tianasen

Tianasen (ASO-GNAO1) is an antisense oligonucleotide (ASO) developed by the Pirogov Russian National Research Medical University for the treatment of GNAO1-associated encephalopathy, a rare genetic disorder characterized by early-onset epilepsy and hyperkinesis. The drug is designed to target the mRNA of the GNAO1 gene, which encodes the Gαo subunit of heterotrimeric G proteins. Mutations in GNAO1 often lead to gain-of-function effects that disrupt normal neuronal signaling. Tianasen aims to reduce the levels of the pathogenic GNAO1 transcript, thereby decreasing the production of the mutant protein and alleviating neurological symptoms. It is administered via intrathecal injection and is currently being evaluated in pediatric patients in an open-label, dose-escalation clinical trial.

Brand names

Tianasen

Other names

Tianasen