- In October 2024, Sarepta Therapeutics faced a significant setback when European regulators paused all clinical trials of its gene therapy, Elevidys, following the death of a U.S. teenager treated with the drug. Elevidys targets Duchenne muscular dystrophy and had been administered to over 800 patients prior to this incident
- In July 2024, GSK acquired CureVac's stakes in their collaborative influenza and COVID-19 vaccine development, including an upfront payment of 400 million euros and potential milestone payments up to 1.05 billion euros. This move aims to strengthen GSK's mRNA technology capabilities
- In March 2024, Eli Lilly's experimental drug, lepodisiran, demonstrated promising results by reducing lipoprotein(a) levels by 94% for six months with a single shot in a Phase 2 trial. Elevated lipoprotein(a) is a significant risk factor for heart attacks and strokes
- In October 2023, Novo Nordisk received FDA approval for Rivfloza, an RNA interference (RNAi) therapy designed to treat a rare kidney-affecting genetic disorder. This therapy utilizes RNAi technology to lower urinary oxalate levels by silencing genes that contribute to the disease
- In July 2023: Alnylam Pharmaceuticals entered into a strategic agreement with Roche to develop and commercialize zilebesiran, an investigational RNAi therapeutic in Phase 2 development for the treatment of hypertension. This collaboration aims to advance RNAi-based treatments for cardiovascular conditions
