Alpha- Antitrypsin Deficiency – Pipeline Review, H2 2017

Global Markets Direct’s, ‘Alpha- Antitrypsin Deficiency – Pipeline Review, H2 2017’, 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

Companies mentioned

Adverum Biotechnologies Inc

Alnylam Pharmaceuticals Inc

Applied Genetic Technologies Corp

Arrowhead Pharmaceuticals Inc

Carolus Therapeutics Inc

Cevec Pharmaceuticals GmbH

Editas Medicine Inc

Grifols SA

Inhibrx LP

Intellia Therapeutics Inc

International Stem Cell Corp

Kamada Ltd

OncBioMune Pharmaceuticals Inc

Polyphor Ltd

ProMetic Life Sciences Inc

rEVO Biologics Inc

Z Factor Ltd

Table of Contents

Table of Contents

Table of Contents 2

List of Tables 4

List of Figures 4

Introduction 5

Global Markets Direct Report Coverage 5

Alpha- Antitrypsin Deficiency - Overview 6

Alpha- Antitrypsin Deficiency - Therapeutics Development 7

Pipeline Overview 7

Pipeline by Companies 8

Pipeline by Universities/Institutes 10

Products under Development by Companies 11

Products under Development by Universities/Institutes 13

Alpha- Antitrypsin Deficiency - Therapeutics Assessment 14

Assessment by Target 14

Assessment by Mechanism of Action 16

Assessment by Route of Administration 18

Assessment by Molecule Type 20

Alpha- Antitrypsin Deficiency - Companies Involved in Therapeutics Development 22

Adverum Biotechnologies Inc 22

Alnylam Pharmaceuticals Inc 22

Applied Genetic Technologies Corp 23

Arrowhead Pharmaceuticals Inc 23

Carolus Therapeutics Inc 24

Cevec Pharmaceuticals GmbH 24

Editas Medicine Inc 25

Grifols SA 25

Inhibrx LP 26

Intellia Therapeutics Inc 26

International Stem Cell Corp 27

Kamada Ltd 27

OncBioMune Pharmaceuticals Inc 28

Polyphor Ltd 28

ProMetic Life Sciences Inc 29

rEVO Biologics Inc 29

Z Factor Ltd 30

Alpha- Antitrypsin Deficiency - Drug Profiles 31

ADVM-043 - Drug Profile 31

AGTC-0106 - Drug Profile 33

ALN-AAT - Drug Profile 35

ALNAAT-02 - Drug Profile 38

alpha-1 proteinase inhibitor (human) - Drug Profile 39

alpha-1 proteinase inhibitor (human) second generation - Drug Profile 40

alpha-1 proteinase inhibitor (recombinant) - Drug Profile 44

ARC-AAT - Drug Profile 46

ARO-AAT - Drug Profile 50

CT-2009 - Drug Profile 51

Drug for Chronic Obstructive Pulmonary Disease and Alpha-1 Antitrypsin Deficiency - Drug Profile 52

Gene Therapy for Alpha-1 Antitrypsin Deficiency - Drug Profile 53

Oligonucleotides to Activate Alpha-1 Antitrypsin for Alpha-Antitrypsin Deficiency - Drug Profile 54

Oligonucleotides to Inhibit Alpha-1 Antitrypsin for Alpha-Antitrypsin Deficiency - Drug Profile 55

POL-6014 - Drug Profile 56

rAAV2-CB-hAAT - Drug Profile 58

Recombinant Human Alpha-1 Antitrypsin - Drug Profile 59

Recombinant Human Alpha-1 Antitrypsin Replacement for Alpha-Antitrypsin Deficiency - Drug Profile 60

Recombinant Protein for Alpha-1 Antitrypsin Deficiency - Drug Profile 61

Small Molecules for Alpha-1 Antitrypsin Deficiency - Drug Profile 62

Small Molecules to Activate SERPINA1 for Alpha-1 Antitrypsin Deficiency - Drug Profile 63

