DATA GRAPHICS | Data Byte
The AATD pipeline
Clinical programs include proteins, RNAi and small molecules, with gene editing getting in the game too
November 2, 2021 1:31 AM UTC
At least 15 programs based on five different mechanistic approaches are in preclinical and clinical testing to treat alpha-antitrypsin deficiency.
The approach of using a small molecule to correct misfolded AAT and restore its function took a step forward Monday, when Centessa Pharmaceuticals plc (NASDAQ:CNTA) and its Z Factor Ltd. subsidiary said they will advance ZF874 into Phase II testing for AATD based on biomarker data suggesting the molecule may raise functional AAT to levels needed for clinical benefit. However, the scant amount of data, which came from just two patients, and the elevated liver enzymes seen in one of those patients, worried investors. ...