Fragile X treatment licensed to Lenire Biosciences by Ïã¸ÛÁùºÏ²Ê
23 June 2022
Fragile X syndrome (FXS) is the most common genetically linked cause of intellectual disabilities and autism
Fragile X syndrome (FXS)Ìýis a neurodevelopmental condition occurring due to a CGG trinucleotide expansion in the FMR1 gene. Polymorphisms and variants in large-conductance calcium-activated potassium channels are increasingly linked to intellectual disability and loss of FMR protein causes reduced large-conductance calcium-activated potassium channel activity leading to abnormalities in synapse function.
Using the endo-cannabinoid-like large-conductance calcium-activated potassium channel activator VSN16R, designed in our lab we rescued behavioural deficits such as repetitive behaviour, hippocampal dependent tests of daily living, hyperactivity and memory in a mouse model of fragile X syndrome (Study published in Brain 2021).
VSN16R was designed and synthesised in the Selwood lab at Ïã¸ÛÁùºÏ²Ê by Cristina Visintin and Masahiro Takahiro and originally developed for Multiple Sclerosis. First studies in people with Fragile X are expected in 2023.
News articles
Publication
- 'Reversal of behavioural phenotype by the cannabinoid-like compound VSN16R in fragile X syndrome mice'Ìý Brain, Volume 145, Issue 1, January 2022, Pages 76–82,Ìý
Wolfson Institute for Biomedical Research Neurosciences (WIBR), Division of Medicine