Alpha- Antitrypsin Deficiency - Pipeline Review, H2 2016

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Date: 31-Aug-2016
No. of pages: 95
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Global Markets Direct's, ‘Alpha- Antitrypsin Deficiency - Pipeline Review, H2 2016', provides an overview of the Alpha- Antitrypsin Deficiency pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Alpha- Antitrypsin Deficiency, 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 Alpha- Antitrypsin Deficiency 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 Alpha- Antitrypsin Deficiency

  • The report reviews pipeline therapeutics for Alpha- Antitrypsin Deficiency 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 Alpha- Antitrypsin Deficiency therapeutics and enlists all their major and minor projects

  • The report assesses Alpha- Antitrypsin Deficiency 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 Alpha- Antitrypsin Deficiency

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 Alpha- Antitrypsin Deficiency

  • 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 Alpha- Antitrypsin 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

Alpha- Antitrypsin Deficiency - Pipeline Review, H2 2016

Table of Contents

Table of Contents 2
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
Alpha- Antitrypsin Deficiency - Therapeutics under Development by Companies 11
Alpha- Antitrypsin Deficiency - Pipeline Products Glance 13
Late Stage Products 13
Clinical Stage Products 14
Early Stage Products 15
Alpha- Antitrypsin Deficiency - Products under Development by Companies 16
Alpha- Antitrypsin Deficiency - Companies Involved in Therapeutics Development 18
Adverum Biotechnologies, Inc. 18
Alnylam Pharmaceuticals, Inc. 19
Applied Genetic Technologies Corporation 20
Arrowhead Pharmaceuticals, Inc. 21
Carolus Therapeutics, Inc. 22
Cevec Pharmaceuticals GmbH 23
Dicerna Pharmaceuticals, Inc. 24
Digna Biotech, S.L. 25
Editas Medicine, Inc. 26
Grifols, S.A. 27
Inhibrx 28
Intellia Therapeutics, Inc. 29
International Stem Cell Corporation 30
Ionis Pharmaceuticals, Inc. 31
Kamada Ltd. 32
Polyphor Ltd. 33
ProMetic Life Sciences Inc. 34
rEVO Biologics, Inc. 35
Sangamo BioSciences, Inc. 36
Alpha- Antitrypsin Deficiency - Therapeutics Assessment 37
Assessment by Monotherapy Products 37
Assessment by Target 38
Assessment by Mechanism of Action 40
Assessment by Route of Administration 42
Assessment by Molecule Type 44
Drug Profiles 46
ALN-AAT - Drug Profile 46
Product Description 46
Mechanism Of Action 46
R&D Progress 46
alpha-1 proteinase inhibitor (human) - Drug Profile 49
Product Description 49
Mechanism Of Action 49
R&D Progress 49
alpha-1 proteinase inhibitor (human) second generation - Drug Profile 50
Product Description 50
Mechanism Of Action 50
R&D Progress 50
ANN-001 - Drug Profile 53
Product Description 53
Mechanism Of Action 53
R&D Progress 53
ARC-AAT - Drug Profile 55
Product Description 55
Mechanism Of Action 55
R&D Progress 55
CT-2009 - Drug Profile 58
Product Description 58
Mechanism Of Action 58
R&D Progress 58
DB-027 - Drug Profile 59
Product Description 59
Mechanism Of Action 59
R&D Progress 59
Drugs for Alpha-1 Antitrypsin Deficiency - Drug Profile 60
Product Description 60
Mechanism Of Action 60
R&D Progress 60
Gene Therapy to Activate Alpha-1 Antitrypsin for Alpha-Antitrypsin Deficiency - Drug Profile 61
Product Description 61
Mechanism Of Action 61
R&D Progress 61
Gene Therapy to Activate Alpha-1 Antitrypsin for Alpha-Antitrypsin Deficiency - Drug Profile 63
Product Description 63
Mechanism Of Action 63
