Award
National Heart, Lung, and Blood Institute 5R01HL169631-03
Managing Alpha-1-Antitrypsin Deficiency (AATD) through Proteostasis Signaling Pathways
Recipient
SCRIPPS RESEARCH INSTITUTE, THE
Award Amount
$855,694.00
Ceiling
$855,694.00
Awarded
February 27, 2026
Identifier
5R01HL169631-03
This award funds research to develop therapies for Alpha-1-antitrypsin deficiency by targeting proteostasis signaling pathways using small molecules and machine learning.
Description
The project aims to manipulate proteostasis pathways (UPR involving ATF6 and IRE1/XBP1s) using small molecules and machine learning tools to develop therapies for Alpha-1-antitrypsin deficiency (AATD), an inherited disease leading to COPD and liver failure due to misfolding of AAT variants.