- In May 2025, researchers at the Broad Institute of MIT and Harvard utilized base editing to introduce a single-letter DNA change, reducing disease-causing prion protein levels in a mouse model by up to 60%, leading to a lifespan extension of approximately 50%
- In April 2025, preclinical studies demonstrated that Antisense Oligonucleotide (ASO) therapy could effectively lower prion protein levels, tripling survival rates in prion-infected animals. A single dose administered after symptom onset prolonged survival by months in some cases
- In March 2025, the development of the CHARMs technology, which silences the gene encoding prion protein, was announced, achieving up to an 80% reduction in prion protein levels in mouse brains
- In February 2025, researchers identified ten compounds capable of reducing prion protein accumulation in infected cells, five of which are already approved for human use, providing an expedited path to clinical application
- In January 2025, Ionis Pharmaceuticals completed enrollment for a Phase 1/2a clinical trial (PrProfile) evaluating ION717 in symptomatic prion disease patients, marking a significant step toward developing effective treatments for prion diseases
