Prostate cancer: Antisense nucleic acids-tough delivery

摘要

First-in-human data of the locked antisense oligonucleotide (ASO) EZN-4176, which binds exon 4 of the androgen receptor (AR) mRNA (encoding the amino terminus), shows dosing to be limited by transaminase elevation with no evidence of target modulation at the highest tested doses. Whether amino-terminus targeting ASOs will advance clinically remains uncertain.