R&D Progress 63
ISIS-AATRx - Drug Profile 64
Product Description 64
Mechanism Of Action 64
R&D Progress 64
Oligonucleotides to Activate Alpha-1 Antitrypsin for Alpha-Antitrypsin Deficiency - Drug Profile 65
Product Description 65
Mechanism Of Action 65
R&D Progress 65
Oligonucleotides to Inhibit Alpha-1 Antitrypsin for Alpha-Antitrypsin Deficiency - Drug Profile 66
Product Description 66
Mechanism Of Action 66
R&D Progress 66
POL-6014 - Drug Profile 67
Product Description 67
Mechanism Of Action 67
R&D Progress 67
rAAV2-CB-hAAT - Drug Profile 69
Product Description 69
Mechanism Of Action 69
R&D Progress 69
Recombinant A1PI - Drug Profile 70
Product Description 70
Mechanism Of Action 70
R&D Progress 70
Recombinant Human Alpha-1 Antitrypsin - Drug Profile 72
Product Description 72
Mechanism Of Action 72
R&D Progress 72
Recombinant Human Alpha-1 Antitrypsin Replacement for Alpha-Antitrypsin Deficiency - Drug Profile 73
Product Description 73
Mechanism Of Action 73
R&D Progress 73
Recombinant Protein for Alpha-1 Antitrypsin Deficiency - Drug Profile 74
Product Description 74
Mechanism Of Action 74
R&D Progress 74
RNAi Oligonucleotide for Alpha-1 Antitrypsin Deficiency and Liver Disease - Drug Profile 75
Product Description 75
Mechanism Of Action 75
R&D Progress 75
Stem Cell Therapy for Alpha-Antitrypsin Deficiency and Crigler-Najjar Syndrome - Drug Profile 77
Product Description 77
Mechanism Of Action 77
R&D Progress 77
Alpha- Antitrypsin Deficiency - Dormant Projects 78
Alpha- Antitrypsin Deficiency - Discontinued Products 79
Alpha- Antitrypsin Deficiency - Product Development Milestones 80
Featured News & Press Releases 80
May 11, 2016: Dicerna Reports Potent Preclinical Activity with GalNAc-DsiRNA-EX Conjugates 80
May 05, 2016: Investigational Gene-Based Therapy for Alpha-1 Antitrypsin Deficiency Demonstrates Durable Response at Five Years 81
Feb 25, 2016: Dicerna Reports Progress with GalNAc-DsiRNA-EX-Conjugates 81
Jan 20, 2016: AGTC Announces Data Evaluating Novel AAV-Based Gene Therapy as a Potential Treatment for Alpha-1 Antitrypsin Deficiency 82
Jan 19, 2016: Arrowhead's ARC-AAT Granted EMA Orphan Drug Designation 83
Dec 08, 2015: Kamada Completes Enrollment of U.S. Phase 2 Study of Inhaled Alpha-1 Antitrypsin for the Treatment of AAT Deficiency 83
Nov 09, 2015: Sangamo BioSciences Announces Publication Of Improved Method For Efficient Targeted Integration In Hematopoietic Stem Cells And T-cells 84
Jul 08, 2015: Arrowhead Receives Regulatory Clearance for ARC-AAT Phase 1 in the United Kingdom and New Zealand 85
Jul 02, 2015: Arrowhead Initiates Dosing of ARC-AAT in Patients with Alpha-1 Antitrypsin Deficiency 85
Jun 10, 2015: Arrowhead Receives Orphan Drug Designation for ARC-AAT 86
May 20, 2015: Kamada Reports Updated Data from European Phase 2/3 Clinical Study of Inhaled Alpha-1 Antitrypsin to Treat AAT Deficiency 86
May 05, 2015: Arrowhead Completes Dosing Healthy Volunteers and Initiates Transition to Patients in Phase 1 Study of ARC-AAT 88
Apr 20, 2015: Kamada Reports Additional Data from European Phase 2/3 Clinical Study of Inhaled Alpha-1 Antitrypsin to Treat AAT Deficiency 89
Feb 23, 2015: Arrowhead Begins Phase 1 Trial of ARC-AAT for Treatment of Liver Disease Associated with Alpha-1 Antitrypsin Deficiency 92
Jan 07, 2015: Kamada Reports Encouraging Discussions with European Co-Rapporteurs Regarding European Filing for Inhaled Alpha-1 Antitrypsin to Treat AATD 93
Appendix 94
Methodology 94
Coverage 94
Secondary Research 94
Primary Research 94
Expert Panel Validation 94
Contact Us 94
Disclaimer 95

