NIH Grant Awarded ($700 K) to Develop Drug to Treat Dravet Syndrome

LifeSplice was awarded a $700,000 NIH grant (NINDS) to use our SMO-based drug development platform technology to develop a greatly improved drug to treat Dravet Syndrome (DS). Dravet Syndrome/Severe Myoclonic Epilepsy of Infancy (SMEI) is a rare catastrophic form of childhood epilepsy. Progressive developmental and behavioral impairments manifest along with the recurrent and varied seizure episodes that advance to include multiple seizure types by age 2. Thus, there is a significant and urgent need for the development of novel approaches to pharmacotherapy. The LifeSplice SMO-based drug is expected to be more efficacious and much safer than current DS drugs. LifeSplice will benefit in the pre-clinical and ultimately clinical advancement of our DS drug from the leadership of our CSO Melanie Tallent, PhD (with exceptional experience studying various epilepsies) and from guidance of our SAB member Dr. Gregory Holmes, MD (Chair of Neurology, University of Vermont Medical School) who is a foremost world leader in studying and treating Dravet Syndrome. Our SMO-based DS drug has the potential to save the lives of children born with Dravet Syndrome that are resistant to currently available pharmacotherapies.