AlphaAntitrypsin Deficiency Pipeline Review, H2 2015
Summary
Global Markets Directs, AlphaAntitrypsin Deficiency Pipeline Review, H2 2015, provides an overview of the AlphaAntitrypsin Deficiencys therapeutic pipeline.
This report provides comprehensive information on the therapeutic development for AlphaAntitrypsin 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 AlphaAntitrypsin Deficiency and special features on late-stage and discontinued projects.
Global Markets Directs 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 Directs 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 Directs 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
List of Tables 6
List of Figures 7
Introduction 8
Global Markets Direct Report Coverage 8
Alpha- Antitrypsin Deficiency Overview 9
Therapeutics Development 10
Pipeline Products for Alpha- Antitrypsin Deficiency - Overview 10
Pipeline Products for Alpha- Antitrypsin Deficiency - Comparative Analysis 11
Alpha- Antitrypsin Deficiency - Therapeutics under Development by Companies 12
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 18
Alpha- Antitrypsin Deficiency - Companies Involved in Therapeutics Development 19
Alnylam Pharmaceuticals, Inc. 19
Applied Genetic Technologies Corporation 20
Arrowhead Research Corporation 21
Carolus Therapeutics, Inc. 22
Cevec Pharmaceuticals GmbH 23
Grifols, S.A. 24
iBio, Inc. 25
Inhibrx 26
International Stem Cell Corporation 27
Intrexon Corporation 28
Isis Pharmaceuticals, Inc. 29
Kamada Ltd. 30
Polyphor Ltd. 31
Promethera Biosciences S.A. 32
rEVO Biologics, Inc. 33
Sangamo BioSciences, Inc. 34
Alpha- Antitrypsin Deficiency - Therapeutics Assessment 35
Assessment by Monotherapy Products 35
Assessment by Target 36
Assessment by Mechanism of Action 38
Assessment by Route of Administration 40
Assessment by Molecule Type 42
Drug Profiles 44
ALN-AAT - Drug Profile 44
Product Description 44
Mechanism of Action 44
R&D Progress 44
Alpha-1 Antitrypsin With rHuPH20 - Drug Profile 45
Product Description 45
Mechanism of Action 45
R&D Progress 45
alpha-1 proteinase inhibitor (human) second generation - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
ARC-AAT - Drug Profile 49
Product Description 49
Mechanism of Action 49
R&D Progress 49
CT-2009 - Drug Profile 50
Product Description 50
Mechanism of Action 50
R&D Progress 50
Gene Therapy to Activate A1AT for Alpha 1-Antitrypsin Deficiency - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
Gene Therapy to Activate Alpha-1 Antitrypsin for Alpha- Antitrypsin Deficiency - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
Gene Therapy to Activate Alpha-1 Antitrypsin for Alpha-Antitrypsin Deficiency - Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
HepaStem - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
ISIS-AATRx - Drug Profile 57
Product Description 57
Mechanism of Action 57
R&D Progress 57
POL-6014 - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
rAAV2-CB-hAAT - Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
Recombinant A1PI - Drug Profile 61
Product Description 61
Mechanism of Action 61
R&D Progress 61
Recombinant Human Alpha-1 Antitrypsin - Drug Profile 63
Product Description 63
Mechanism of Action 63
R&D Progress 63
Recombinant Human Alpha-1 Antitrypsin - Drug Profile 64
Product Description 64
Mechanism of Action 64
R&D Progress 64
Recombinant Protein for Alpha-1 Antitrypsin Deficiency - Drug Profile 65
Product Description 65
Mechanism of Action 65
R&D Progress 65
Recombinant Protein to Replace Alpha-1 Antitrypsin for Alpha-Antitrypsin Deficiency - Drug Profile 66
Product Description 66
Mechanism of Action 66
R&D Progress 66
Stem Cell Therapy for Alpha-Antitrypsin Deficiency and Crigler-Najjar Syndrome - Drug Profile 67
Product Description 67
Mechanism of Action 67
R&D Progress 67
Alpha- Antitrypsin Deficiency - Recent Pipeline Updates 68
Alpha- Antitrypsin Deficiency - Dormant Projects 75
Alpha- Antitrypsin Deficiency - Discontinued Products 76
Alpha- Antitrypsin Deficiency - Product Development Milestones 77
Featured News & Press Releases 77
Jul 08, 2015: Arrowhead Receives Regulatory Clearance for ARC-AAT Phase 1 in the United Kingdom and New Zealand 77
Jul 02, 2015: Arrowhead Initiates Dosing of ARC-AAT in Patients with Alpha-1 Antitrypsin Deficiency 77
