DelveInsight’s, “Alpha1-Antitrypsin Deficiency Pipeline Insight 2023” report provides comprehensive insights about 12+ companies and 12+ pipeline drugs in the Alpha1-Antitrypsin Deficiency pipeline landscape. It covers the Alpha1-Antitrypsin Deficiency pipeline drug profiles, including Alpha1-Antitrypsin Deficiency clinical trials and nonclinical stage products. It also covers the Alpha1-Antitrypsin Deficiency therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
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Key Takeaways from the Alpha-1 Antitrypsin Deficiency Pipeline Report
- 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.
- The leading Alpha-1 Antitrypsin Deficiency Companies include Octapharma, Dicerna Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, Vertex Pharmaceuticals, AstraZeneca, Kamada, Ltd, and Others.
- Promising Alpha-1 Antitrypsin Deficiency Pipeline Therapies include Fazirsiran Injection, Alpha-1 MP, ARO AAT, ALN-AAT02, and others
- In March 2023, Takeda initiated a randomized, double-blind, placebo-controlled, Phase III study to evaluate theefficacy and safety of Fazirsiran in the treatment of Alpha-1 Antitrypsin Deficiency-Associated liver disease with METAVIR StageF2 to F4 fibrosis. The main aim of this study is to learn if fazirsiran reduces liver scarring (fibrosis) compared to placebo. Otheraims are to learn if fazirsiran slows down the disease worsening in the liver, to get information on how fazirsiran affects thebody (called pharmacodynamics), to learn if fazirsiran reduces other liver injury (inflammation) and the abnormal Z-AAT proteinin the liver, to get information on how the body processes fazirsiran (called pharmacokinetics), to test how well fazirsiran workscompared with a placebo in improving measures of liver scarring including imaging and liver biomarkers (substances in theblood that the body normally makes and help show if liver function is improving, staying the same, or getting worse) as well asto check for side effects in participants treated with fazirsiran compared with those who received placebo. The trial is currentlyrecruiting participants and is expected to be completed by March 2027, with an estimated enrollment of 160 participants.
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Alpha-1 Antitrypsin Deficiency Overview
Alpha-1-antitrypsin (AAT) is a protein produced in the liver that protects the body's tissues from being damaged by infection-fighting agents released by its immune system. In alpha-1 antitrypsin deficiency, the body’s normal production of AAT is reduced, resulting in the destruction of sensitive lung tissue. AAT deficiency is inherited. The severity of disease depends in part on the abnormalities present in the genes inherited from each parent. It is estimated that there are between 80,000 to 100,000 individuals affected by AAT deficiency in the United States.
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.
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Alpha-1 Antitrypsin Deficiency Pipeline Therapeutics Assessment
There are approx. 12+ key companies which are developing the therapies for Alpha-1 Antitrypsin Deficiency. The companies which have their Alpha-1 Antitrypsin Deficiency drug candidates in the most advanced stage, i.e. phase III include, Deerland Probiotics & Enzymes.
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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, Alpha-1 MP, ARO AAT, ALN-AAT02, and others
- Alpha-1 Antitrypsin Deficiency Pipeline Segmentation: Product Type, Molecule Type, Route of Administration
Got Queries? Find out the related information on Alpha-1 Antitrypsin Deficiency Mergers and acquisitions, Alpha-1 Antitrypsin Deficiency Licensing Activities @ Alpha-1 Antitrypsin Deficiency Emerging Drugs, and Recent Trends
Table of Content
- Introduction
- Executive Summary
- Alpha-1 Antitrypsin Deficiency: Overview
- Pipeline Therapeutics
- 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
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