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甲一型抗胰蛋白酵素缺乏症:開發中產品分析

Alpha- Antitrypsin Deficiency - Pipeline Review, H2 2016

出版商 Global Markets Direct 商品編碼 203700
出版日期 內容資訊 英文 95 Pages
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甲一型抗胰蛋白酵素缺乏症:開發中產品分析 Alpha- Antitrypsin Deficiency - Pipeline Review, H2 2016
出版日期: 2016年08月31日 內容資訊: 英文 95 Pages
簡介

甲一型抗胰蛋白酵素缺乏症 (AAT缺乏症)是遺傳性症狀,是由於欠缺保護肺臟之蛋白質甲一型抗胰蛋白酵素 (AAT) 所造成。主要的症狀有呼吸困難·喘鳴聲音和肺感染疾病,心跳,體重減少,視力異常等。此外主要發病因素有抽煙和暴露於塵埃,化學煙霧,感染疾病等。

甲一型抗胰蛋白酵素缺乏症 (AAT缺乏症)是遺傳性症狀,是由於欠缺保護肺臟之蛋白質甲一型抗胰蛋白酵素 (AAT) 所造成。主要的症狀有呼吸困難·喘鳴聲音和肺感染疾病,心跳,體重減少,視力異常等。此外主要發病因素有抽煙和暴露於塵埃,化學煙霧,感染疾病等。

本報告提供全球各國治療甲一型抗胰蛋白酵素缺乏症所用的開發中產品開發情形相關分析,提供您開發中產品的最新趨勢,及臨床實驗的各階段產品一覽,主要企業簡介,主要藥物概要 (產品概要,功能機制,研究開發 (R&D)的發展情形),最新的產業趨勢等調查。

簡介

  • 分析範圍

甲一型抗胰蛋白酵素缺乏症概要

治療藥的開發

  • 甲一型抗胰蛋白酵素缺乏症開發中產品:概要

各企業開發中的甲一型抗胰蛋白酵素缺乏症治療藥

開發中產品的概要

  • 後期階段的產品
  • 臨床實驗階段的產品
  • 初期階段的產品

甲一型抗胰蛋白酵素缺乏症治療藥:開發中的產品的一覽 (各企業)

甲一型抗胰蛋白酵素缺乏症開發治療藥的企業

  • 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.

甲一型抗胰蛋白酵素缺乏症:治療藥的評估

  • 單劑治療藥的情況
  • 標的別
  • 各作用機制
  • 各投藥法
  • 各分子類型

藥物簡介

甲一型抗胰蛋白酵素缺乏症治療藥:開發暫停的產品

甲一型抗胰蛋白酵素缺乏症治療藥:開發中止的產品

甲一型抗胰蛋白酵素缺乏症相關產品的開發的里程碑

  • 值得注意的最新趨勢·新聞稿

附錄

圖表一覽

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目錄
Product Code: GMDHC8435IDB

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

Table of Contents

  • Table of Contents
    • List of Tables
    • List of Figures
  • 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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