Jun 10, 2015: Arrowhead Receives Orphan Drug Designation for ARC-AAT 78
May 20, 2015: Kamada Reports Updated Data from European Phase 2/3 Clinical Study of Inhaled Alpha-1 Antitrypsin to Treat AAT Deficiency 78
May 05, 2015: Arrowhead Completes Dosing Healthy Volunteers and Initiates Transition to Patients in Phase 1 Study of ARC-AAT 80
Apr 20, 2015: Kamada Reports Additional Data from European Phase 2/3 Clinical Study of Inhaled Alpha-1 Antitrypsin to Treat AAT Deficiency 81
Feb 23, 2015: Arrowhead Begins Phase 1 Trial of ARC-AAT for Treatment of Liver Disease Associated with Alpha-1 Antitrypsin Deficiency 84
Jan 07, 2015: Kamada Reports Encouraging Discussions with European Co-Rapporteurs Regarding European Filing for Inhaled Alpha-1 Antitrypsin to Treat AATD 85
Nov 18, 2014: Arrowhead Files For Regulatory Permission To Begin Phase 1 Trial Of RNAi Therapeutic ARC-AAT 86
Nov 10, 2014: Arrowhead Presents Data on ARC-AAT at AASLD The Liver Meeting 2014 86
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 Alpha- Antitrypsin Deficiency, H2 2015 10
Number of Products under Development for Alpha- Antitrypsin Deficiency - Comparative Analysis, H2 2015 11
Number of Products under Development by Companies, H2 2015 12
Number of Products under Investigation by Universities/Institutes, H2 2015 13
Comparative Analysis by Late Stage Development, H2 2015 14
Comparative Analysis by Clinical Stage Development, H2 2015 15
Comparative Analysis by Early Stage Development, H2 2015 16
Products under Development by Companies, H2 2015 17
Products under Investigation by Universities/Institutes, H2 2015 18
Alpha- Antitrypsin Deficiency - Pipeline by Alnylam Pharmaceuticals, Inc., H2 2015 19
Alpha- Antitrypsin Deficiency - Pipeline by Applied Genetic Technologies Corporation, H2 2015 20
Alpha- Antitrypsin Deficiency - Pipeline by Arrowhead Research Corporation, H2 2015 21
Alpha- Antitrypsin Deficiency - Pipeline by Carolus Therapeutics, Inc., H2 2015 22
Alpha- Antitrypsin Deficiency - Pipeline by Cevec Pharmaceuticals GmbH, H2 2015 23
Alpha- Antitrypsin Deficiency - Pipeline by Grifols, S.A., H2 2015 24
Alpha- Antitrypsin Deficiency - Pipeline by iBio, Inc., H2 2015 25
Alpha- Antitrypsin Deficiency - Pipeline by Inhibrx, H2 2015 26
Alpha- Antitrypsin Deficiency - Pipeline by International Stem Cell Corporation, H2 2015 27
Alpha- Antitrypsin Deficiency - Pipeline by Intrexon Corporation, H2 2015 28
Alpha- Antitrypsin Deficiency - Pipeline by Isis Pharmaceuticals, Inc., H2 2015 29
Alpha- Antitrypsin Deficiency - Pipeline by Kamada Ltd., H2 2015 30
Alpha- Antitrypsin Deficiency - Pipeline by Polyphor Ltd., H2 2015 31
Alpha- Antitrypsin Deficiency - Pipeline by Promethera Biosciences S.A., H2 2015 32
Alpha- Antitrypsin Deficiency - Pipeline by rEVO Biologics, Inc., H2 2015 33
Alpha- Antitrypsin Deficiency - Pipeline by Sangamo BioSciences, Inc., H2 2015 34
Assessment by Monotherapy Products, H2 2015 35
Number of Products by Stage and Target, H2 2015 37
Number of Products by Stage and Mechanism of Action, H2 2015 39
Number of Products by Stage and Route of Administration, H2 2015 41
Number of Products by Stage and Molecule Type, H2 2015 43
Alpha- Antitrypsin Deficiency Therapeutics - Recent Pipeline Updates, H2 2015 68
Alpha- Antitrypsin Deficiency - Dormant Projects, H2 2015 75
Alpha- Antitrypsin Deficiency - Discontinued Products, H2 2015 76
List of Figures
Number of Products under Development for Alpha- Antitrypsin Deficiency, H2 2015 10
Number of Products under Development for Alpha- Antitrypsin Deficiency - Comparative Analysis, H2 2015 11
Number of Products under Development by Companies, H2 2015 12
Comparative Analysis by Clinical Stage Development, H2 2015 15
Comparative Analysis by Early Stage Products, H2 2015 16
Assessment by Monotherapy Products, H2 2015 35
Number of Products by Top 10 Targets, H2 2015 36
Number of Products by Stage and Top 10 Targets, H2 2015 36
Number of Products by Top 10 Mechanism of Actions, H2 2015 38
Number of Products by Stage and Top 10 Mechanism of Actions, H2 2015 38
Number of Products by Top 10 Routes of Administration, H2 2015 40
Number of Products by Stage and Top 10 Routes of Administration, H2 2015 40
Number of Products by Top 10 Molecule Types, H2 2015 42
Number of Products by Stage and Top 10 Molecule Types, H2 2015 42
