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Alpha-1 Antitrypsin Deficiency Clinical Trials and Studies: EMA, PDMA, FDA Approvals, Mechanism of Action, ROA, NDA, IND, and Companies by DelveInsight

Alpha-1 Antitrypsin Deficiency Clinical Trials and Studies: EMA, PDMA, FDA Approvals, Mechanism of Action, ROA, NDA, IND, and Companies by DelveInsight

Globe and Mail12-02-2025
DelveInsight's, ' Alpha 1 Antitrypsin Deficiency Pipeline Insight ' report provides comprehensive insights about 12+ companies and 12+ pipeline drugs in Alpha 1 Antitrypsin Deficiency pipeline landscape. It covers the Alpha 1 Antitrypsin Deficiency pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Alpha 1 Antitrypsin Deficiency therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive Alpha 1 Antitrypsin Deficiency pipeline products in this space.
Stay ahead with the latest insights! Download DelveInsight's comprehensive Alpha 1 Antitrypsin Deficiency Pipeline Report to explore emerging therapies, key Alpha 1 Antitrypsin Deficiency Companies, and future Alpha 1 Antitrypsin Deficiency treatment landscapes @ Alpha 1 Antitrypsin Deficiency Pipeline Outlook Report
Key Takeaways from the Alpha 1 Antitrypsin Deficiency Pipeline Report
In February 2025:- Grifols Therapeutics Inc.:- The purpose of this study is to evaluate the safety and tolerability of 72 milligrams per kilogram (mg/kg) and 180 mg/kg Alpha-1 15%, administered as a single-dose subcutaneous (SC) infusion and subsequently as weekly SC infusions over 8 weeks in participants with Alpha1-Antitrypsin Deficiency (AATD).
In January 2025:- Takeda:- The liver produces a protein called alpha-1 antitrypsin (AAT). AAT is normally released into the bloodstream. In some people, the liver makes an abnormal version of the AAT protein, called Z-AAT. Making an abnormal version of the AAT protein can result in liver disease as Z-AAT builds up in liver cells, which leads to liver problems such as liver scarring (fibrosis), continuing liver damage (cirrhosis), and eventually endstage liver disease.
In January 2025:- Korro Bio Inc.:- The purpose of this first-in human (FIH) study is to assess the safety, tolerability, and pharmacokinetics (PK) of single and multiple doses of KRRO-110 in both healthy adult participants and in clinically stable patients with Alpha-1 antitrypsin deficiency (AATD).
In January 2025:- Arrowhead Pharmaceuticals: - The purpose of AROAAT2001 (SEQUOIA) is to evaluate the safety, efficacy and tolerability of multiple doses of the investigational product, Fazirsiran Injection, administered subcutaneously to participants with alpha-1 antitrypsin deficiency (AATD).
In January 2025:- Kamada Ltd:- The goal of this clinical trial is to learn if AAT for inhalation, at a dose of 80 mg/day can slow the progression of lung disease in people who have lung disease caused by severe genetic deficiency in Alpha 1 Antitrypsin (AATD).
DelveInsight's Alpha 1 Antitrypsin Deficiency pipeline report depicts a robust space with 12+ active players working to develop 12+ pipeline therapies for Alpha 1 Antitrypsin Deficiency treatment.
The leading Alpha 1 Antitrypsin Deficiency Companies such as Octapharma, Dicerna Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, Vertex Pharmaceuticals, AstraZeneca, Kamada Ltd and others.
Promising Alpha-1 Antitrypsin Deficiency Pipeline Therapies such as Fazirsiran Injection, Aerosolized, Recombinant Alpha 1-Antitrypsin, INBRX-101/rhAAT-Fc, ADVM-043, and others.
