Development of pharmacological correctors for Alpha-1 antitrypsin deficiency (ATD) using VIPER
Challenge: Drug discovery by high-throughput screening of candidate compounds is usually limited in efficacy as their functional potential is not assessed in relevant biological systems, such as patient cells, during this first selection; new platforms are needed that enable functional testing within specific biological systems. An example of protein trafficking that would benefit from such platforms is Alpha-1 antitrypsin deficiency (ATD), an orphan disease, whose behaviour in the endoplasmic reticulum (ER) is complex, rendering the design of therapeutic candidates difficult. Solution:…