BGA002

BGA002 is a synthetic antigene oligonucleotide designed to specifically inhibit the MYCN proto-oncogene, a key driver in several aggressive pediatric and adult cancers such as neuroblastoma and small cell lung cancer. It acts by binding directly to the MYCN gene sequence, thereby blocking its transcription and reducing N-Myc protein levels. This leads to inhibition of tumorigenic activity, restoration of retinoic acid sensitivity (in neuroblastoma), induction of differentiation or apoptosis in cancer cells, and suppression of mTOR pathway signaling with subsequent autophagy reactivation. BGA002 has demonstrated efficacy in preclinical models for MYCN-amplified neuroblastoma (MNA-NB) and small cell lung cancer (SCLC), including overcoming multidrug resistance and inhibiting tumor vascularization. The drug has received orphan drug designation from both the FDA and EMA for SCLC and NB[2][3][4][5][8].

Other names

16 base single stranded peptide nucleic acid oligonucleotide16-base single-stranded peptide nucleic acid oligonucleotide linked to 7-amino acid peptide