
Potential treatment for CJD
Looks like Sonia Vallabh and Eric Minikel have found a potential treatment for CJD which involves small interfering RNA (siRNA).
The phase 1 trial will enroll 15 patients diagnosed with prion disease who have symptoms of the neurodegenerative disorder.
The drug candidate is a divalent small interfering RNA (siRNA) molecule designed to cut target RNA, so cells produce less of the disease-causing prion protein.
The trial will test the safety and dosing of the siRNA, and help determine whether the drug candidate should advance to larger clinical trials.