Alpha- Antitrypsin Deficiency-Pipeline Review, H1 2016

Alpha- Antitrypsin Deficiency-Pipeline Review, H1 2016


  • Products Id :- GMDHC7735IDB
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  • Pages: 91
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Executive Summary

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



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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 Figures

Number of Products under Development for Alpha- Antitrypsin Deficiency, H1 2016 10

Number of Products under Development by Companies, H1 2016 11

Comparative Analysis by Clinical Stage Development, H1 2016 14

Comparative Analysis by Early Stage Products, H1 2016 15

Assessment by Monotherapy Products, H1 2016 37

Number of Products by Targets, H1 2016 38

Number of Products by Stage and Targets, H1 2016 38

Number of Products by Mechanism of Actions, H1 2016 40

Number of Products by Stage and Mechanism of Actions, H1 2016 40

Number of Products by Routes of Administration, H1 2016 42

Number of Products by Stage and Routes of Administration, H1 2016 42

Number of Products by Molecule Types, H1 2016 44

Number of Products by Stage and Molecule Types, H1 2016 44

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

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Alnylam Pharmaceuticals, Inc. Annapurna Therapeutics Applied Genetic Technologies Corporation Arrowhead Research Corporation Carolus Therapeutics, Inc. Cevec Pharmaceuticals GmbH Dicerna Pharmaceuticals, Inc. Digna Biotech, S.L. Editas Medicine, Inc. Grifols, S.A. iBio, Inc. Inhibrx 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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