He received the 2022 Shaw Prize in Life science and medicine, together with Michael J. Welsh, for their work that uncovered the etiology of cystic fibrosis and developed effective medications.
He was planning to move to the University of Connecticut when Roger Y. Tsien asked him to join a startup company he was forming called Aurora Biosciences.
[10] Via high-throughput screening, his team at Vertex Pharmaceuticals discovered ivacaftor, a small-molecule potentiator that increases the probability that mutated CFTR gates will open.
[13] Of note, the expanded approval in 2017 was based solely on in vitro data, due to the small number of patients carrying those rare mutations making clinical trials impossible.
[14] Negulescu also led the discovery of another cystic fibrosis drug, lumacaftor, which is known as a "corrector" as it acts as a chaperone to help the CFTR protein fold correctly.
[15] Thus, it can be used in patients with Class II mutations, which create misfolded CFTR protein that cannot reaching the cell surface.