Alpha- Antitrypsin Deficiency – Pipeline Review, H1 2017

Global Markets Direct’s, ‘Alpha- Antitrypsin Deficiency – Pipeline Review, H1 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

Apollo Therapeutics LLC

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

Ionis Pharmaceuticals Inc

Kamada Ltd

Polyphor Ltd

ProMetic Life Sciences Inc

rEVO Biologics Inc

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Alpha- Antitrypsin Deficiency - Overview

Alpha- Antitrypsin Deficiency - Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Alpha- Antitrypsin Deficiency - Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Alpha- Antitrypsin Deficiency - Companies Involved in Therapeutics Development

Adverum Biotechnologies Inc

Alnylam Pharmaceuticals Inc

Apollo Therapeutics LLC

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

Ionis Pharmaceuticals Inc

Kamada Ltd

Polyphor Ltd

ProMetic Life Sciences Inc

rEVO Biologics Inc

Alpha- Antitrypsin Deficiency - Drug Profiles

ADVM-043 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

AGTC-0106 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ALN-AAT - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ALNAAT-02 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

alpha-1 proteinase inhibitor (human) - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

ARO-AAT - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

CT-2009 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Drugs for Alpha-1 Antitrypsin Deficiency - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ISIS-AATRx - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

POL-6014 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

rAAV2-CB-hAAT - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Recombinant A1PI - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Recombinant Human Alpha-1 Antitrypsin - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

Recombinant Protein for Alpha-1 Antitrypsin Deficiency - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules for Alpha-1 Antitrypsin Deficiency - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

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

Product Description

Mechanism Of Action

R&D Progress

Alpha- Antitrypsin Deficiency - Dormant Projects

Alpha- Antitrypsin Deficiency - Discontinued Products

Alpha- Antitrypsin Deficiency - Product Development Milestones

Featured News & Press Releases

Oct 14, 2016: Adverum Biotechnologies Provides Update on Alpha-1 Antitrypsin Deficiency Program

Aug 30, 2016: Kamada Meets Primary Endpoint of U.S. Phase 2 Study of Inhaled Alpha-1 Antitrypsin for the Treatment of Alpha-1 Antitrypsin Deficiency

May 05, 2016: Investigational Gene-Based Therapy for Alpha-1 Antitrypsin Deficiency Demonstrates Durable Response at Five Years

Jan 20, 2016: AGTC Announces Data Evaluating Novel AAV-Based Gene Therapy as a Potential Treatment for Alpha-1 Antitrypsin Deficiency

Dec 08, 2015: Kamada Completes Enrollment of U.S. Phase 2 Study of Inhaled Alpha-1 Antitrypsin for the Treatment of AAT Deficiency

May 20, 2015: Kamada Reports Updated Data from European Phase 2/3 Clinical Study of Inhaled Alpha-1 Antitrypsin to Treat AAT Deficiency

Apr 20, 2015: Kamada Reports Additional Data from European Phase 2/3 Clinical Study of Inhaled Alpha-1 Antitrypsin to Treat AAT Deficiency

Jan 07, 2015: Kamada Reports Encouraging Discussions with European Co-Rapporteurs Regarding European Filing for Inhaled Alpha-1 Antitrypsin to Treat AATD

Sep 04, 2014: Kamada Reports Final Results from Phase 2/3 Clinical Trial of Inhaled Alpha-1 Antitrypsin to Treat Alpha-1 Antitrypsin Deficiency

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

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

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

Mar 24, 2014: Kamada Announces Initiation of a Phase 2 U.S. Clinical Trial of Inhaled AAT to Treat Alpha-1 Antitrypsin Deficiency

Mar 21, 2014: Kamada to Report Top-Line Data from Phase 2/3 Clinical Trial of Inhaled AAT to Treat Alpha-1 Antitrypsin Deficiency by Late April or Early May

Dec 11, 2013: Kamada Announces Completion of Pivotal Phase II/III Clinical Trial in Europe and Canada of Inhaled AAT to Treat Alpha-1 Antitrypsin Deficiency

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

List of Tables

Number of Products under Development for Alpha- Antitrypsin Deficiency, H1 2017

Number of Products under Development by Companies, H1 2017

Number of Products under Development by Universities/Institutes, H1 2017

Products under Development by Companies, H1 2017

Products under Development by Companies, H1 2017 (Contd..1), H1 2017

Products under Development by Universities/Institutes, H1 2017

Number of Products by Stage and Target, H1 2017

Number of Products by Stage and Mechanism of Action, H1 2017

Number of Products by Stage and Route of Administration, H1 2017

Number of Products by Stage and Molecule Type, H1 2017

Alpha- Antitrypsin Deficiency – Pipeline by Adverum Biotechnologies Inc, H1 2017

Alpha- Antitrypsin Deficiency – Pipeline by Alnylam Pharmaceuticals Inc, H1 2017

Alpha- Antitrypsin Deficiency – Pipeline by Apollo Therapeutics LLC, H1 2017

Alpha- Antitrypsin Deficiency – Pipeline by Applied Genetic Technologies Corp, H1 2017

Alpha- Antitrypsin Deficiency – Pipeline by Arrowhead Pharmaceuticals Inc, H1 2017

Alpha- Antitrypsin Deficiency – Pipeline by Carolus Therapeutics Inc, H1 2017

Alpha- Antitrypsin Deficiency – Pipeline by Cevec Pharmaceuticals GmbH, H1 2017

Alpha- Antitrypsin Deficiency – Pipeline by Editas Medicine Inc, H1 2017

Alpha- Antitrypsin Deficiency – Pipeline by Grifols SA, H1 2017

Alpha- Antitrypsin Deficiency – Pipeline by Inhibrx LP, H1 2017

Alpha- Antitrypsin Deficiency – Pipeline by Intellia Therapeutics Inc, H1 2017

Alpha- Antitrypsin Deficiency – Pipeline by International Stem Cell Corp, H1 2017

Alpha- Antitrypsin Deficiency – Pipeline by Ionis Pharmaceuticals Inc, H1 2017

Alpha- Antitrypsin Deficiency – Pipeline by Kamada Ltd, H1 2017

Alpha- Antitrypsin Deficiency – Pipeline by Polyphor Ltd, H1 2017

Alpha- Antitrypsin Deficiency – Pipeline by ProMetic Life Sciences Inc, H1 2017

Alpha- Antitrypsin Deficiency – Pipeline by rEVO Biologics Inc, H1 2017

Alpha- Antitrypsin Deficiency – Dormant Projects, H1 2017

Alpha- Antitrypsin Deficiency – Discontinued Products, H1 2017

List of Figures

List of Figures

Number of Products under Development for Alpha- Antitrypsin Deficiency, H1 2017

Number of Products under Development by Companies, H1 2017

Number of Products by Targets, H1 2017

Number of Products by Stage and Targets, H1 2017

Number of Products by Mechanism of Actions, H1 2017

Number of Products by Stage and Mechanism of Actions, H1 2017

Number of Products by Routes of Administration, H1 2017

Number of Products by Stage and Routes of Administration, H1 2017

Number of Products by Top 10 Molecule Types, H1 2017

Number of Products by Stage and Top 10 Molecule Types, H1 2017

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