New molecules such as CA-4948 (IRAK4 inhibitor), ibrutinib (BTK inhibitor acting as NRLP3 inhibitor), bortezomib (NFKb inhibitor), Cx-01 (TLR-4 inhibitor), or OPN-305 (TLR-2 inhibitor) are actually being evaluated in early-stage clinical studies for MDS and AML [50,128,129,130,131,132]. The gene discussed is BTK; the disease is myelodysplastic syndrome.