Alpha-1 Antitrypsin Deficiency Pipeline Analysis 2024: FDA Approvals, Clinical Trials, Therapies, Mechanism of Action, ROA, and Companies by DelveInsight | Fazirsiran Injection, Aerosolized, Recombinant Alpha 1-Antitrypsin, INBRX-101/rhAAT-Fc, ADVM-043, m

(Albany, USA) DelveInsight's, "Alpha1-Antitrypsin Deficiency Pipeline Insight 2024" report provides comprehensive insights about 12+ companies and 12+ pipeline drugs in the Alpha1-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 pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

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 treatment.
• The leading companies working in the Alpha-1 Antitrypsin Deficiency Market include Octapharma, Dicerna Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, Vertex Pharmaceuticals, AstraZeneca, Kamada Ltd and others.
• Promising Alpha-1 Antitrypsin Deficiency Pipeline Therapies in the various stages of development include Fazirsiran Injection, Aerosolized, Recombinant Alpha 1-Antitrypsin, INBRX-101/rhAAT-Fc, ADVM-043, and others.
• October 2023: Takeda announced a study of Phase 3 clinical trials for Fazirsiran Injection. The main aim of this study is to learn if fazirsiran reduces liver scarring (fibrosis) compared to placebo. Other aims are to learn if fazirsiran slows down the disease worsening in the liver, to get information on how fazirsiran affects the body (called pharmacodynamics), to learn if fazirsiran reduces other liver injury (inflammation) and the abnormal Z-AAT protein in the liver, to get information on how the body processes fazirsiran (called pharmacokinetics), to test how well fazirsiran works compared with a placebo in improving measures of liver scarring including imaging and liver biomarkers (substances in the blood that the body normally makes and help show if liver function is improving, staying the same, or getting worse) as well as to check for side effects in participants treated with fazirsiran compared with those who received placebo.
• October 2023: Grifols Therapeutics LLC announced a study of Phase 1 & 2 clinical trials for Alpha-1 15%. The purpose of this study is to evaluate the safety and tolerability of 72 milligrams per kilogram (mg/kg) and 144 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).

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In the Alpha-1 Antitrypsin Deficiency pipeline report, detailed description of the drug is given which includes mechanism of action of the drug, Alpha-1 Antitrypsin Deficiency clinical trials, NDA approvals (if any), and product development activities comprising the technology, Alpha-1 Antitrypsin Deficiency collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Alpha-1 Antitrypsin Deficiency Overview
Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder that primarily affects the lungs and liver. It results from mutations in the SERPINA1 gene, which encodes alpha-1 antitrypsin (AAT), a protein that protects tissues from enzyme damage during inflammation. AATD leads to reduced levels or dysfunctional AAT, allowing enzymes like neutrophil elastase to damage lung tissue. This can cause chronic obstructive pulmonary disease (COPD), emphysema, and chronic bronchitis, typically manifesting in adulthood. Symptoms include shortness of breath, wheezing, chronic cough, and increased risk of respiratory infections.
Liver disease in AATD, caused by the accumulation of abnormal AAT protein in liver cells, can range from mild elevations in liver enzymes to cirrhosis and hepatocellular carcinoma. Some individuals may also develop panniculitis, a rare skin condition.
Diagnosis involves blood tests to measure AAT levels, genetic testing for SERPINA1 mutations, and liver function tests. Management includes avoiding smoking and pollutants, vaccinations to prevent infections, bronchodilators, and in severe cases, intravenous AAT augmentation therapy. For advanced lung or liver disease, organ transplantation may be considered. Early diagnosis and intervention are crucial to managing symptoms and improving quality of life.

Find out more about Alpha-1 Antitrypsin Deficiency Therapeutics Assessment @ Alpha-1 Antitrypsin Deficiency Preclinical and Discovery Stage Products- https://www.delveinsight.com/sample-request/alpha-1-antitrypsin-deficiency-aatd-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

Alpha-1 Antitrypsin Deficiency Emerging Drugs Profile
• ARO AAT: Arrowhead Pharmaceuticals
• ALN-AAT02: Alnylam Pharmaceuticals

Alpha-1 Antitrypsin Deficiency Pipeline Therapeutics Assessment
There are approx. 12+ key companies which are developing the Alpha-1 Antitrypsin Deficiency therapies. The Alpha-1 Antitrypsin Deficiency companies which have their Alpha-1 Antitrypsin Deficiency drug candidates in the most advanced stage, i.e. phase III include, Deerland Probiotics & Enzymes.

DelveInsight's Alpha-1 Antitrypsin Deficiency pipeline report covers around 12+ products under different phases of clinical development like
• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

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 Pipeline Products have been categorized under various Molecule types such as
• Oligonucleotide
• Peptide
• Small molecule

Learn more about the emerging Alpha-1 Antitrypsin Deficiency Pipeline Therapies @ Alpha-1 Antitrypsin Deficiency Clinical Trials Assessment- https://www.delveinsight.com/sample-request/alpha-1-antitrypsin-deficiency-aatd-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

Scope of the Alpha-1 Antitrypsin Deficiency Pipeline Report
• Coverage- Global
• 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
• 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.

Dive deep into rich insights for new drugs for Alpha-1 Antitrypsin Deficiency Treatment, Visit @ Alpha-1 Antitrypsin Deficiency Market Drivers and Barriers, and Future Perspective- https://www.delveinsight.com/sample-request/alpha-1-antitrypsin-deficiency-aatd-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

Table of Content
1. Introduction
2. Alpha-1 Antitrypsin Deficiency Executive Summary
3. Alpha-1 Antitrypsin Deficiency: Overview
4. Alpha-1 Antitrypsin Deficiency Pipeline Therapeutics
5. Alpha-1 Antitrypsin Deficiency Pipeline 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

For further information on the Alpha-1 Antitrypsin Deficiency Pipeline therapeutics, reach out to Alpha-1 Antitrypsin Deficiency Unmet Needs and Analyst Views- https://www.delveinsight.com/sample-request/alpha-1-antitrypsin-deficiency-aatd-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

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