List of Tables

Number of Products under Development for Alpha- Antitrypsin Deficiency, H2 2016 10
Number of Products under Development by Companies, H2 2016 12
Comparative Analysis by Late Stage Development, H2 2016 13
Comparative Analysis by Clinical Stage Development, H2 2016 14
Comparative Analysis by Early Stage Development, H2 2016 15
Products under Development by Companies, H2 2016 16
Products under Development by Companies, H2 2016 (Contd..1) 17
Alpha- Antitrypsin Deficiency - Pipeline by Adverum Biotechnologies, Inc., H2 2016 18
Alpha- Antitrypsin Deficiency - Pipeline by Alnylam Pharmaceuticals, Inc., H2 2016 19
Alpha- Antitrypsin Deficiency - Pipeline by Applied Genetic Technologies Corporation, H2 2016 20
Alpha- Antitrypsin Deficiency - Pipeline by Arrowhead Pharmaceuticals, Inc., H2 2016 21
Alpha- Antitrypsin Deficiency - Pipeline by Carolus Therapeutics, Inc., H2 2016 22
Alpha- Antitrypsin Deficiency - Pipeline by Cevec Pharmaceuticals GmbH, H2 2016 23
Alpha- Antitrypsin Deficiency - Pipeline by Dicerna Pharmaceuticals, Inc., H2 2016 24
Alpha- Antitrypsin Deficiency - Pipeline by Digna Biotech, S.L., H2 2016 25
Alpha- Antitrypsin Deficiency - Pipeline by Editas Medicine, Inc., H2 2016 26
Alpha- Antitrypsin Deficiency - Pipeline by Grifols, S.A., H2 2016 27
Alpha- Antitrypsin Deficiency - Pipeline by Inhibrx, H2 2016 28
Alpha- Antitrypsin Deficiency - Pipeline by Intellia Therapeutics, Inc., H2 2016 29
Alpha- Antitrypsin Deficiency - Pipeline by International Stem Cell Corporation, H2 2016 30
Alpha- Antitrypsin Deficiency - Pipeline by Ionis Pharmaceuticals, Inc., H2 2016 31
Alpha- Antitrypsin Deficiency - Pipeline by Kamada Ltd., H2 2016 32
Alpha- Antitrypsin Deficiency - Pipeline by Polyphor Ltd., H2 2016 33
Alpha- Antitrypsin Deficiency - Pipeline by ProMetic Life Sciences Inc., H2 2016 34
Alpha- Antitrypsin Deficiency - Pipeline by rEVO Biologics, Inc., H2 2016 35
Alpha- Antitrypsin Deficiency - Pipeline by Sangamo BioSciences, Inc., H2 2016 36
Assessment by Monotherapy Products, H2 2016 37
Number of Products by Stage and Target, H2 2016 39
Number of Products by Stage and Mechanism of Action, H2 2016 41
Number of Products by Stage and Route of Administration, H2 2016 43
Number of Products by Stage and Molecule Type, H2 2016 45
Alpha- Antitrypsin Deficiency - Dormant Projects, H2 2016 78
Alpha- Antitrypsin Deficiency - Discontinued Products, H2 2016 79

List of Figures

Number of Products under Development for Alpha- Antitrypsin Deficiency, H2 2016 10
Number of Products under Development by Companies, H2 2016 11
Comparative Analysis by Late Stage Development, H2 2016 13
Comparative Analysis by Clinical Stage Development, H2 2016 14
Comparative Analysis by Early Stage Products, H2 2016 15
Assessment by Monotherapy Products, H2 2016 37
Number of Products by Targets, H2 2016 38
Number of Products by Stage and Targets, H2 2016 38
Number of Products by Mechanism of Actions, H2 2016 40
Number of Products by Stage and Mechanism of Actions, H2 2016 40
Number of Products by Routes of Administration, H2 2016 42
Number of Products by Stage and Routes of Administration, H2 2016 42
Number of Products by Molecule Types, H2 2016 44
Number of Products by Stage and Molecule Types, H2 2016 44
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