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CG009301 is a highly selective, investigational small molecule degrader of GSPT1 (G1 to S phase transition 1), developed using Cullgen’s proprietary uSMITE platform for targeted protein degradation. It is designed as a proteolysis-targeting chimera (PROTAC) that induces the ubiquitination and subsequent proteasomal degradation of GSPT1, a protein implicated in cell cycle regulation and survival of malignant cells. The drug is being developed primarily for the treatment of relapsed or refractory hematological malignancies, including acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), and high-risk myelodysplastic syndromes (MDS). There is also potential for its use in solid tumors with MYC amplification. As of late 2024, the Chinese National Medical Products Administration has allowed an IND application for Phase I clinical trials in China[1][2][3][8].
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