
Alpha-1 Antitrypsin Deficiency (A1AD) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
Description
Alpha-1 Antitrypsin Deficiency (A1AD) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update
Summary
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Alpha-1 Antitrypsin Deficiency (A1AD) - Drugs In Development, 2022, provides an overview of the Alpha-1 Antitrypsin Deficiency (A1AD) (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-1 Antitrypsin Deficiency (A1AD) - Drugs In Development, 2022, provides comprehensive information on the therapeutics under development for Alpha-1 Antitrypsin Deficiency (A1AD) (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-1 Antitrypsin Deficiency (A1AD) (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Alpha-1 Antitrypsin Deficiency (A1AD) 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under Development by Companies /Universities /Institutes, the molecules developed by Companies in Filing rejected/Withdrawn, Phase III, Phase II, Phase I, Preclinical, Discovery and Unknown stages are 1, 1, 7, 2, 24, 5 and 2 respectively.
Alpha-1 Antitrypsin Deficiency (A1AD) (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
Summary
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Alpha-1 Antitrypsin Deficiency (A1AD) - Drugs In Development, 2022, provides an overview of the Alpha-1 Antitrypsin Deficiency (A1AD) (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-1 Antitrypsin Deficiency (A1AD) - Drugs In Development, 2022, provides comprehensive information on the therapeutics under development for Alpha-1 Antitrypsin Deficiency (A1AD) (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-1 Antitrypsin Deficiency (A1AD) (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Alpha-1 Antitrypsin Deficiency (A1AD) 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, 7, 2, 24, 5 and 2 respectively.
Alpha-1 Antitrypsin Deficiency (A1AD) (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-1 Antitrypsin Deficiency (A1AD) (Genetic Disorders).
- The pipeline guide reviews pipeline therapeutics for Alpha-1 Antitrypsin Deficiency (A1AD) (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-1 Antitrypsin Deficiency (A1AD) (Genetic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Alpha-1 Antitrypsin Deficiency (A1AD) (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-1 Antitrypsin Deficiency (A1AD) (Genetic Disorders)
- 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-1 Antitrypsin Deficiency (A1AD) (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-1 Antitrypsin Deficiency (A1AD) (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.
Table of Contents
119 Pages
- Introduction
- Global Markets Direct Report Coverage
- Alpha-1 Antitrypsin Deficiency (A1AD) – Overview
- Alpha-1 Antitrypsin Deficiency (A1AD) – Therapeutics Development
- Pipeline Overview
- Pipeline by Companies
- Products under Development by Companies
- Alpha-1 Antitrypsin Deficiency (A1AD) – Therapeutics Assessment
- Assessment by Target
- Assessment by Mechanism of Action
- Assessment by Route of Administration
- Assessment by Molecule Type
- Alpha-1 Antitrypsin Deficiency (A1AD) – Companies Involved in Therapeutics Development
- ADARx Pharmaceuticals Inc
- Alnylam Pharmaceuticals Inc
- Apic Bio Inc
- Applied Genetic Technologies Corp
- Arrowhead Pharmaceuticals Inc
- Beam Therapeutics Inc
- BioMarin Pharmaceutical Inc
- Caravella Biopharma SA
- Centessa Pharmaceuticals Plc
- Editas Medicine Inc
- Evolve Biologics Inc
- Graphite Bio Inc
- Grifols SA
- Hepatx Corp
- Inhibrx Inc
- Intellia Therapeutics Inc
- International Stem Cell Corp
- Kamada Pharmaceuticals
- Korro Bio Inc
- Krystal Biotech Inc
- LEXEO Therapeutics LLC
- Liminal BioSciences Inc
- Logicbio Therapeutics Inc
- Mereo Biopharma Group Plc
- Moderna Inc
- Novo Nordisk AS
- pH Pharma Co Ltd
- PlantForm Corp
- Promethera Biosciences SA
- Renovion Inc
- Santhera Pharmaceuticals Holding AG
- Shape Therapeutics Inc
- Takeda Pharmaceutical Co Ltd
- Vera Therapeutics Inc
- Vertex Pharmaceuticals Inc
- Wave Life Sciences Ltd
- Alpha-1 Antitrypsin Deficiency (A1AD) – Drug Profiles
- ALNAAT-02 – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Alpha-1 Antitrypsin Deficiency – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- alpha-1 proteinase inhibitor (human) – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- alpha-1 proteinase inhibitor (human) second generation – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- alvelestat – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- APB-101 – Drug Profile
- Product Description
- Mechanism Of Action
- ascorbic acid – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- belcesiran – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Blood Derivative for Alpha-1 Antitrypsin Deficiency – Drug Profile
- Product Description
- Mechanism Of Action
- BMN-349 – Drug Profile
- Product Description
- Mechanism Of Action
- fazirsiran sodium – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Gene Therapies for Genetic Disorders and Primary Hyperoxaluria – Drug Profile
