Posted by Michael Wonder on 16 Jul 2020
Knopp Biosciences receives rare paediatric disease designation for Kv7 activator KB-3061 for treatment of KCNQ2 epileptic encephalopathy
15 July 2020 - Knopp Biosciences announced today that it has received rare paediatric disease designation from the U.S. FDA for its therapeutic candidate KB-3061, an activator of voltage-gated Kv7.2/7.3 potassium channels, for the treatment of KCNQ2 epileptic encephalopathy.
Knopp is advancing the development of KB-3061 as a potential precision medicine for KCNQ2-EE, a rare genetic disease associated with seizures beginning in the first days of life and profound neurodevelopmental delay.
The disease is caused by dominant-negative mutations in the KCNQ2 gene, which encodes for Kv7.2, a potassium channel that plays a critical role in early brain development.
