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
Editas Medicine Inc
Grifols SA
Inhibrx LP
Intellia Therapeutics Inc
International Stem Cell Corp
Kamada Ltd
Logicbio Therapeutics Inc
OncBioMune Pharmaceuticals, Inc.
PlantForm Corp
Promethera Biosciences SA
ProMetic Life Sciences Inc
Santhera Pharmaceuticals Holding AG
Vertex Pharmaceuticals Inc
Z Factor Ltd
Alpha- Antitrypsin Deficiency - Drug Profiles
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) - 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
Drug for Chronic Obstructive Pulmonary Disease and 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 A1AT 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
Gene Therapy to Activate SERPINA1 for Alpha-Antitrypsin Deficiency - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Inhibit 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
INBRX-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
LB-201 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
POL-6014 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant Alpha-1 Antitrypsin Replacement for Alpha-1 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 Molecule to Activate SERPINA1 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 - Discontinued Products
Alpha- Antitrypsin Deficiency - Product Development Milestones
Featured News & Press Releases
Mar 18, 2019: Arrowhead Pharmaceuticals files IND for pivotal phase 2/3 study of ARO-AAT for treatment of alpha-1 liver disease
Nov 05, 2018: First patient dosed in phase 2 alpha-1 antitrypsin deficiency study
Nov 01, 2018: Grifols affirms commitment to patients during Alpha-1 Awareness Month
Oct 11, 2018: Arrowhead to present late-breaking clinical data on ARO-AAT at AASLD Liver Meeting 2018
Sep 03, 2018: Arrowhead concludes dosing in Phase I ARO-AAT trial
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 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
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 2019
Number of Products under Development by Companies, H1 2019
Number of Products under Development by Companies, H1 2019 (Contd..1), H1 2019
Number of Products under Development by Universities/Institutes, H1 2019
Products under Development by Companies, H1 2019
Products under Development by Companies, H1 2019 (Contd..1), H1 2019
Products under Development by Universities/Institutes, H1 2019
Number of Products by Stage and Target, H1 2019
Number of Products by Stage and Mechanism of Action, H1 2019
Number of Products by Stage and Route of Administration, H1 2019
Number of Products by Stage and Molecule Type, H1 2019
Alpha- Antitrypsin Deficiency - Pipeline by Adverum Biotechnologies Inc, H1 2019
Alpha- Antitrypsin Deficiency - Pipeline by Alnylam Pharmaceuticals Inc, H1 2019
Alpha- Antitrypsin Deficiency - Pipeline by Applied Genetic Technologies Corp, H1 2019
Alpha- Antitrypsin Deficiency - Pipeline by Arrowhead Pharmaceuticals Inc, H1 2019
Alpha- Antitrypsin Deficiency - Pipeline by AstraZeneca Plc, H1 2019
Alpha- Antitrypsin Deficiency - Pipeline by Editas Medicine Inc, H1 2019
Alpha- Antitrypsin Deficiency - Pipeline by Grifols SA, H1 2019
Alpha- Antitrypsin Deficiency - Pipeline by Inhibrx LP, H1 2019
Alpha- Antitrypsin Deficiency - Pipeline by Intellia Therapeutics Inc, H1 2019
Alpha- Antitrypsin Deficiency - Pipeline by International Stem Cell Corp, H1 2019
Alpha- Antitrypsin Deficiency - Pipeline by Kamada Ltd, H1 2019
Alpha- Antitrypsin Deficiency - Pipeline by Logicbio Therapeutics Inc, H1 2019
Alpha- Antitrypsin Deficiency - Pipeline by OncBioMune Pharmaceuticals, Inc., H1 2019
Alpha- Antitrypsin Deficiency - Pipeline by PlantForm Corp, H1 2019
Alpha- Antitrypsin Deficiency - Pipeline by Promethera Biosciences SA, H1 2019
Alpha- Antitrypsin Deficiency - Pipeline by ProMetic Life Sciences Inc, H1 2019
Alpha- Antitrypsin Deficiency - Pipeline by Santhera Pharmaceuticals Holding AG, H1 2019
Alpha- Antitrypsin Deficiency - Pipeline by Vertex Pharmaceuticals Inc, H1 2019
Alpha- Antitrypsin Deficiency - Pipeline by Z Factor Ltd, H1 2019
Alpha- Antitrypsin Deficiency - Dormant Projects, H1 2019
Alpha- Antitrypsin Deficiency - Discontinued Products, H1 2019

List of Figures
Number of Products under Development for Alpha- Antitrypsin Deficiency, H1 2019
Number of Products under Development by Companies, H1 2019
Number of Products by Targets, H1 2019
Number of Products by Stage and Targets, H1 2019
Number of Products by Mechanism of Actions, H1 2019
Number of Products by Stage and Mechanism of Actions, H1 2019
Number of Products by Routes of Administration, H1 2019
Number of Products by Stage and Routes of Administration, H1 2019
Number of Products by Molecule Types, H1 2019
Number of Products by Stage and Molecule Types, H1 2019

Companies Mentioned
• Adverum Biotechnologies Inc
• Alnylam Pharmaceuticals Inc
• Applied Genetic Technologies Corp
• Arrowhead Pharmaceuticals Inc
• AstraZeneca Plc
• Editas Medicine Inc
• Grifols SA
• Inhibrx LP
• Intellia Therapeutics Inc
• International Stem Cell Corp
• Kamada Ltd
• Logicbio Therapeutics Inc
• OncBioMune Pharmaceuticals, Inc.
• PlantForm Corp
• Promethera Biosciences SA
• ProMetic Life Sciences Inc
• Santhera Pharmaceuticals Holding AG
• Vertex Pharmaceuticals Inc
• Z Factor Ltd