• Current AATD treatment, augmentation therapy, involves weekly infusions but doesn't reverse lung or liver damage, spurring the development of novel therapies.
• Fazirsiran, an RNAi therapy by Arrowhead and Takeda, shows promise in reducing Z-AAT levels in Phase II trials, with Phase III trials underway for AATD-associated liver disease.
• Beam Therapeutics is exploring BEAM-302, a liver-targeting base editing therapy, while Wave Life Sciences, AIRNA, and Korro Bio are advancing RNA-editing oligonucleotides for AATD.
• With multiple investigational treatments showing promise, augmentation therapy could become obsolete within the next decade, potentially transforming AATD treatment.