Sections
Title | Starting Page | Number of Pages |
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Table of Contents | 2 | 6 |
List of Tables | 6 | 1 |
List of Figures | 7 | 1 |
Introduction | 8 | 1 |
Global Markets Direct Report Coverage | 8 | 1 |
Alpha- Antitrypsin Deficiency Overview | 9 | 1 |
Therapeutics Development | 10 | 1 |
Pipeline Products for Alpha- Antitrypsin Deficiency Overview | 10 | 1 |
Alpha- Antitrypsin Deficiency Therapeutics under Development by Companies | 11 | 2 |
Alpha- Antitrypsin Deficiency Pipeline Products Glance | 13 | 3 |
Late Stage Products | 13 | 1 |
Clinical Stage Products | 14 | 1 |
Early Stage Products | 15 | 1 |
Alpha- Antitrypsin Deficiency Products under Development by Companies | 16 | 2 |
Alpha- Antitrypsin Deficiency Companies Involved in Therapeutics Development | 18 | 19 |
Adverum Biotechnologies, Inc. | 18 | 1 |
Alnylam Pharmaceuticals, Inc. | 19 | 1 |
Applied Genetic Technologies Corporation | 20 | 1 |
Arrowhead Pharmaceuticals, Inc. | 21 | 1 |
Carolus Therapeutics, Inc. | 22 | 1 |
Cevec Pharmaceuticals GmbH | 23 | 1 |
Dicerna Pharmaceuticals, Inc. | 24 | 1 |
Digna Biotech, S.L. | 25 | 1 |
Editas Medicine, Inc. | 26 | 1 |
Grifols, S.A. | 27 | 1 |
Inhibrx | 28 | 1 |
Intellia Therapeutics, Inc. | 29 | 1 |
International Stem Cell Corporation | 30 | 1 |
Ionis Pharmaceuticals, Inc. | 31 | 1 |
Kamada Ltd. | 32 | 1 |
Polyphor Ltd. | 33 | 1 |
ProMetic Life Sciences Inc. | 34 | 1 |
rEVO Biologics, Inc. | 35 | 1 |
Sangamo BioSciences, Inc. | 36 | 1 |
Alpha- Antitrypsin Deficiency Therapeutics Assessment | 37 | 9 |
Assessment by Monotherapy Products | 37 | 1 |
Assessment by Target | 38 | 2 |
Assessment by Mechanism of Action | 40 | 2 |
Assessment by Route of Administration | 42 | 2 |
Assessment by Molecule Type | 44 | 2 |
Drug Profiles | 46 | 32 |
ALN-AAT Drug Profile | 46 | 3 |
alpha-1 proteinase inhibitor (human) Drug Profile | 49 | 1 |
alpha-1 proteinase inhibitor (human) second generation Drug Profile | 50 | 3 |
ANN-001 Drug Profile | 53 | 2 |
ARC-AAT Drug Profile | 55 | 3 |
CT-2009 Drug Profile | 58 | 1 |
DB-027 Drug Profile | 59 | 1 |
Drugs for Alpha-1 Antitrypsin Deficiency Drug Profile | 60 | 1 |
Gene Therapy to Activate Alpha-1 Antitrypsin for Alpha-Antitrypsin Deficiency Drug Profile | 61 | 2 |
Gene Therapy to Activate Alpha-1 Antitrypsin for Alpha-Antitrypsin Deficiency Drug Profile | 63 | 1 |
ISIS-AATRx Drug Profile | 64 | 1 |
Oligonucleotides to Activate Alpha-1 Antitrypsin for Alpha-Antitrypsin Deficiency Drug Profile | 65 | 1 |
Oligonucleotides to Inhibit Alpha-1 Antitrypsin for Alpha-Antitrypsin Deficiency Drug Profile | 66 | 1 |
POL-6014 Drug Profile | 67 | 2 |
rAAV2-CB-hAAT Drug Profile | 69 | 1 |
Recombinant A1PI Drug Profile | 70 | 2 |
