Future Therapeutic Options for Alpha-1 Antitrypsin Deficiency

Authors: Farah H , Steinbeck K , Hong D and Sadikot R

DOI: 10.23880/oajprs-16000142

Volume 6, Issue 1

Abstract

Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder that results in low circulating levels of alpha-1 antitrypsin (AAT). Low levels of AAT can result in damaged lung tissue, and pro-inflammatory state. The accumulation of polymerized Z-AAT protein within hepatocytes also causes cytotoxicity, resulting in liver cirrhosis. The current therapy includes weekly augmentation with plasma derived products. This has not proven to affect the progression of liver disease. Current research is targeting new modalities of treatment that will improve the progression of liver toxicity related to the polymerization of the Alpha-1 protein in the hepatocytes, in addition to offering patients more flexible treatment options, including oral therapy and less frequent intravenous infusions. These novel therapies include recombinant protein therapy, small molecule correctors,inhaled AAT therapy, RNA interference and monoclonal antibodies. The anti-inflammatory effect of Alpha-1 therapy is being explored in other disease states, including COVID-19 Pneumonia.

Keywords: Alpha-1 Antitrypsin Deficiency; Alpha-1 antitrypsin; INBRX-101

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