DelveInsight’s “Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease – Pipeline Insights, 2023,” report provides comprehensive insights about 8+ companies and 10+ pipeline drugs in Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease pipeline landscape.
Key Takeaways from the Alpha-1 Antitrypsin Deficiency (a1atd) Liver Disease Pipeline Report
Alpha-1 Antitrypsin Deficiency (a1atd) Liver Disease Overview
Alpha-1 antitrypsin deficiency (A1ATD) is a common inherited cause of liver disease, with the most severe mutation found in 1:3500 live births and currently 180,000 individuals worldwide. In children, A1ATD is the most frequent genetic etiology for pediatric liver disease and transplantation. The diagnosis of A1ATD is confirmed with laboratory analysis. Serum levels of A1AT can result in a false positive result because it is also an acute phase reactant and therefore elevated in the setting of inflammation. Phenotyping by isoelectric focusing is the gold standard of diagnosis. Phenotypic expression varies within and between families. In adults, smoking is the major factor in accelerating the development of COPD; nonsmokers may have a normal life span, but can also develop lung and/or liver disease. Although reported, emphysema in children with AATD is extremely rare. AATD-associated liver disease, which is present in only a small portion of affected children, manifests as neonatal cholestasis. The incidence of liver disease increases with age. Liver disease in adults (manifesting as cirrhosis and fibrosis) may occur in the absence of a history of neonatal or childhood liver disease. The risk for hepatocellular carcinoma (HCC) is increased in individuals with AATD.
Alpha-1 Antitrypsin Deficiency (a1atd) Liver Disease Pipeline Analysis: Drug Profile
ARO-AAT: Arrowhead Pharmaceuticals
ARO-AAT, also known as TAK-999, the company’s second-generation investigational RNA interference (RNAi) therapeutic being co-developed with Takeda Pharmaceutical Company Limited (“Takeda”) as a treatment for the rare genetic liver disease associated with alpha-1 antitrypsin deficiency (AATD).
Discover more about the emerging Alpha-1 Antitrypsin Deficiency (a1atd) Liver Disease drugs @ Alpha-1 Antitrypsin Deficiency (a1atd) Liver Disease Treatment Drugs
Alpha-1 Antitrypsin Deficiency (a1atd) Liver Disease Pipeline Therapies and Key Companies
And many others
Alpha-1 Antitrypsin Deficiency (a1atd) Liver Disease Pipeline Therapeutics Assessment
Phases
DelveInsight’s Report covers around 10+ products under different phases of clinical development like
Route of Administration
Alpha-1 Antitrypsin Deficiency (A1ATD) Liver Disease pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
Molecule Type
Products have been categorized under various Molecule types such as
Product Type
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
Scope of the Alpha-1 Antitrypsin Deficiency (a1atd) Liver Disease Pipeline Report
Find out more about the Alpha-1 Antitrypsin Deficiency (a1atd) Liver Disease treatment options in development @ Alpha-1 Antitrypsin Deficiency (a1atd) Liver Disease Clinical Trials
Table of Contents
1. Alpha-1 Antitrypsin Deficiency (a1atd) Liver Disease Introduction
2. Alpha-1 Antitrypsin Deficiency (a1atd) Liver Disease Executive Summary
3. Alpha-1 Antitrypsin Deficiency (a1atd) Liver Disease Overview
4. Alpha-1 Antitrypsin Deficiency (a1atd) Liver Disease Pipeline Therapeutics
5. Alpha-1 Antitrypsin Deficiency (a1atd) Liver Disease Late-Stage Products (Phase III)
6. Alpha-1 Antitrypsin Deficiency (a1atd) Liver Disease Mid-Stage Products (Phase II)
7. Alpha-1 Antitrypsin Deficiency (a1atd) Liver Disease Early Stage Products (Phase I/II)
8. Alpha-1 Antitrypsin Deficiency (a1atd) Liver Disease Preclinical Stage Products
9. Alpha-1 Antitrypsin Deficiency (a1atd) Liver Disease Discovery Stage Products
10. Alpha-1 Antitrypsin Deficiency (a1atd) Liver Disease Therapeutic Assessment
11. Alpha-1 Antitrypsin Deficiency (a1atd) Liver Disease Inactive Products
12. Alpha-1 Antitrypsin Deficiency (a1atd) Liver Disease Collaborations Assessment- Licensing / Partnering / Funding
13. Alpha-1 Antitrypsin Deficiency (a1atd) Liver Disease Unmet Needs
14. Alpha-1 Antitrypsin Deficiency (a1atd) Liver Disease Market Drivers and Barriers
15. Appendix
16. About DelveInsight
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