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Alpha- Antitrypsin Deficiency - Pipeline Review, H1 2017

Published By :

Global Markets Direct

Published Date : Mar 2017

Category :

Pharmaceutical

No. of Pages : 82 Pages

Alpha- Antitrypsin Deficiency - Pipeline Review, H1 2017

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Alpha- Antitrypsin Deficiency - Pipeline Review, H1 2017, provides an overview of the Alpha- Antitrypsin Deficiency (Genetic Disorders) pipeline landscape.

Alpha-antitrypsin deficiency is an inherited disease that occurs due to lack of alpha-1 antitrypsin (AAT), a protein that protects the lungs. Symptoms include shortness of breath and wheezing, lung infections, rapid heartbeat, weight loss and vision problems. Predisposing factors include smoking, exposure to dust, chemical fumes and infections.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Alpha- Antitrypsin Deficiency - Pipeline Review, H1 2017, provides comprehensive information on the therapeutics under development for Alpha- Antitrypsin Deficiency (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Alpha- Antitrypsin Deficiency (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Alpha- Antitrypsin Deficiency and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 1, 3, 1, 9 and 5 respectively. Similarly, the Universities portfolio in Discovery stages comprises 1 molecules, respectively.

Alpha- Antitrypsin Deficiency (Genetic Disorders) pipeline guide 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. The guide is built using data and information sourced from Global Markets Directs 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

- The pipeline guide provides a snapshot of the global therapeutic landscape of Alpha- Antitrypsin Deficiency (Genetic Disorders).
- The pipeline guide reviews pipeline therapeutics for Alpha- Antitrypsin Deficiency (Genetic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
- The pipeline guide reviews key companies involved in Alpha- Antitrypsin Deficiency (Genetic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Alpha- Antitrypsin Deficiency (Genetic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
- The pipeline guide reviews latest news related to pipeline therapeutics for Alpha- Antitrypsin Deficiency (Genetic Disorders)

Reasons to buy

- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Alpha- Antitrypsin Deficiency (Genetic Disorders).
- Classify potential new clients or partners in the target demographic.
- Develop tactical initiatives by understanding the focus areas of leading companies.
- Plan mergers and acquisitions meritoriously by identifying key players and its most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Alpha- Antitrypsin Deficiency (Genetic Disorders) 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.
- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.

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
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
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

List of Table

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