Arrowhead begins Phase II trial of ARC-AAT to treat AATD

US-based biopharmaceutical firm Arrowhead has commenced its Phase II clinical trial of ARC-AAT to treat liver disease associated with alpha-1 antitrypsin deficiency (AATD).
Source: Drug Development Technology - Category: Pharmaceuticals Source Type: news

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α1-antitrypsin deficiency (AATD) is a hereditary disorder associated with a risk of developing liver disease and pulmonary emphysema, and other chronic respiratory disorders (mainly asthma and bronchiectasis); Z variant is the commonest deficient variant of AAT. Determining AAT concentration in serum or plasma and identifying allelic variants by phenotyping or genotyping are fundamental in the diagnosis of AATD. Initial evaluation and annual follow-up measurement of lung function, including post-bronchodilator forced expiratory volume in 1 s and gas transfer inform on disease progression. Lung densitometry is th...
Source: European Respiratory Review - Category: Respiratory Medicine Authors: Tags: COPD and smoking, Genetics Review Source Type: research
CONCLUSIONS: Beside the role in the first diagnostic step of liver injuries, the utility of liver transaminases is also maintained during the follow-up of liver diseases and in their prognostic assessment. PMID: 31994373 [PubMed - as supplied by publisher]
Source: Minerva Gastroenterologica e Dietologica - Category: Gastroenterology Tags: Minerva Gastroenterol Dietol Source Type: research
ConclusionsLiver organoid model recapitulates the key features of Z-AAT deficiency and provides a useful tool for disease modeling.
Source: Hepatology International - Category: Infectious Diseases Source Type: research
In this study, we developed a diagnostic model combining ultrasonography with biomarkers to identify mild NAFLD, with MRS as the reference standard. A total of 422 eligible subjects were enrolled. The serum levels of fibroblast growth factor 21 (FGF21), cytokeratin 18 M65ED, proteinase 3, neutrophil elastase, alpha-1 antitrypsin, and neutrophil elastase/alpha-1 antitrypsin were measured using ELISA assays. We found that among the six biomarkers, only serum FGF21 was independently associated with intrahepatic triglyceride content (IHTC, standardized β = 0.185, P 
Source: Acta Pharmacologica Sinica - Category: Drugs & Pharmacology Authors: Tags: Acta Pharmacol Sin Source Type: research
Conclusions: Generation of liver organoids might be a useful tool to investigate the factors contributing to hepatic disease in AAT deficiency.
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Molecular pathology and funct. genomics Source Type: research
Conclusions: To the best of our knowledge, this is the first controlled study to assess the relationship between AATD and ascending aortic diameter, and suggests AATD is associated with accelerated aortic wall degeneration.
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Genes and environment Source Type: research
Conclusions: We have identified a genetic signature that differentiates the different risk groups and the presence of emphysema and hepatopathy in AATD patients.
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Molecular pathology and funct. genomics Source Type: research
This article describes hereditary hemochromatosis, Gilbert syndrome, alpha-1 antitrypsin deficiency, Wilson disease, PFIC, BRIC, and LAL-D. The most common cause of hereditary hemochromatosis is a C282Y mutation in the HFE gene. Gilbert syndrome is a benign cause of indirect hyperbilirubinemia. Alpha-1 antitrypsin deficiency causes both lung and liver disease. Wilson disease can cause neurologic disease and liver disease. Progressive familial intrahepatic cholestasis and benign recurrent intrahepatic cholestasis are rare causes of cholestasis. LAL-D is a rare disease that can appear similar to NAFLD in adults. PMID: 3...
Source: The Medical Clinics of North America - Category: General Medicine Authors: Tags: Med Clin North Am Source Type: research
We would like to congratulate Clark et al. for their pioneering work characterizing histological liver injury in patients with the classic severe alpha-1 antitrypsin (AAT) deficiency (genotype Pi*ZZ).1 The Pi*ZZ genotype is seen in 1:3,000 Caucasians and the associated liver disease is greatly understudied despite the fact that it is more frequent than several well-established liver disorders such as autoimmune hepatitis or primary sclerosing cholangitis.2,3 While Clark et al. greatly enhanced our understanding of the clinical, biochemical and histological liver phenotype of these individuals, we would like to further disc...
Source: Journal of Hepatology - Category: Gastroenterology Authors: Tags: Letter to the Editor Source Type: research
This article describes hereditary hemochromatosis, Gilbert syndrome, alpha-1 antitrypsin deficiency, Wilson disease, PFIC, BRIC, and LAL-D. The most common cause of hereditary hemochromatosis is a C282Y mutation in the HFE gene. Gilbert syndrome is a benign cause of indirect hyperbilirubinemia. Alpha-1 antitrypsin deficiency causes both lung and liver disease. Wilson disease can cause neurologic disease and liver disease. Progressive familial intrahepatic cholestasis and benign recurrent intrahepatic cholestasis are rare causes of cholestasis. LAL-D is a rare disease that can appear similar to NAFLD in adults.
Source: Medical Clinics of North America - Category: Primary Care Authors: Source Type: research
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