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G2B-002 is a peptide-drug conjugate (PDC) developed by Gate2Brain for the treatment of aggressive pediatric brain tumors, specifically diffuse intrinsic pontine glioma (DIPG) and pediatric glioblastoma (pGBM). The drug utilizes a proprietary peptide-shuttle technology, specifically the MiniAp4 peptide (derived from apamin), to facilitate the transport of the chemotherapeutic agent SN-38 (the active metabolite of irinotecan) across the blood-brain barrier (BBB). By overcoming the BBB, G2B-002 aims to deliver therapeutic concentrations of the anticancer payload directly to the central nervous system, showing significantly higher brain penetration compared to conventional treatments in preclinical models. It has received Orphan Drug Designation from both the EMA and FDA and is currently in advanced preclinical development with Phase I/IIa trials planned for 2028.
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