Hybridization Liquid Chromatography-Tandem Mass Spectrometry: An Alternative Bioanalytical Method for Antisense Oligonucleotide Quantitation in Plasma and Tissue Samples.
Li P, Gong Y, Kim J, Liu X, Gilbert J, Kerns HM, Groth R, Rooney M. Anal Chem. 2020 Jul 17. Pubmed
Dose-Finding Study and Pharmacokinetics Profile of the Novel 13-Mer Antisense miR-221 Inhibitor in Sprague-Dawley Rats
Di Martino MT, Arbitrio M, Caracciolo D, Scionti F, Tagliaferri P, Tassone P. Mol Ther Nucleic Acids. 2020 Jun 5;20:73-85. Pubmed
Safety, Tolerability, and Efficacy of Viltolarsen in Boys with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial
Clemens PR, Rao VK, Connolly AM, Harper AD, Mah JK, Smith EC, McDonald CM, Zaidman CM, Morgenroth LP, Osaki H, Satou Y, Yamashita T, Hoffman EP; CINRG DNHS Investigators. JAMA Neurol. 2020 May 26 Pubmed
Effect of inclisiran, the siRNA against PCSK9, on platelets, immune cells and immunological biomarkers – a pre-specified analysis from ORION-1
Landmesser U, Haghikia A, Leiter LA, Wright RS, Kallend D, Wijngaard P, Stoekenbroek R, Kastelein JJP, Ray KK. Cardiovasc Res. 2020 Apr 3. PubMed
Nonclinical Safety Profile of Revusiran, a 1st-Generation GalNAc-siRNA Conjugate for Treatment of Hereditary Transthyretin-Mediated Amyloidosis.
Sutherland JE, Hettinger JL, Chan A, Gilbert J, Warner GL, Davis WP. Nucleic Acid Ther. 2019 Dec 10. PubMed
Impurity Qualification Toxicology Study for a 2′-O-Methoxyethyl-Modified Antisense Inhibitor in Mice.
Kim TW, Papagiannis C, Capaldi D, McPherson AK, Mahdi F, Luu N, Rodriguez AA, Hoffmaster C, Serota D, Henry SP. Nucleic Acid Ther. 2019 Nov 5. PubMed
Therapy with 2′-O-Me Phosphorothioate Antisense Oligonucleotides Causes Reversible Proteinuria by Inhibiting Renal Protein Reabsorption
Janssen MJ, Nieskens TTG, Steevels TAM, Caetano-Pinto P, den Braanker D, Mulder M, Ponstein Y, Jones S, Masereeuw R, den Besten C, Wilmer MJ. Mol Ther Nucleic Acids. 2019 Sep 6;18:298-307. PubMed
Hybrid Mouse Diversity Panel Identifies Genetic Architecture Associated with the Acute Antisense Oligonucleotide-Mediated Inflammatory Response to a 2′-O-Methoxyethyl Antisense Oligonucleotide
Pirie E, Cauntay P, Fu W, Ray S, Pan C, Lusis AJ, Hsiao J, Burel SA, Narayanan P, Crooke RM, Lee RG. Nucleic Acid Ther. 2019 Aug 1. PubMed
In Vitro Drug-Drug Interaction Evaluation of GalNAc conjugated siRNAs Against CYP450 Enzymes and Transporters
Ramsden D, Wu JT, Zerler B, Iqbal S, Jiang J, Clausen V, Aluri K, Gu Y, Dennin S, Kim J, Chong S. Drug Metab Dispos. 2019 Jul 3. PubMed
Safety evaluation of 2′-deoxy-2′-fluoro nucleotides in GalNAc-siRNA conjugates
Janas MM, Zlatev I, Liu J, Jiang Y, Barros SA, Sutherland JE, Davis WP, Liu J, Brown CR, Liu X, Schlegel MK, Blair L, Zhang X, Das B, Tran C, Aluri K, Li J, Agarwal S, Indrakanti R, Charisse K, Nair J, Matsuda S, Rajeev ...