Ann Barbier, MD, PhD, Chief Medical Officer at CAMP4 Therapeutics, discusses positive animal model data from CAMP4’s investigational RNA-based therapy for Dravet syndrome, CMP-SCN (CO-3527).

Dravet syndrome is a rare neurological condition that usually appears during the first year of life as frequent febrile seizures. As the condition progresses, other types of seizures typically occur, including myoclonus and status epilepticus. Moderate to severe cognitive impairment is also common. Most cases of Dravet syndrome occur due to a mutation of the SCN1A gene. It can be inherited in an autosomal dominant pattern, but most people with Dravet syndrome do not have a family history of the condition. 

CMP-SCN is an investigational RNA-based therapy designed to directly upregulate gene expression of SCN1A by targeting a subset of regulatory RNAs that control mRNA transcription. The result is the upregulation of SCN1A two- or three-fold.

As Dr. Barbier explains, in a mouse model of Dravet syndrome, treatment with CMP-SCN led to an increase by 25% in the activity of the SCN1A gene. This was accompanied by a reduction of approximately 70% of seizure frequency and duration. In non-human primates, CMP-SCN also led to an increase in both SCN1A mRNA and SCN1A protein levels.

Dr. Barbier also notes that CAMP4 Therapeutics is planning to submit an investigational new drug (IND) application in the U.S. to conduct its first in-human studies by the end of 2022.

More recent data from this program as well as from other CAMP4 programs will be presented at the 2nd Annual Oligonucleotides for CNS Summit in Boston June 15, 2022 at 1-1:30 p.m. ET and at the Dravet Syndrome Foundation’s 5th Biennial Conference in Fort Worth, Texas, June 23, 2022 at 5:05-5:25 p.m. CT.

To learn more about Dravet syndrome and other rare neurological disorders, visit checkrare.com/diseases/neurology