Alpha- Antitrypsin Deficiency - Pipeline Review, H2 2014

Published: Nov, 2014 | Pages: 80 | Publisher: Global Markets Direct
Industry: Healthcare | Report Format: Electronic (PDF)

Alpha- Antitrypsin Deficiency - Pipeline Review, H2 2014

Summary

Global Markets Direct's, 'Alpha- Antitrypsin Deficiency - Pipeline Review, H2 2014', 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 Alpha- Antitrypsin 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 Alpha- Antitrypsin Deficiency and enlists all their major and minor projects
- The report summarizes all the dormant and discontinued pipeline projects 
- A review of the Alpha- Antitrypsin 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 Alpha- Antitrypsin 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 Alpha- Antitrypsin Deficiency
- Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
- 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 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
Clinical Stage Products 14
Early Stage Products 15
Alpha- Antitrypsin Deficiency - Products under Development by Companies 16
Alpha- Antitrypsin Deficiency - Products under Investigation by Universities/Institutes 17
Alpha- Antitrypsin Deficiency - Companies Involved in Therapeutics Development 18
Applied Genetic Technologies Corporation 18
Arrowhead Research Corporation 19
Carolus Therapeutics, Inc. 20
Cevec Pharmaceuticals GmbH 21
Dicerna Pharmaceuticals, Inc. 22
Grifols, S.A. 23
iBio, Inc. 24
International Stem Cell Corporation 25
Intrexon Corporation 26
Kamada Ltd. 27
OrphageniX, Inc. 28
Polyphor Ltd. 29
rEVO Biologics 30
Sangamo BioSciences, Inc. 31
Alpha- Antitrypsin Deficiency - 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
Alpha-1 Antitrypsin With rHuPH20 - Drug Profile 41
Product Description 41
Mechanism of Action 41
R&D Progress 41
alpha-1-proteinase inhibitor (human) - Drug Profile 42
Product Description 42
Mechanism of Action 42
R&D Progress 42
ARC-AAT - Drug Profile 46
Product Description 46
Mechanism of Action 46
R&D Progress 46
CT-2009 - Drug Profile 47
Product Description 47
Mechanism of Action 47
R&D Progress 47
DC-AIM - Drug Profile 48
Product Description 48
Mechanism of Action 48
R&D Progress 48
DNA Oligonucleotides for Alpha-1 Antitrypsin Deficiency - 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
POL-6014 - Drug Profile 52
Product Description 52
Mechanism of Action 52
R&D Progress 52
rAAV1-CB-hAAT - Drug Profile 54
Product Description 54
Mechanism of Action 54
R&D Progress 54
rAAV2-CB-hAAT - Drug Profile 55
Product Description 55
Mechanism of Action 55
R&D Progress 55
Recombinant A1PI - Drug Profile 56
Product Description 56
Mechanism of Action 56
R&D Progress 56
Recombinant Human Alpha-1 Antitrypsin - Drug Profile 58
Product Description 58
Mechanism of Action 58
R&D Progress 58
Recombinant Human Alpha-1 Antitrypsin - Drug Profile 59
Product Description 59
Mechanism of Action 59
R&D Progress 59
Recombinant Protein to Replace Alpha-1 Antitrypsin for Alpha-Antitrypsin Deficiency - Drug Profile 60
Product Description 60
Mechanism of Action 60
R&D Progress 60
RNAi Oligonucleotide for Alpha- Antitrypsin Deficiency - Drug Profile 61
Product Description 61
Mechanism of Action 61
R&D Progress 61
Stem Cell Therapy for Alpha-Antitrypsin Deficiency and Crigler-Najjar Syndrome - Drug Profile 62
Product Description 62
Mechanism of Action 62
R&D Progress 62
Alpha- Antitrypsin Deficiency - Recent Pipeline Updates 63
Alpha- Antitrypsin Deficiency - Dormant Projects 68
Alpha- Antitrypsin Deficiency - Discontinued Products 69
Alpha- Antitrypsin Deficiency - Product Development Milestones 70
Featured News & Press Releases 70
Nov 18, 2014: Arrowhead Files For Regulatory Permission To Begin Phase 1 Trial Of RNAi Therapeutic ARC-AAT 70
Nov 10, 2014: Arrowhead Presents Data on ARC-AAT at AASLD The Liver Meeting 2014 70
Sep 29, 2014: Kamada Announces Second Extension of Strategic Agreement with Baxter 71
Sep 04, 2014: Kamada Reports Final Results from Phase 2/3 Clinical Trial of Inhaled Alpha-1 Antitrypsin to Treat Alpha-1 Antitrypsin Deficiency 72
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 73
Jun 19, 2014: Arrowhead Announces New Clinical Candidate ARC-AAT for Treatment of Alpha-1 Antitrypsin Deficiency-Associated Liver Disease 73
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 74
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 76
Apr 24, 2014: Kamada Announces Significantly Improved Infusion Rate for Glassia 76
Mar 24, 2014: Kamada Announces Initiation of a Phase 2 U.S. Clinical Trial of Inhaled AAT to Treat Alpha-1 Antitrypsin Deficiency 77
Appendix 79
Methodology 79
Coverage 79
Secondary Research 79
Primary Research 79
Expert Panel Validation 79
Contact Us 80
Disclaimer 80

