The scientific histories behind human disease breakthroughs.

Daraxonrasib for KRAS G12D-mutant pancreatic cancer

Daraxonrasib (KRAS G12D inhibitor) · Pancreatic ductal adenocarcinoma

KRAS oncogene discovery → structural biology → covalent inhibitor chemistry → translational oncology

Luxturna for RPE65-mediated inherited retinal disease

Voretigene neparvovec (AAV2-RPE65) · Leber congenital amaurosis (RPE65)

RPE65 biology → AAV vector development → animal models → subretinal delivery → rare-disease trials

ION582 for Angelman syndrome

ION582 (obudanersen), antisense oligonucleotide · Angelman syndrome

UBE3A imprinting → antisense transcript biology → ASO chemistry → CNS delivery

BMS-986278 for idiopathic pulmonary fibrosis

BMS-986278, oral LPA1 receptor antagonist · Idiopathic pulmonary fibrosis

Fibrosis pathology → lipid signaling → LPA1 pharmacology → antifibrotic drug development

Semaglutide for cardiovascular risk reduction in obesity (Wegovy / SELECT)

Semaglutide 2.4 mg (Wegovy), GLP-1 receptor agonist · Cardiovascular disease in adults with obesity

GLP-1 discovery → incretin biology → peptide pharmacology → cardiovascular outcomes trial

Pembrolizumab for MSI-H/dMMR solid tumors

Pembrolizumab (anti-PD-1 monoclonal antibody) · MSI-H/dMMR solid tumors (tissue-agnostic)

PD-1 biology → T-cell regulation → mismatch repair deficiency → microsatellite instability → basket trials → tissue-agnostic regulatory logic