Investigating the Feasibility of ASO therapy in Pitt Hopkins Syndrome

La Jolla Labs, CA

ASOs are an established class of drug showing increasing success in treating neurological diseases. To determine if ASOs would make good therapeutics for Pitt Hopkins Syndrome (PHS), the PHRF has arranged a collaboration with Mahzi Therapeutics and La Jolla Labs. Currently, this project is in the very early phases of scientific development, working to establish proper biological systems to evaluate this strategy. Once experimental conditions are worked out, this collaboration will test the ability of ASOs to restore the protein levels of TCF4 and effectively reverse the cause of PTHS.

ASO’s are currently being explored in several Angelman trials and the idea of this ASO, if successful will be to upregulate the good copy of the TCF4 thereby increasing the protein overall.