List of Tables
Number of Products under Development for Alpha- Antitrypsin Deficiency, H2 2014 9
Number of Products under Development for Alpha- Antitrypsin Deficiency - Comparative Analysis, H2 2014 10
Number of Products under Development by Companies, H2 2014 12
Number of Products under Investigation by Universities/Institutes, H2 2014 13
Comparative Analysis by Clinical Stage Development, H2 2014 14
Comparative Analysis by Early Stage Development, H2 2014 15
Products under Development by Companies, H2 2014 16
Products under Investigation by Universities/Institutes, H2 2014 17
Alpha- Antitrypsin Deficiency - Pipeline by Applied Genetic Technologies Corporation, H2 2014 18
Alpha- Antitrypsin Deficiency - Pipeline by Arrowhead Research Corporation, H2 2014 19
Alpha- Antitrypsin Deficiency - Pipeline by Carolus Therapeutics, Inc., H2 2014 20
Alpha- Antitrypsin Deficiency - Pipeline by Cevec Pharmaceuticals GmbH, H2 2014 21
Alpha- Antitrypsin Deficiency - Pipeline by Dicerna Pharmaceuticals, Inc., H2 2014 22
Alpha- Antitrypsin Deficiency - Pipeline by Grifols, S.A., H2 2014 23
Alpha- Antitrypsin Deficiency - Pipeline by iBio, Inc., H2 2014 24
Alpha- Antitrypsin Deficiency - Pipeline by International Stem Cell Corporation, H2 2014 25
Alpha- Antitrypsin Deficiency - Pipeline by Intrexon Corporation, H2 2014 26
Alpha- Antitrypsin Deficiency - Pipeline by Kamada Ltd., H2 2014 27
Alpha- Antitrypsin Deficiency - Pipeline by OrphageniX, Inc., H2 2014 28
Alpha- Antitrypsin Deficiency - Pipeline by Polyphor Ltd., H2 2014 29
Alpha- Antitrypsin Deficiency - Pipeline by rEVO Biologics, H2 2014 30
Alpha- Antitrypsin Deficiency - Pipeline by Sangamo BioSciences, Inc., H2 2014 31
Assessment by Monotherapy Products, H2 2014 32
Number of Products by Stage and Target, H2 2014 34
Number of Products by Stage and Mechanism of Action, H2 2014 36
Number of Products by Stage and Route of Administration, H2 2014 38
Number of Products by Stage and Molecule Type, H2 2014 40
Alpha- Antitrypsin Deficiency Therapeutics - Recent Pipeline Updates, H2 2014 63
Alpha- Antitrypsin Deficiency - Dormant Projects, H2 2014 68
Alpha- Antitrypsin Deficiency - Discontinued Products, H2 2014 69