Recombinant Human Alpha-1 Antitrypsin Drug Profile | 72 | 1 |
Recombinant Human Alpha-1 Antitrypsin Replacement for Alpha-Antitrypsin Deficiency Drug Profile | 73 | 1 |
Recombinant Protein for Alpha-1 Antitrypsin Deficiency Drug Profile | 74 | 1 |
RNAi Oligonucleotide for Alpha-1 Antitrypsin Deficiency and Liver Disease Drug Profile | 75 | 2 |
Stem Cell Therapy for Alpha-Antitrypsin Deficiency and Crigler-Najjar Syndrome Drug Profile | 77 | 1 |
Alpha- Antitrypsin Deficiency Dormant Projects | 78 | 1 |
Alpha- Antitrypsin Deficiency Discontinued Products | 79 | 1 |
Alpha- Antitrypsin Deficiency Product Development Milestones | 80 | 14 |
Featured News &Press Releases | 80 | 1 |
May 11, 2016: Dicerna Reports Potent Preclinical Activity with GalNAc-DsiRNA-EX Conjugates | 80 | 1 |
May 05, 2016: Investigational Gene-Based Therapy for Alpha-1 Antitrypsin Deficiency Demonstrates Durable Response at Five Years | 81 | 1 |
Feb 25, 2016: Dicerna Reports Progress with GalNAc-DsiRNA-EX-Conjugates | 81 | 1 |
Jan 20, 2016: AGTC Announces Data Evaluating Novel AAV-Based Gene Therapy as a Potential Treatment for Alpha-1 Antitrypsin Deficiency | 82 | 1 |
Jan 19, 2016: Arrowhead's ARC-AAT Granted EMA Orphan Drug Designation | 83 | 1 |
Dec 08, 2015: Kamada Completes Enrollment of U.S. Phase 2 Study of Inhaled Alpha-1 Antitrypsin for the Treatment of AAT Deficiency | 83 | 1 |
Nov 09, 2015: Sangamo BioSciences Announces Publication Of Improved Method For Efficient Targeted Integration In Hematopoietic Stem Cells And T-cells | 84 | 1 |
Jul 08, 2015: Arrowhead Receives Regulatory Clearance for ARC-AAT Phase 1 in the United Kingdom and New Zealand | 85 | 1 |
Jul 02, 2015: Arrowhead Initiates Dosing of ARC-AAT in Patients with Alpha-1 Antitrypsin Deficiency | 85 | 1 |
Jun 10, 2015: Arrowhead Receives Orphan Drug Designation for ARC-AAT | 86 | 1 |
May 20, 2015: Kamada Reports Updated Data from European Phase 2/3 Clinical Study of Inhaled Alpha-1 Antitrypsin to Treat AAT Deficiency | 86 | 2 |
May 05, 2015: Arrowhead Completes Dosing Healthy Volunteers and Initiates Transition to Patients in Phase 1 Study of ARC-AAT | 88 | 1 |
Apr 20, 2015: Kamada Reports Additional Data from European Phase 2/3 Clinical Study of Inhaled Alpha-1 Antitrypsin to Treat AAT Deficiency | 89 | 3 |
Feb 23, 2015: Arrowhead Begins Phase 1 Trial of ARC-AAT for Treatment of Liver Disease Associated with Alpha-1 Antitrypsin Deficiency | 92 | 1 |
Jan 07, 2015: Kamada Reports Encouraging Discussions with European Co-Rapporteurs Regarding European Filing for Inhaled Alpha-1 Antitrypsin to Treat AATD | 93 | 1 |
Appendix | 94 | 2 |
Methodology | 94 | 1 |
Coverage | 94 | 1 |
Secondary Research | 94 | 1 |
Primary Research | 94 | 1 |
Expert Panel Validation | 94 | 1 |
Contact Us | 94 | 1 |
Disclaimer | 95 | 1 |