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Automated Flow Synthesis of Peptide-PNA Conjugates.

Chengxi LiAlex J CallahanKruttika S PhadkeBryan H BellaireCharlotte E FarquharGenwei ZhangCarly K SchisselAlexander J MijalisNina HartrampfAndrei LoasDavid E VerhoevenBradley L Pentelute
Published in: ACS central science (2021)
Antisense peptide nucleic acids (PNAs) have yet to translate to the clinic because of poor cellular uptake, limited solubility, and rapid elimination. Cell-penetrating peptides (CPPs) covalently attached to PNAs may facilitate clinical development by improving uptake into cells. We report an efficient technology that utilizes a fully automated fast-flow instrument to manufacture CPP-conjugated PNAs (PPNAs) in a single shot. The machine is rapid, with each amide bond being formed in 10 s. Anti-IVS2-654 PPNA synthesized with this instrument presented threefold activity compared to transfected PNA in a splice-correction assay. We demonstrated the utility of this approach by chemically synthesizing eight anti-SARS-CoV-2 PPNAs in 1 day. A PPNA targeting the 5' untranslated region of SARS-CoV-2 genomic RNA reduced the viral titer by over 95% in a live virus infection assay (IC 50 = 0.8 μM). Our technology can deliver PPNA candidates to further investigate their potential as antiviral agents.
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