α1-アンチトリプシン欠乏症治療薬の世界市場:パイプラインレビュー(2018下半期)

【英語タイトル】Alpha- Antitrypsin Deficiency - Pipeline Review, H2 2018

Global Markets Directが出版した調査資料(DATA906A014)・商品コード:DATA906A014
・発行会社(調査会社):Global Markets Direct
・発行日:2018年8月21日
・ページ数:83
・レポート言語:英語
・レポート形式:PDF
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【レポートの概要】

Alpha- Antitrypsin Deficiency – Pipeline Review, H2 2018
Summary

Global Markets Direct’s latest Pharmaceutical and Healthcare disease pipeline guide Alpha- Antitrypsin Deficiency – Pipeline Review, H2 2018, 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, H2 2018, 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under Development by Companies /Universities /Institutes, the molecules developed by Companies in Filing rejected/Withdrawn, Phase III, Phase II, Phase I, Preclinical, Discovery and Unknown stages are 1, 1, 3, 5, 12, 2 and 1 respectively.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 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. 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 it’s 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.

【レポートの目次】

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
Applied Genetic Technologies Corp
Arrowhead Pharmaceuticals Inc
AstraZeneca Plc
Carolus Therapeutics Inc
Editas Medicine Inc
Grifols SA
Inhibrx LP
Intellia Therapeutics Inc
International Stem Cell Corp
Kamada Ltd
Octapharma AG
OncBioMune Pharmaceuticals Inc
PlantForm Corp
Promethera Biosciences SA
ProMetic Life Sciences Inc
rEVO Biologics Inc
Santhera Pharmaceuticals Holding AG
Therapure Biopharma Inc
Z Factor Ltd
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
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
alvelestat – Drug Profile
Product Description
Mechanism Of Action
R&D Progress
APB-101 – 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
Drug for Chronic Obstructive Pulmonary Disease and Alpha-1 Antitrypsin Deficiency – Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Fusion Protein for Alpha-1 Antitrypsin Deficiency – Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy 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
H2Stem – Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotides to Activate SERPINA1 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
Recombinant Human Alpha-1 Antitrypsin – 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 Activate SERPINA1 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
Jul 10, 2018: Kamada Receives Positive Scientific Advice from European Medicines Agency on a New Phase 3 Study Design for Inhaled AAT
Jun 29, 2018: Arrowhead reports initial positive results from AROAAT1001 trial
Jun 27, 2018: Arrowhead Receives Positive EMA Opinion on Orphan Designation for ARO-AAT for Treatment of Congenital Alpha-1 Antitrypsin Deficiency
Mar 12, 2018: Arrowhead Begins Dosing in Phase 1 Study of ARO-AAT for Treatment of Alpha-1 Liver Disease
Feb 15, 2018: Arrowhead Pharmaceuticals Receives Orphan Drug Designation for ARO-AAT
Dec 20, 2017: Arrowhead Pharmaceuticals Files for Regulatory Clearance to Begin Phase 1 Study of ARO-AAT
Oct 23, 2017: Arrowhead Presents Promising Preclinical Data on Development of ARO-AAT for Treatment of Alpha-1 Liver Disease at Liver Meeting 2017
Oct 10, 2017: Arrowhead Pharmaceuticals to Present Preclinical Data on ARO-AAT at The Liver Meeting
Sep 25, 2017: Alnylam Continues Leadership in RNAi Technologies and Delivery with New Pre-Clinical Data Presented on “Enhanced Stabilization Chemistry Plus” GalNAc-siRNA Conjugate Platform at 13th Annual Meeting of the Oligonucleotide Therapeutics Society
Sep 22, 2017: FDA approves Grifols Prolastin-C Liquid [alpha-1 proteinase inhibitor, liquid] for the treatment of alpha-1 antitrypsin deficiency
Aug 22, 2017: Apic Bio Launches to Advance First-in-Class Gene Therapy for Treatment of Alpha-1 Antitrypsin Deficiency
Jul 20, 2017: Kamada Submits Proposed Phase 3 Protocol to FDA for Inhaled Alpha-1 Antitrypsin for Treatment of Alpha-1 Antitrypsin Deficiency Disease
