Paxalisib, a dual PI3K/mTOR inhibitor specifically developed for the treatment of GBM, has demonstrated favourable safety profile and promising efficacy as a first-line treatment in a Phase 2 trial, and is being further investigated in patients with newly diagnosed or recurrent GBM as a part of the AGILE GBM trial [102, 103]. The gene discussed is MTOR; the disease is glioblastoma.