Discover how the Alpha 1 Antitrypsin Deficiency treatment paradigm is evolving. Access DelveInsight's in-depth Alpha 1 Antitrypsin Deficiency Pipeline Analysis for a closer look at promising breakthroughs @ Alpha 1 Antitrypsin Deficiency Clinical Trials and Studies
Alpha 1 Antitrypsin Deficiency Emerging Drugs Profile
ARO AAT: Arrowhead Pharmaceuticals
ARO AAT is a second generation, N-Acetylgalactosamine (GalNAc) based, subcutaneously administered RNA interference based therapeutic. ARO-AAT is designed to knock down the hepatic production of the mutant alpha-1 antitrypsin (Z-AAT) protein, the cause of progressive liver disease in AATD patients. In June 2019, the US FDA has granted Fast Track designation to ARO-AAT. ARO-AAT is in Phase III clinical studies for the treatment of liver disease associated with alpha-1 antitrypsin deficiency (AATD).
ALN-AAT02: Alnylam Pharmaceuticals
ALN AAT02 is a subcutaneously administered RNAi therapeutic that targets alpha-1 antitrypsin (AAT). ALN-AAT02 is based on the Alnylam's enhanced stabilization chemistry plus (ESC+)-GalNAc-conjugate technology, which enables subsequent subcutaneous dosing with increased selectivity and a wide therapeutic index. The drug is currently in Phase I/II clinical development for the treatment of ZZ Type AAT Deficiency Liver Disease.
The Alpha 1 Antitrypsin Deficiency pipeline report provides insights into
The report provides detailed insights about companies that are developing therapies for the treatment of Alpha 1 Antitrypsin Deficiency with aggregate therapies developed by each company for the same.
It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Alpha 1 Antitrypsin Deficiency Treatment.
Alpha 1 Antitrypsin Deficiency Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
Alpha 1 Antitrypsin Deficiency Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Alpha 1 Antitrypsin Deficiency market.
Get a detailed analysis of the latest innovations in the Alpha 1 Antitrypsin Deficiency pipeline. Explore DelveInsight's expert-driven report today! @ Alpha 1 Antitrypsin Deficiency Unmet Needs
Alpha 1 Antitrypsin Deficiency Companies
Octapharma, Dicerna Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, Vertex Pharmaceuticals, AstraZeneca, Kamada Ltd and others.
Alpha 1 Antitrypsin Deficiency pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
Intra-articular
Intraocular
Intrathecal
Intravenous
Ophthalmic
Oral
Parenteral
Subcutaneous
Topical
Transdermal
Alpha 1 Antitrypsin Deficiency Products have been categorized under various Molecule types such as
Oligonucleotide
Peptide
Small molecule
Download DelveInsight's latest report to gain strategic insights into upcoming Alpha 1 Antitrypsin Deficiency Therapies and key Alpha 1 Antitrypsin Deficiency Developments @ Alpha 1 Antitrypsin Deficiency Market Drivers and Barriers, and Future Perspectives
Scope of the Alpha 1 Antitrypsin Deficiency Pipeline Report
Coverage- Global
Alpha 1 Antitrypsin Deficiency Companies- Octapharma, Dicerna Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, Vertex Pharmaceuticals, AstraZeneca, Kamada Ltd and others.
Alpha-1 Antitrypsin Deficiency Pipeline Therapies - Fazirsiran Injection, Aerosolized, Recombinant Alpha 1-Antitrypsin, INBRX-101/rhAAT-Fc, ADVM-043, and others.
Alpha 1 Antitrypsin Deficiency Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
Alpha 1 Antitrypsin Deficiency Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
Which companies are leading the race in Alpha 1 Antitrypsin Deficiency drug development? Find out in DelveInsight's exclusive Alpha 1 Antitrypsin Deficiency Pipeline Report—access it now! @ Alpha 1 Antitrypsin Deficiency Emerging Drugs and Major Companies
Table of Content
Introduction
Alpha-1 Antitrypsin Deficiency Executive Summary
Alpha-1 Antitrypsin Deficiency: Overview
Alpha-1 Antitrypsin Deficiency Pipeline Therapeutics
Alpha-1 Antitrypsin Deficiency Pipeline Therapeutic Assessment
Alpha-1 Antitrypsin Deficiency– DelveInsight's Analytical Perspective
Late Stage Products (Phase III)
ARO AAT: Arrowhead Pharmaceuticals
Drug profiles in the detailed report…..
Mid Stage Products (Phase II)
Drug Name: Company Name
Drug profiles in the detailed report…..
