Strnad emphasizes the promise offered by RNA therapeutics in liver disease and what potential fazirsiran may hold for AATD liver disease in a broad spectrum of patients.
RNA therapeutics have rapidly emerged as a transformative approach in modern medicine, offering the ability to selectively target disease at the genetic level. In hepatology, this precision is particularly valuable, as many RNA-based agents are naturally directed to the liver.
Among these novel therapies, fazirsiran stands out as a promising siRNA-based treatment for liver disease caused by alpha-1 antitrypsin deficiency (AATD), an area of significant unmet clinical need. A hepatocyte-targeted investigational RNA interference therapeutic, fazirsiran is designed to degrade alpha-1 antitrypsin and mutant misfolded Z alpha-1 antitrypsin (Z-AAT) messenger RNA in hepatocytes, subsequently reducing protein synthesis and inhibiting further accumulation of Z-AAT polymers to allow normal cellular mechanisms to dispose of mutant proteins.
At the American Association for the Study of Liver Diseases (AASLD) The Liver Meeting 2025, Pavel Strnad, MD, a full professor and leading physician at the University Hospital Aachen, presented data highlighting the promise of RNA therapeutics and fazirsiran in AATD liver disease across a broad spectrum of liver injury.
Read more here: https://www.hcplive.com/view/fazirsiran-future-sirna-therapy-aatd-liver-disease-with-pavel-strnad-md?brid=CJekvbTzpODInLAQ2S69qg