- Product Description
- Mechanism Of Action
- Gene Therapy for Alpha-1 Antitrypsin Deficiency – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Gene Therapy to Activate Alpha 1 Antitrypsin for Alpha- Antitrypsin Deficiency – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- H2Stem – Drug Profile
- Product Description
- Mechanism Of Action
- HX-003 – Drug Profile
- Product Description
- Mechanism Of Action
- INBRX-101 – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- KB-408 – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- LB-201 – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- LEX-01 – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- lonodelestat – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- nedosiran sodium – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- NTLA-2003 – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- NTLA-3001 – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Oligonucleotide to Activate SERPINA1 for Alpha 1 Antitrypsin Deficiency – Drug Profile
- Product Description
- Mechanism Of Action
- Oligonucleotides for Alpha-1 Antitrypsin Deficiency – Drug Profile
- Product Description
- Mechanism Of Action
- Oligonucleotides for Genetic Disorders – Drug Profile
- Product Description
- Mechanism Of Action
- Oligonucleotides to Activate SERPINA 1 for Alpha-1 Antitrypsin Deficiency – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Oligonucleotides to Activate SERPINA1 for Alpha-1 Antitrypsin Deficiency – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- PHP-303 – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Recombinant Alpha-1 Antitrypsin Replacement for Alpha-1 Antitrypsin Deficiency – Drug Profile
- Product Description
- Mechanism Of Action
- SHP-221 – Drug Profile
- Product Description
- Mechanism Of Action
- Small Molecules to Activate Alpha 1 Antitrypsin for Alpha-1 Antitrypsin Deficiency – Drug Profile
- Product Description
- Mechanism Of Action
- Stem Cell Therapy for Alpha-Antitrypsin Deficiency, Crigler-Najjar Syndrome and Liver Cirrhosis – Drug Profile
- Product Description
- Mechanism Of Action
- Therapeutic Protein Production (alpha-globin) – Drug Profile
- Product Description
- Mechanism Of Action
- VX-864 – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- ZF-874 – Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- ZF-887 – Drug Profile
- Product Description
- Mechanism Of Action
- Alpha-1 Antitrypsin Deficiency (A1AD) – Dormant Projects
- Alpha-1 Antitrypsin Deficiency (A1AD) – Discontinued Products
- Alpha-1 Antitrypsin Deficiency (A1AD) – Product Development Milestones
- Featured News & Press Releases
- Apr 05, 2022: Inhibrx to present INBRX-101 data at ATS 2022 Annual Meeting
- Mar 04, 2022: US FDA grants orphan drug status to Inhibrx’s AATD treatment
- Mar 03, 2022: Inhibrx receives FDA Orphan-Drug Designation for INBRX-101 for the treatment of Alpha-1 Antitrypsin Deficiency
- Mar 03, 2022: Mereo BioPharma to hold virtual R&D day on monday, march 14, 2022
- Nov 12, 2021: Arrowhead presents additional clinical data on investigational ARO-AAT Treatment at AASLD Liver Meeting
- Nov 12, 2021: Dicerna Presents data from phase 1 trial of belcesiran at American Association for the Study of Liver Diseases (AASLD) The Liver Meeting 2021
- Nov 01, 2021: Centessa Pharmaceuticals demonstrates proof-of-mechanism from first three PiMZ subjects dosed in part B of phase 1 study evaluating ZF874
- Oct 26, 2021: Mereo BioPharma receives U.S. Orphan Drug Designation for alvelestat in the treatment of alpha-1 antitrypsin deficiency
- Oct 14, 2021: Krystal Biotech announces virtual presentation of pre-clinical data on KB408 for the treatment of alpha-1 antitrypsin deficiency at ESGCT
- Oct 14, 2021: Dicerna Announces Poster Presentation on Belcesiran at American Association for the Study of Liver Diseases (AASLD) The Liver Meeting® 2021 in November
- Oct 12, 2021: INBRX-101 shows favorable safety profile in patients with alpha-1 antitrypsin deficiency and demonstrates the potential to achieve normal alpha-1 antitrypsin levels with monthly dosing
- Oct 11, 2021: Inhibrx to host webcast presentation of interim results from the phase 1 trial of INBRX-101, its recombinant human AAT-Fc protein for the treatment of alpha-1 antitrypsin deficiency
- Sep 28, 2021: Wave Life Sciences announces new data for leading RNA editing capability across multiple tissues and provides update on AATD program during analyst and investor research webcast
- Jul 29, 2021: Arrowhead Pharmaceuticals receives Breakthrough Therapy designation from U.S. FDA for ARO-AAT for the treatment of alpha-1 antitrypsin deficiency associated liver disease
- Jul 21, 2021: Dicerna announces interim results from phase 1 trial of Belcesiran for treatment of alpha-1 antitrypsin deficiency-associated liver disease
- Appendix
- Methodology
- Coverage
- Secondary Research
- Primary Research
- Expert Panel Validation
- Contact Us
- Disclaimer
- List of Tables
