CAMP4’s RNA-based Therapy Shows Promise in Preclinical Studies

Note: This story was updated Feb. 7, 2022, to clarify that CAMP4 will continue preclinical studies for its lead candidate. Also, CAMP4 licensed the antisense oligonucleotides from OPKO Health, not the RNA Actuating Platform, which was developed in-house. CAMP4 Therapeutics‘ investigational RNA-based therapy — called CMP-SCN (CO-3527) — increased…

STK-001 Seen to Reduce Seizures in Children With Severe Dravet

Single and multiple doses of Stoke Therapeutics’ investigational therapy STK-001 were well-tolerated and led to a significant reduction in the frequency of convulsive seizures among children and adolescents with Dravet syndrome in the first three months of follow-up, interim data from the ongoing Phase 1/2a MONARCH trial show.