Cognigenics CEO John L. Mee announces breakthrough results from an animal study published today in PNAS Nexus. The findings point to the potential of non-invasively delivering neuromodulating gene therapies to the brain for chronic mental health conditions using adeno-associated viruses introduced intranasally.

“While our findings are preliminary, they may lead to a new class of long-lasting therapeutics with fewer side effects than SSRIs for mental health conditions including anxiety, depression and ADHD, as well as for some genetic conditions.”

"Our patent-pending CRISPR/Cas9 delivery system enabled us to bypass the blood-brain barrier by intranasally administering therapeutics to reach the central nervous system via the neural pathways in the nasal cavity,” said Director of Preclinical Studies Troy Rohn, PhD, principal investigator of the study. “In the study, a single dose significantly reduced anxiety in mice, demonstrating that complex behavioral traits can be directly modified by this non-invasive technique.”

Chief Medical Officer Barry J. Linder, MD added, "While our findings are preliminary, they may lead to a new class of long-lasting therapeutics with fewer side effects than SSRIs for mental health conditions including anxiety, depression and ADHD, as well as for some genetic conditions."

Significant reduction in anxiety

The animal study tested a platform of CRISPR/Cas9 utilizing two adeno-associated viruses (AAVs) to deliver anti-anxiety reagents in vivo to the part of the brain that is overactive in anxiety. The method requires two delivery vectors because of the limited cargo capacity of the viruses. Each virus was modified using Cognigenics' proprietary CRISPR gene-editing technology.

Treated mice showed 35.7% and 14.8% decreases in two standard measures of anxiety.

No toxicity or neurologic damage

Importantly, there was no evidence of toxicity at any concentration following treatment, and neurons were healthy.