Proteomic analysis identified treatment-responsive protein biomarkers in patients with alpha-1 antitrypsin deficiency-associated liver disease

Proteomic analysis identified fazirsiran treatment-responsive protein biomarkers in patients with alpha-1 antitrypsin deficiency-associated liver disease.

Alpha-1 antitrypsin deficiency (AATD) is a rare genetic disease that causes low levels of the protein alpha-1 antitrypsin (AAT) in the blood, mainly affecting the lungs and liver. When someone has the Pi*ZZ genotype, the AAT protein is misfolded (called Z-AAT), leading to harmful aggregates in the liver and reduced protection in the lungs. This misfolding can cause liver damage, inflammation, and fibrosis due to the build up of these harmful proteins. Currently, no pharmacological treatments for AATD-associated liver disease (AATD-LD) exist.

Dr. Jen-Chieh Chuang, Director and Translational Biomarker Lead for Liver Diseases, will describe the proteomic analysis that identified treatment-responsive protein biomarkers for AATD-associated liver disease, enabling treatment to become a possibility for those affected.

Key learning objectives

• Understand how new proteomics tools are enabling the discovery of biomarkers for possible therapies for diseases that currently do not exist
• Discover how these tools can help you to understand the mechanism of action of disease states to better understand what target would be best for therapies

Who should attend?

• Anyone working in drug development 
• Anyone working in a disease area where therapies or treatments are lacking
• Anyone working in the area of precision medicine

Certificate of attendance
All webinar participants can request a certificate of attendance, including a learning outcomes summary, for continuing education purposes.