Early Stage Products (Phase I/II)
ALN-AAT02: Alnylam Pharmaceuticals
Drug profiles in the detailed report…..
Preclinical and Discovery Stage Products
Drug Name: Company Name
Drug profiles in the detailed report…..
Inactive Products
Alpha-1 Antitrypsin Deficiency Key Companies
Alpha-1 Antitrypsin Deficiency Key Products
Alpha-1 Antitrypsin Deficiency- Unmet Needs
Alpha-1 Antitrypsin Deficiency- Market Drivers and Barriers
Alpha-1 Antitrypsin Deficiency- Future Perspectives and Conclusion
Alpha-1 Antitrypsin Deficiency Analyst Views
Alpha-1 Antitrypsin Deficiency Key Companies
Appendix
About Us
DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve.
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Company Name: DelveInsight Business Research LLP
Contact Person: Yash Bhardwaj
Email: Send Email
Phone: 9650213330
Address: 304 S. Jones Blvd #2432
City: Las Vegas
State: Nevada
Country: United States
Website: https://www.delveinsight.com/report-store/surgical-lasers-market
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Products have been categorized under various ROAs such as Oral Intravenous Subcutaneous Parenteral Topical ER+/ HER2 -ve Breast Cancer Products have been categorized under various Molecule types such as Recombinant fusion proteins Small molecule Monoclonal antibody Peptide Polymer Gene therapy Download DelveInsight's latest report to gain strategic insights into upcoming ER+/ HER2 -ve Breast Cancer Therapies and key developments @ ER+/ HER2 -ve Breast Cancer Market Drivers and Barriers, and Future Perspectives Scope of the ER+/ HER2 -ve Breast Cancer Pipeline Report Coverage- Global ER+/ HER2 -ve Breast Cancer Companies- AstraZeneca, Atossa Therapeutics, Inc., Accutar Biotechnology Inc, VelosBio Inc, Merus N.V., Pfizer, Olema Pharmaceuticals, Inc., Eli Lilly and Company, Novartis Pharmaceuticals, Hoffmann-La Roche, Ellipses Pharma, BeiGene and others. ER+/ HER2 -ve Breast Cancer Therapies- Entinostat, Aromatase Inhibitor (AI) Therapy, cetuximab, cisplatin, Palbociclib 125Mg Tab and others. ER+/ HER2 -ve Breast Cancer Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination ER+/ HER2 -ve Breast Cancer Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Which companies are leading the race in ER+/ HER2 -ve Breast Cancer drug development? Find out in DelveInsight's exclusive pipeline Report—access it now! @ ER+/ HER2 -ve Breast Cancer Emerging Drugs and Major Companies Table of Content Introduction Executive Summary ER+/ HER2-VE Breast Cancer: Overview Pipeline Therapeutics Therapeutic Assessment ER positive, HER2 negative Breast Cancer– DelveInsight's Analytical Perspective Late Stage Products (Phase III) Camizestrant: AstraZeneca Drug profiles in the detailed report….. Mid Stage Products (Phase II) (Z)-endoxifen: Atossa Therapeutics, Inc. Drug profiles in the detailed report….. Early Stage Products (Phase I) AC699: Accutar Biotechnology Inc Drug profiles in the detailed report….. Preclinical and Discovery Stage Products Drug Name: Company Name Drug profiles in the detailed report….. Inactive Products ER+/ HER2-VE Breast Cancer Key Companies ER+/ HER2-VE Breast Cancer Key Products ER+/ HER2-VE Breast Cancer- Unmet Needs ER+/ HER2-VE Breast Cancer- Market Drivers and Barriers ER+/ HER2-VE Breast Cancer- Future Perspectives and Conclusion ER+/ HER2-VE Breast Cancer Analyst Views ER+/ HER2-VE Breast Cancer Key Companies Appendix About Us DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve. Media Contact Company Name: DelveInsight Business Research LLP Contact Person: Yash Bhardwaj Email: Send Email Phone: 09650213330 Address: 304 S. Jones Blvd #2432 City: Las Vegas State: NV Country: United States Website:

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