Alpha- Antitrypsin Deficiency - Pipeline Review, H1 2016

Published: Mar, 2016 | Pages: 91 | Publisher: Global Markets Direct
Industry: Pharmaceuticals & Healthcare | Report Format: Electronic (PDF)

Alpha- Antitrypsin Deficiency - Pipeline Review, H1 2016

Summary

Global Markets Direct's, 'Alpha- Antitrypsin Deficiency - Pipeline Review, H1 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

 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
Alnylam Pharmaceuticals, Inc. 18
Annapurna Therapeutics 19
Applied Genetic Technologies Corporation 20
Arrowhead Research Corporation 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
iBio, Inc. 28
Inhibrx 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 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
alpha-1 proteinase inhibitor (human) second generation - Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
ANN-001 - Drug Profile 51
Product Description 51
Mechanism of Action 51
R&D Progress 51
ARC-AAT - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
CT-2009 - Drug Profile 53
Product Description 53
Mechanism of Action 53
R&D Progress 53
DB-027 - Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
Drugs for Alpha-1 Antitrypsin Deficiency - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
Gene Therapy to Activate Alpha-1 Antitrypsin for Alpha-Antitrypsin Deficiency - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
Gene Therapy to Activate Alpha-1 Antitrypsin for Alpha-Antitrypsin Deficiency - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
ISIS-AATRx - Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
POL-6014 - Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
rAAV2-CB-hAAT - Drug Profile 62
Product Description 62
Mechanism of Action 62
R&D Progress 62
Recombinant A1PI - Drug Profile 63
Product Description 63
Mechanism of Action 63
R&D Progress 63
Recombinant Alpha-1 Antitrypsin Replacement for Alpha-Antitrypsin Deficiency - Drug Profile 65
Product Description 65
Mechanism of Action 65
R&D Progress 65
Recombinant Human Alpha-1 Antitrypsin - Drug Profile 66
Product Description 66
Mechanism of Action 66
R&D Progress 66
Recombinant Human Alpha-1 Antitrypsin - Drug Profile 67
Product Description 67
Mechanism of Action 67
R&D Progress 67
Recombinant Protein for Alpha-1 Antitrypsin Deficiency - Drug Profile 68
Product Description 68
Mechanism of Action 68
R&D Progress 68
RNAi Oligonucleotide for Alpha- Antitrypsin Deficiency and Liver Disease - Drug Profile 69
Product Description 69
Mechanism of Action 69
R&D Progress 69
Stem Cell Therapy for Alpha-Antitrypsin Deficiency and Crigler-Najjar Syndrome - Drug Profile 71
Product Description 71
Mechanism of Action 71
R&D Progress 71
Alpha- Antitrypsin Deficiency - Recent Pipeline Updates 72
Alpha- Antitrypsin Deficiency - Dormant Projects 80
Alpha- Antitrypsin Deficiency - Discontinued Products 81
Alpha- Antitrypsin Deficiency - Product Development Milestones 82
Featured News & Press Releases 82
Feb 25, 2016: Dicerna Reports Progress with GalNAc-DsiRNA-EX-Conjugates 82
Jan 20, 2016: AGTC Announces Data Evaluating Novel AAV-Based Gene Therapy as a Potential Treatment for Alpha-1 Antitrypsin Deficiency 83
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 84
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 87
May 05, 2015: Arrowhead Completes Dosing Healthy Volunteers and Initiates Transition to Patients in Phase 1 Study of ARC-AAT 88
Appendix 90
Methodology 90
Coverage 90
Secondary Research 90
Primary Research 90
Expert Panel Validation 90
Contact Us 90
Disclaimer 91

List of Tables

Number of Products under Development for Alpha- Antitrypsin Deficiency, H1 2016 10
Number of Products under Development by Companies, H1 2016 12
Comparative Analysis by Late Stage Development, H1 2016 13
Comparative Analysis by Clinical Stage Development, H1 2016 14
Comparative Analysis by Early Stage Development, H1 2016 15
Products under Development by Companies, H1 2016 16
Products under Development by Companies, H1 2016 (Contd..1) 17
Alpha- Antitrypsin Deficiency - Pipeline by Alnylam Pharmaceuticals, Inc., H1 2016 18
Alpha- Antitrypsin Deficiency - Pipeline by Annapurna Therapeutics, H1 2016 19
Alpha- Antitrypsin Deficiency - Pipeline by Applied Genetic Technologies Corporation, H1 2016 20
Alpha- Antitrypsin Deficiency - Pipeline by Arrowhead Research Corporation, H1 2016 21
Alpha- Antitrypsin Deficiency - Pipeline by Carolus Therapeutics, Inc., H1 2016 22
Alpha- Antitrypsin Deficiency - Pipeline by Cevec Pharmaceuticals GmbH, H1 2016 23
Alpha- Antitrypsin Deficiency - Pipeline by Dicerna Pharmaceuticals, Inc., H1 2016 24
Alpha- Antitrypsin Deficiency - Pipeline by Digna Biotech, S.L., H1 2016 25
Alpha- Antitrypsin Deficiency - Pipeline by Editas Medicine, Inc., H1 2016 26
Alpha- Antitrypsin Deficiency - Pipeline by Grifols, S.A., H1 2016 27
Alpha- Antitrypsin Deficiency - Pipeline by iBio, Inc., H1 2016 28
Alpha- Antitrypsin Deficiency - Pipeline by Inhibrx, H1 2016 29
Alpha- Antitrypsin Deficiency - Pipeline by International Stem Cell Corporation, H1 2016 30
Alpha- Antitrypsin Deficiency - Pipeline by Ionis Pharmaceuticals, Inc., H1 2016 31
Alpha- Antitrypsin Deficiency - Pipeline by Kamada Ltd., H1 2016 32
Alpha- Antitrypsin Deficiency - Pipeline by Polyphor Ltd., H1 2016 33
Alpha- Antitrypsin Deficiency - Pipeline by ProMetic Life Sciences Inc., H1 2016 34
Alpha- Antitrypsin Deficiency - Pipeline by rEVO Biologics, Inc., H1 2016 35
Alpha- Antitrypsin Deficiency - Pipeline by Sangamo BioSciences, Inc., H1 2016 36
Assessment by Monotherapy Products, H1 2016 37
Number of Products by Stage and Target, H1 2016 39
Number of Products by Stage and Mechanism of Action, H1 2016 41
Number of Products by Stage and Route of Administration, H1 2016 43
Number of Products by Stage and Molecule Type, H1 2016 45
Alpha- Antitrypsin Deficiency Therapeutics - Recent Pipeline Updates, H1 2016 72
Alpha- Antitrypsin Deficiency - Dormant Projects, H1 2016 80
Alpha- Antitrypsin Deficiency - Discontinued Products, H1 2016 81