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Alpha- Antitrypsin Deficiency-Pipeline Review, H2 2016

Alpha- Antitrypsin Deficiency-Pipeline Review, H2 2016


  • Products Id :- GMDHC8435IDB
  • |
  • Pages: 95
  • |
  • August 2016
  • |
  • |
  Request for Sample Report

Executive Summary

Alpha- Antitrypsin Deficiency-Pipeline Review, H2 2016

Summary

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



Contact Us:
Ken Research
Ankur Gupta, Head Marketing & Communications
ankur [@] kenresearch.com
+91-9015378249
www.kenresearch.com

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 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

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

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Adverum Biotechnologies, Inc.

Alnylam Pharmaceuticals, Inc.

Applied Genetic Technologies Corporation

Arrowhead Pharmaceuticals, Inc.

Carolus Therapeutics, Inc.

Cevec Pharmaceuticals GmbH

Dicerna Pharmaceuticals, Inc.

Digna Biotech, S.L.

Editas Medicine, Inc.

Grifols, S.A.

Inhibrx

Intellia Therapeutics, Inc.

International Stem Cell Corporation

Ionis Pharmaceuticals, Inc.

Kamada Ltd.

Polyphor Ltd.

ProMetic Life Sciences Inc.

rEVO Biologics, Inc.

Sangamo BioSciences, Inc.

Alpha- Antitrypsin Deficiency Therapeutic Products under Development, Key Players in Alpha- Antitrypsin Deficiency Therapeutics, Alpha- Antitrypsin Deficiency Pipeline Overview, Alpha- Antitrypsin Deficiency Pipeline, Alpha- Antitrypsin Deficiency Pipeline Assessment


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