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Alpha- Antitrypsin Deficiency - Pipeline Review, H2 2016

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


    Introduction
    Global Markets Direct Report Coverage
    Alpha- Antitrypsin Deficiency Overview
    Therapeutics Development
    Pipeline Products for Alpha- Antitrypsin Deficiency - Overview
    Alpha- Antitrypsin Deficiency - Therapeutics under Development by Companies
    Alpha- Antitrypsin Deficiency - Pipeline Products Glance
    Late Stage Products
    Clinical Stage Products
    Early Stage Products
    Alpha- Antitrypsin Deficiency - Products under Development by Companies
    Alpha- Antitrypsin Deficiency - Companies Involved in Therapeutics Development
    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 - Therapeutics Assessment
    Assessment by Monotherapy Products
    Assessment by Target
    Assessment by Mechanism of Action
    Assessment by Route of Administration
    Assessment by Molecule Type
    Drug Profiles
    ALN-AAT - 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
    ANN-001 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    ARC-AAT - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    CT-2009 - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    DB-027 - 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
    Gene Therapy to Activate Alpha-1 Antitrypsin for Alpha-Antitrypsin Deficiency - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Gene Therapy to Activate Alpha-1 Antitrypsin for Alpha-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
    RNAi Oligonucleotide for Alpha-1 Antitrypsin Deficiency and Liver Disease - Drug Profile
    Product Description
    Mechanism Of Action
    R&D Progress
    Stem Cell Therapy for Alpha-Antitrypsin Deficiency and Crigler-Najjar Syndrome - 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
    May 11, 2016: Dicerna Reports Potent Preclinical Activity with GalNAc-DsiRNA-EX Conjugates
    May 05, 2016: Investigational Gene-Based Therapy for Alpha-1 Antitrypsin Deficiency Demonstrates Durable Response at Five Years
    Feb 25, 2016: Dicerna Reports Progress with GalNAc-DsiRNA-EX-Conjugates
    Jan 20, 2016: AGTC Announces Data Evaluating Novel AAV-Based Gene Therapy as a Potential Treatment for Alpha-1 Antitrypsin Deficiency
    Jan 19, 2016: Arrowhead's ARC-AAT Granted EMA Orphan Drug Designation
    Dec 08, 2015: Kamada Completes Enrollment of U.S. Phase 2 Study of Inhaled Alpha-1 Antitrypsin for the Treatment of AAT Deficiency
    Nov 09, 2015: Sangamo BioSciences Announces Publication Of Improved Method For Efficient Targeted Integration In Hematopoietic Stem Cells And T-cells
    Jul 08, 2015: Arrowhead Receives Regulatory Clearance for ARC-AAT Phase 1 in the United Kingdom and New Zealand
    Jul 02, 2015: Arrowhead Initiates Dosing of ARC-AAT in Patients with Alpha-1 Antitrypsin Deficiency
    Jun 10, 2015: Arrowhead Receives Orphan Drug Designation for ARC-AAT
    May 20, 2015: Kamada Reports Updated Data from European Phase 2/3 Clinical Study of Inhaled Alpha-1 Antitrypsin to Treat AAT Deficiency
    May 05, 2015: Arrowhead Completes Dosing Healthy Volunteers and Initiates Transition to Patients in Phase 1 Study of ARC-AAT
    Apr 20, 2015: Kamada Reports Additional Data from European Phase 2/3 Clinical Study of Inhaled Alpha-1 Antitrypsin to Treat AAT Deficiency
    Feb 23, 2015: Arrowhead Begins Phase 1 Trial of ARC-AAT for Treatment of Liver Disease Associated with Alpha-1 Antitrypsin Deficiency
    Jan 07, 2015: Kamada Reports Encouraging Discussions with European Co-Rapporteurs Regarding European Filing for Inhaled Alpha-1 Antitrypsin to Treat AATD
    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, H2 2016
    Number of Products under Development by Companies, H2 2016
    Comparative Analysis by Late Stage Development, H2 2016
    Comparative Analysis by Clinical Stage Development, H2 2016
    Comparative Analysis by Early Stage Development, H2 2016
    Products under Development by Companies, H2 2016
    Products under Development by Companies, H2 2016 (Contd..1)
    Alpha- Antitrypsin Deficiency - Pipeline by Adverum Biotechnologies, Inc., H2 2016
    Alpha- Antitrypsin Deficiency - Pipeline by Alnylam Pharmaceuticals, Inc., H2 2016
    Alpha- Antitrypsin Deficiency - Pipeline by Applied Genetic Technologies Corporation, H2 2016
    Alpha- Antitrypsin Deficiency - Pipeline by Arrowhead Pharmaceuticals, Inc., H2 2016
    Alpha- Antitrypsin Deficiency - Pipeline by Carolus Therapeutics, Inc., H2 2016
    Alpha- Antitrypsin Deficiency - Pipeline by Cevec Pharmaceuticals GmbH, H2 2016
    Alpha- Antitrypsin Deficiency - Pipeline by Dicerna Pharmaceuticals, Inc., H2 2016
    Alpha- Antitrypsin Deficiency - Pipeline by Digna Biotech, S.L., H2 2016
    Alpha- Antitrypsin Deficiency - Pipeline by Editas Medicine, Inc., H2 2016
    Alpha- Antitrypsin Deficiency - Pipeline by Grifols, S.A., H2 2016
    Alpha- Antitrypsin Deficiency - Pipeline by Inhibrx, H2 2016
    Alpha- Antitrypsin Deficiency - Pipeline by Intellia Therapeutics, Inc., H2 2016
    Alpha- Antitrypsin Deficiency - Pipeline by International Stem Cell Corporation, H2 2016
    Alpha- Antitrypsin Deficiency - Pipeline by Ionis Pharmaceuticals, Inc., H2 2016
    Alpha- Antitrypsin Deficiency - Pipeline by Kamada Ltd., H2 2016
    Alpha- Antitrypsin Deficiency - Pipeline by Polyphor Ltd., H2 2016
    Alpha- Antitrypsin Deficiency - Pipeline by ProMetic Life Sciences Inc., H2 2016
    Alpha- Antitrypsin Deficiency - Pipeline by rEVO Biologics, Inc., H2 2016
    Alpha- Antitrypsin Deficiency - Pipeline by Sangamo BioSciences, Inc., H2 2016
    Assessment by Monotherapy Products, H2 2016
    Number of Products by Stage and Target, H2 2016
    Number of Products by Stage and Mechanism of Action, H2 2016
    Number of Products by Stage and Route of Administration, H2 2016
    Number of Products by Stage and Molecule Type, H2 2016
    Alpha- Antitrypsin Deficiency - Dormant Projects, H2 2016
    Alpha- Antitrypsin Deficiency - Discontinued Products, H2 2016
    List of Figures
    Number of Products under Development for Alpha- Antitrypsin Deficiency, H2 2016
    Number of Products under Development by Companies, H2 2016
    Comparative Analysis by Late Stage Development, H2 2016
    Comparative Analysis by Clinical Stage Development, H2 2016
    Comparative Analysis by Early Stage Products, H2 2016
    Assessment by Monotherapy Products, H2 2016
    Number of Products by Targets, H2 2016
    Number of Products by Stage and Targets, H2 2016
    Number of Products by Mechanism of Actions, H2 2016
    Number of Products by Stage and Mechanism of Actions, H2 2016
    Number of Products by Routes of Administration, H2 2016
    Number of Products by Stage and Routes of Administration, H2 2016
    Number of Products by Molecule Types, H2 2016
    Number of Products by Stage and Molecule Types, H2 2016

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