under Development for Alpha- Antitrypsin Deficiency - Comparative Analysis, H1 2015 10 Number of Products under Development by Companies, H1 2015 12 Number of Products under Investigation by Universities/Institutes, H1 2015 13 Comparative Analysis by Late Stage Development, H1 2015 14 Comparative Analysis by Clinical Stage Development, H1 2015 15 Comparative Analysis by Early Stage Development, H1 2015 16 Products under Development by Companies, H1 2015 17 Products under Development by Companies, H1 2015 (Contd..1) 18 Products under Investigation by Universities/Institutes, H1 2015 19 Alpha- Antitrypsin Deficiency - Pipeline by Applied Genetic Technologies Corporation, H1 2015 20 Alpha- Antitrypsin Deficiency - Pipeline by Arrowhead Research Corporation, H1 2015 21 Alpha- Antitrypsin Deficiency - Pipeline by Carolus Therapeutics, Inc., H1 2015 22 Alpha- Antitrypsin Deficiency - Pipeline by Cevec Pharmaceuticals GmbH, H1 2015 23 Alpha- Antitrypsin Deficiency - Pipeline by Dicerna Pharmaceuticals, Inc., H1 2015 24 Alpha- Antitrypsin Deficiency - Pipeline by Grifols, S.A., H1 2015 25 Alpha- Antitrypsin Deficiency - Pipeline by iBio, Inc., H1 2015 26 Alpha- Antitrypsin Deficiency - Pipeline by Inhibrx, H1 2015 27 Alpha- Antitrypsin Deficiency - Pipeline by International Stem Cell Corporation, H1 2015 28 Alpha- Antitrypsin Deficiency - Pipeline by Intrexon Corporation, H1 2015 29 Alpha- Antitrypsin Deficiency - Pipeline by Kamada Ltd., H1 2015 30 Alpha- Antitrypsin Deficiency - Pipeline by Polyphor Ltd., H1 2015 31 Alpha- Antitrypsin Deficiency - Pipeline by Promethera Biosciences S.A., H1 2015 32 Alpha- Antitrypsin Deficiency - Pipeline by Sangamo BioSciences, Inc., H1 2015 33 Assessment by Monotherapy Products, H1 2015 34 Number of Products by Stage and Target, H1 2015 36 Number of Products by Stage and Mechanism of Action, H1 2015 38 Number of Products by Stage and Route of Administration, H1 2015 40 Number of Products by Stage and Molecule Type, H1 2015 42 Alpha- Antitrypsin Deficiency Therapeutics - Recent Pipeline Updates, H1 2015 66 Alpha- Antitrypsin Deficiency - Dormant Projects, H1 2015 70 Alpha- Antitrypsin Deficiency - Discontinued Products, H1 2015 71
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