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AI is reshaping drug discovery, and nucleic acid–based medicines, including mRNAs, gene therapy, and oligonucleotide therapeutics, are no exception. By optimizing sequences and chemical modifications for experimental testing, AI accelerates discovery timelines, which is particularly critical for oligo therapeutics, a modality central to the n=1 rare diseases, which afflict mostly young patients for whom there is additional urgency.
However, a familiar caveat remains: AI is only as powerful as the data from which it learns….