- Table 1: Number of Products under Development for Alpha-1 Antitrypsin Deficiency (A1AD), 2022
- Table 2: Number of Products under Development by Companies, 2022
- Table 3: Number of Products under Development by Companies, 2022 (Contd..1)
- Table 4: Products under Development by Companies, 2022
- Table 5: Products under Development by Companies, 2022 (Contd..1)
- Table 6: Products under Development by Companies, 2022 (Contd..2)
- Table 7: Number of Products by Stage and Target, 2022
- Table 8: Number of Products by Stage and Mechanism of Action, 2022
- Table 9: Number of Products by Stage and Route of Administration, 2022
- Table 10: Number of Products by Stage and Molecule Type, 2022
- Table 11: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by ADARx Pharmaceuticals Inc, 2022
- Table 12: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Alnylam Pharmaceuticals Inc, 2022
- Table 13: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Apic Bio Inc, 2022
- Table 14: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Applied Genetic Technologies Corp, 2022
- Table 15: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Arrowhead Pharmaceuticals Inc, 2022
- Table 16: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Beam Therapeutics Inc, 2022
- Table 17: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by BioMarin Pharmaceutical Inc, 2022
- Table 18: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Caravella Biopharma SA, 2022
- Table 19: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Centessa Pharmaceuticals Plc, 2022
- Table 20: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Editas Medicine Inc, 2022
- Table 21: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Evolve Biologics Inc, 2022
- Table 22: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Graphite Bio Inc, 2022
- Table 23: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Grifols SA, 2022
- Table 24: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Hepatx Corp, 2022
- Table 25: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Inhibrx Inc, 2022
- Table 26: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Intellia Therapeutics Inc, 2022
- Table 27: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by International Stem Cell Corp, 2022
- Table 28: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Kamada Pharmaceuticals, 2022
- Table 29: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Korro Bio Inc, 2022
- Table 30: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Krystal Biotech Inc, 2022
- Table 31: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by LEXEO Therapeutics LLC, 2022
- Table 32: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Liminal BioSciences Inc, 2022
- Table 33: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Logicbio Therapeutics Inc, 2022
- Table 34: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Mereo Biopharma Group Plc, 2022
- Table 35: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Moderna Inc, 2022
- Table 36: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Novo Nordisk AS, 2022
- Table 37: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by pH Pharma Co Ltd, 2022
- Table 38: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by PlantForm Corp, 2022
- Table 39: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Promethera Biosciences SA, 2022
- Table 40: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Renovion Inc, 2022
- Table 41: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Santhera Pharmaceuticals Holding AG, 2022
- Table 42: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Shape Therapeutics Inc, 2022
- Table 43: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Takeda Pharmaceutical Co Ltd, 2022
- Table 44: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Vera Therapeutics Inc, 2022
- Table 45: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Vertex Pharmaceuticals Inc, 2022
- Table 46: Alpha-1 Antitrypsin Deficiency (A1AD) – Pipeline by Wave Life Sciences Ltd, 2022
- Table 47: Alpha-1 Antitrypsin Deficiency (A1AD) – Dormant Projects, 2022
- Table 48: Alpha-1 Antitrypsin Deficiency (A1AD) – Discontinued Products, 2022
- List of Figures
- Figure 1: Number of Products under Development for Alpha-1 Antitrypsin Deficiency (A1AD), 2022
- Figure 2: Number of Products under Development by Companies, 2022
- Figure 3: Number of Products by Targets, 2022
- Figure 4: Number of Products by Stage and Targets, 2022
- Figure 5: Number of Products by Mechanism of Actions, 2022
- Figure 6: Number of Products by Stage and Mechanism of Actions, 2022
- Figure 7: Number of Products by Routes of Administration, 2022
- Figure 8: Number of Products by Stage and Top 10 Routes of Administration, 2022
- Figure 9: Number of Products by Molecule Types, 2022
- Figure 10: Number of Products by Stage and Top 10 Molecule Types, 2022
Pricing
Currency Rates
Questions or Comments?
Our team has the ability to search within reports to verify it suits your needs. We can also help maximize your budget by finding sections of reports you can purchase.