Small Molecules to Inhibit Alpha-1 Antitrypsin Polymerization for Alpha-1 Antitrypsin Deficiency - Drug Profile 64

Stem Cell Therapy for Alpha-Antitrypsin Deficiency, Crigler-Najjar Syndrome and Liver Cirrhosis - Drug Profile 65

Alpha- Antitrypsin Deficiency - Dormant Projects 66

Alpha- Antitrypsin Deficiency - Discontinued Products 67

Alpha- Antitrypsin Deficiency - Product Development Milestones 68

Featured News & Press Releases 68

Appendix 80

Methodology 80

Coverage 80

Secondary Research 80

Primary Research 80

Expert Panel Validation 80

Contact Us 80

Disclaimer 81

List of Tables

List of Tables

Number of Products under Development for Alpha- Antitrypsin Deficiency, H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products under Development by Universities/Institutes, H2 2017

Products under Development by Companies, H2 2017

Products under Development by Companies, H2 2017 (Contd..1), H2 2017

Products under Development by Universities/Institutes, H2 2017

Number of Products by Stage and Target, H2 2017

Number of Products by Stage and Mechanism of Action, H2 2017

Number of Products by Stage and Route of Administration, H2 2017

Number of Products by Stage and Molecule Type, H2 2017

Alpha- Antitrypsin Deficiency – Pipeline by Adverum Biotechnologies Inc, H2 2017

Alpha- Antitrypsin Deficiency – Pipeline by Alnylam Pharmaceuticals Inc, H2 2017

Alpha- Antitrypsin Deficiency – Pipeline by Applied Genetic Technologies Corp, H2 2017

Alpha- Antitrypsin Deficiency – Pipeline by Arrowhead Pharmaceuticals Inc, H2 2017

Alpha- Antitrypsin Deficiency – Pipeline by Carolus Therapeutics Inc, H2 2017

Alpha- Antitrypsin Deficiency – Pipeline by Cevec Pharmaceuticals GmbH, H2 2017

Alpha- Antitrypsin Deficiency – Pipeline by Editas Medicine Inc, H2 2017

Alpha- Antitrypsin Deficiency – Pipeline by Grifols SA, H2 2017

Alpha- Antitrypsin Deficiency – Pipeline by Inhibrx LP, H2 2017

Alpha- Antitrypsin Deficiency – Pipeline by Intellia Therapeutics Inc, H2 2017

Alpha- Antitrypsin Deficiency – Pipeline by International Stem Cell Corp, H2 2017

Alpha- Antitrypsin Deficiency – Pipeline by Kamada Ltd, H2 2017

Alpha- Antitrypsin Deficiency – Pipeline by OncBioMune Pharmaceuticals Inc, H2 2017

Alpha- Antitrypsin Deficiency – Pipeline by Polyphor Ltd, H2 2017

Alpha- Antitrypsin Deficiency – Pipeline by ProMetic Life Sciences Inc, H2 2017

Alpha- Antitrypsin Deficiency – Pipeline by rEVO Biologics Inc, H2 2017

Alpha- Antitrypsin Deficiency – Pipeline by Z Factor Ltd, H2 2017

Alpha- Antitrypsin Deficiency – Dormant Projects, H2 2017

Alpha- Antitrypsin Deficiency – Discontinued Products, H2 2017

List of Figures

List of Figures

Number of Products under Development for Alpha- Antitrypsin Deficiency, H2 2017

Number of Products under Development by Companies, H2 2017

Number of Products by Targets, H2 2017

Number of Products by Stage and Targets, H2 2017

Number of Products by Mechanism of Actions, H2 2017

Number of Products by Stage and Mechanism of Actions, H2 2017

Number of Products by Routes of Administration, H2 2017

Number of Products by Stage and Routes of Administration, H2 2017

Number of Products by Molecule Types, H2 2017

Number of Products by Stage and Molecule Types, H2 2017

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