Jun 22, 2017: Kamada Announces Withdrawal of European Marketing Authorization Application for Inhaled Alpha-1 Antitrypsin for Treatment of Alpha-1 Antitrypsin Deficiency Disease
Jun 08, 2017: Data Published in Molecular Therapy Demonstrate Five-Year Response Following a Single Dose of an Investigational Gene-Based Therapy for Alpha-1 Antitrypsin (AAT) Deficiency
May 24, 2017: Kamada Presents Updated Data from Phase 2 Clinical Trial of Inhaled Alpha-1-Antitrypsin for Treatment of Alpha-1 Antitrypsin Deficiency at 2017 American Thoracic Society International Conference
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 2018
Number of Products under Development by Companies, H2 2018
Number of Products under Development by Companies, H2 2018 (Contd..1), H2 2018
Number of Products under Development by Universities/Institutes, H2 2018
Products under Development by Companies, H2 2018
Products under Development by Companies, H2 2018 (Contd..1), H2 2018
Products under Development by Universities/Institutes, H2 2018
Number of Products by Stage and Target, H2 2018
Number of Products by Stage and Mechanism of Action, H2 2018
Number of Products by Stage and Route of Administration, H2 2018
Number of Products by Stage and Molecule Type, H2 2018
Alpha- Antitrypsin Deficiency - Pipeline by Adverum Biotechnologies Inc, H2 2018
Alpha- Antitrypsin Deficiency - Pipeline by Alnylam Pharmaceuticals Inc, H2 2018
Alpha- Antitrypsin Deficiency - Pipeline by Applied Genetic Technologies Corp, H2 2018
Alpha- Antitrypsin Deficiency - Pipeline by Arrowhead Pharmaceuticals Inc, H2 2018
Alpha- Antitrypsin Deficiency - Pipeline by AstraZeneca Plc, H2 2018
Alpha- Antitrypsin Deficiency - Pipeline by Carolus Therapeutics Inc, H2 2018
Alpha- Antitrypsin Deficiency - Pipeline by Editas Medicine Inc, H2 2018
Alpha- Antitrypsin Deficiency - Pipeline by Grifols SA, H2 2018
Alpha- Antitrypsin Deficiency - Pipeline by Inhibrx LP, H2 2018
Alpha- Antitrypsin Deficiency - Pipeline by Intellia Therapeutics Inc, H2 2018
Alpha- Antitrypsin Deficiency - Pipeline by International Stem Cell Corp, H2 2018
Alpha- Antitrypsin Deficiency - Pipeline by Kamada Ltd, H2 2018
Alpha- Antitrypsin Deficiency - Pipeline by Octapharma AG, H2 2018
Alpha- Antitrypsin Deficiency - Pipeline by OncBioMune Pharmaceuticals Inc, H2 2018
Alpha- Antitrypsin Deficiency - Pipeline by PlantForm Corp, H2 2018
Alpha- Antitrypsin Deficiency - Pipeline by Promethera Biosciences SA, H2 2018
Alpha- Antitrypsin Deficiency - Pipeline by ProMetic Life Sciences Inc, H2 2018
Alpha- Antitrypsin Deficiency - Pipeline by rEVO Biologics Inc, H2 2018
Alpha- Antitrypsin Deficiency - Pipeline by Santhera Pharmaceuticals Holding AG, H2 2018
Alpha- Antitrypsin Deficiency - Pipeline by Therapure Biopharma Inc, H2 2018
Alpha- Antitrypsin Deficiency - Pipeline by Z Factor Ltd, H2 2018
Alpha- Antitrypsin Deficiency - Dormant Projects, H2 2018
Alpha- Antitrypsin Deficiency - Discontinued Products, H2 2018

List of Figures
Number of Products under Development for Alpha- Antitrypsin Deficiency, H2 2018
Number of Products under Development by Companies, H2 2018
Number of Products by Targets, H2 2018
Number of Products by Stage and Targets, H2 2018
Number of Products by Mechanism of Actions, H2 2018
Number of Products by Stage and Mechanism of Actions, H2 2018
Number of Products by Routes of Administration, H2 2018
Number of Products by Stage and Routes of Administration, H2 2018
Number of Products by Molecule Types, H2 2018
Number of Products by Stage and Molecule Types, H2 2018

【掲載企業】

Adverum Biotechnologies Inc、Alnylam Pharmaceuticals Inc、Applied Genetic Technologies Corp、Arrowhead Pharmaceuticals Inc、AstraZeneca Plc、Carolus Therapeutics Inc、Editas Medicine Inc、Grifols SA、Inhibrx LP、Intellia Therapeutics Inc、International Stem Cell Corp、Kamada Ltd、Octapharma AG、OncBioMune Pharmaceuticals Inc、PlantForm Corp、Promethera Biosciences SA、ProMetic Life Sciences Inc、rEVO Biologics Inc、Santhera Pharmaceuticals Holding AG、Therapure Biopharma Inc、Z Factor Ltd

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