Alpha-1 Antitrypsin Deficiency Pipeline | Companies – Octapharma, Dicerna Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, Vertex Pharmaceuticals, AstraZeneca, Kamada, Ltd

June 01 08:52 2023
Alpha-1 Antitrypsin Deficiency Pipeline | Companies - Octapharma, Dicerna Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, Vertex Pharmaceuticals, AstraZeneca, Kamada, Ltd

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.

 

To explore more information on the latest breakthroughs in the Alpha-1 Antitrypsin Deficiency Pipeline treatment landscape of the report, click here @ Alpha-1 Antitrypsin Deficiency Pipeline Outlook

 

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.

 

For further information, refer to the detailed Alpha-1 Antitrypsin Deficiency Unmet Needs, Alpha-1 Antitrypsin Deficiency Market Drivers, and Alpha-1 Antitrypsin Deficiency Market Barriers, click here for Alpha-1 Antitrypsin Deficiency Ongoing Clinical Trial Analysis

 

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.

 

Request a sample and discover the recent advances in Alpha-1 Antitrypsin Deficiency Ongoing Clinical Trial Analysis and Medications, click here @ Alpha-1 Antitrypsin Deficiency Treatment Landscape

 

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.

 

Dive deep into rich insights for drugs for Alpha-1 Antitrypsin Deficiency Market Drivers and Alpha-1 Antitrypsin Deficiency Market Barriers, click here @ Alpha-1 Antitrypsin Deficiency Unmet Needs and Analyst Views

 

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

  1. Introduction
  2. Executive Summary
  3. Alpha-1 Antitrypsin Deficiency: Overview
  4. Pipeline Therapeutics
  5. Therapeutic Assessment
  6. Alpha-1 Antitrypsin Deficiency– DelveInsight’s Analytical Perspective
  7. Late Stage Products (Phase III)
  8. ARO AAT: Arrowhead Pharmaceuticals
  9. Drug profiles in the detailed report…..
  10. Mid Stage Products (Phase II)
  11. Drug Name: Company Name
  12. Drug profiles in the detailed report…..
  13. Early Stage Products (Phase I/II)
  14. ALN-AAT02: Alnylam Pharmaceuticals
  15. Drug profiles in the detailed report…..
  16. Preclinical and Discovery Stage Products
  17. Drug Name: Company Name
  18. Drug profiles in the detailed report…..
  19. Inactive Products
  20. Alpha-1 Antitrypsin Deficiency Key Companies
  21. Alpha-1 Antitrypsin Deficiency Key Products
  22. Alpha-1 Antitrypsin Deficiency- Unmet Needs
  23. Alpha-1 Antitrypsin Deficiency- Market Drivers and Barriers
  24. Alpha-1 Antitrypsin Deficiency- Future Perspectives and Conclusion
  25. Alpha-1 Antitrypsin Deficiency Analyst Views
  26. Alpha-1 Antitrypsin Deficiency Key Companies
  27. Appendix

 

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