Publication Cover
Xenobiotica
the fate of foreign compounds in biological systems
Latest Articles
358
Views
0
CrossRef citations to date
0
Altmetric
Review Article

Bioanalytical Approaches to Support the Development of Antibody-Oligonucleotide Conjugate (AOC) Therapeutic Proteins

, &
Received 16 Feb 2024, Accepted 03 Apr 2024, Accepted author version posted online: 12 Apr 2024
Accepted author version

References

  • Agrawal K, Kang L, Ji S, Tena J, Jian W. 2023. Evaluating the use of locked nucleic acid capture probes in hybrid LC-MS/MS analysis of siRNA analytes. Bioanalysis. 15(18):1129-1146.
  • Baumer N, Appel N, Terheyden L, Buchholz F, Rossig C, Muller-Tidow C, Berdel WE, Baumer S. 2016. Antibody-coupled siRNA as an efficient method for in vivo mRNA knockdown. Nat Protoc. 11(1):22-36.
  • Baumer S, Baumer N, Appel N, Terheyden L, Fremerey J, Schelhaas S, Wardelmann E, Buchholz F, Berdel WE, Muller-Tidow C. 2015. Antibody-mediated delivery of anti-KRAS-siRNA in vivo overcomes therapy resistance in colon cancer. Clin Cancer Res. 21(6):1383-1394.
  • Beaumont K, Pike A, Davies M, Savoca A, Vasalou C, Harlfinger S, Ramsden D, Ferguson D, Hariparsad N, Jones O et al. 2022. ADME and DMPK considerations for the discovery and development of antibody drug conjugates (ADCs). Xenobiotica. 52(8):770-785.
  • Berman CL, Antonsson M, Batkai S, Bosgra S, Chopda GR, Driessen W, Foy J, Hassan C, Hu XS, Jang HG et al. 2023. OSWG Recommended Approaches to the Nonclinical Pharmacokinetic (ADME) Characterization of Therapeutic Oligonucleotides. Nucleic Acid Ther. 33(5):287-305.
  • Beverly M, Hartsough K, Machemer L. 2005. Liquid chromatography/electrospray mass spectrometric analysis of metabolites from an inhibitory RNA duplex. Rapid Commun Mass Spectrom. 19(12):1675-1682.
  • Camperi J, Goyon A, Guillarme D, Zhang K, Stella C. 2021. Multi-dimensional LC-MS: the next generation characterization of antibody-based therapeutics by unified online bottom-up, middle-up and intact approaches. Analyst. 146(3):747-769.
  • Camperi J, Guillarme D, Stella C. 2020. Targeted Bottom-up Characterization of Recombinant Monoclonal Antibodies by Multidimensional LC/MS. Anal Chem. 92(19):13420-13426.
  • Chen B, Bartlett M. 2012. A one-step solid phase extraction method for bioanalysis of a phosphorothioate oligonucleotide and its 3' n-1 metabolite from rat plasma by uHPLC-MS/MS. AAPS J. 14(4):772-780.
  • Chernikov IV, Vlassov VV, Chernolovskaya EL. 2019. Current Development of siRNA Bioconjugates: From Research to the Clinic. Front Pharmacol. 10:444.
  • Crooke ST, Liang XH, Baker BF, Crooke RM. 2021. Antisense technology: A review. J Biol Chem. 296:100416.
  • Cuellar TL, Barnes D, Nelson C, Tanguay J, Yu SF, Wen X, Scales SJ, Gesch J, Davis D, van Brabant Smith A et al. 2015. Systematic evaluation of antibody-mediated siRNA delivery using an industrial platform of THIOMAB-siRNA conjugates. Nucleic Acids Res. 43(2):1189-1203.
  • Damen CW, Schellens JH, Beijnen JH. 2009. Bioanalytical methods for the quantification of therapeutic monoclonal antibodies and their application in clinical pharmacokinetic studies. Hum Antibodies. 18(3):47-73.
  • Donegan M, Nguyen JM, Gilar M. 2022. Effect of ion-pairing reagent hydrophobicity on liquid chromatography and mass spectrometry analysis of oligonucleotides. J Chromatogr A. 1666:462860.
  • Dong Y, Siegwart DJ, Anderson DG. 2019. Strategies, design, and chemistry in siRNA delivery systems. Adv Drug Deliv Rev. 144:133-147.
  • Dugal-Tessier J, Thirumalairajan S, Jain N. 2021. Antibody-Oligonucleotide Conjugates: A Twist to Antibody-Drug Conjugates. J Clin Med. 10(4).
  • Fire A, Xu S, Montgomery MK, Kostas SA, Driver SE, Mello CC. 1998. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature. 391(6669):806-811.
  • Furlong MT, Ouyang Z, Wu S, Tamura J, Olah T, Tymiak A, Jemal M. 2012. A universal surrogate peptide to enable LC-MS/MS bioanalysis of a diversity of human monoclonal antibody and human Fc-fusion protein drug candidates in pre-clinical animal studies. Biomed Chromatogr. 26(8):1024-1032.
  • Gorovits B, Alley SC, Bilic S, Booth B, Kaur S, Oldfield P, Purushothama S, Rao C, Shord S, Siguenza P. 2013. Bioanalysis of antibody-drug conjugates: American Association of Pharmaceutical Scientists Antibody-Drug Conjugate Working Group position paper. Bioanalysis. 5(9):997-1006.
  • Haegele JA, Boyanapalli R, Goyal J. 2022. Improvements to Hybridization-Ligation ELISA Methods to Overcome Bioanalytical Challenges Posed by Novel Oligonucleotide Therapeutics. Nucleic Acid Ther. 32(4):350-359.
  • Hu B, Zhong L, Weng Y, Peng L, Huang Y, Zhao Y, Liang XJ. 2020. Therapeutic siRNA: state of the art. Signal Transduct Target Ther. 5(1):101.
  • Huang Y, Mou S, Wang Y, Mu R, Liang M, Rosenbaum AI. 2021. Characterization of Antibody-Drug Conjugate Pharmacokinetics and in Vivo Biotransformation Using Quantitative Intact LC-HRMS and Surrogate Analyte LC-MRM. Anal Chem. 93(15):6135-6144.
  • Humphreys SC, Thayer MB, Campuzano IDG, Netirojjanakul C, Rock BM. 2019. Quantification of siRNA-Antibody Conjugates in Biological Matrices by Triplex-Forming Oligonucleotide ELISA. Nucleic Acid Ther. 29(3):161-166.
  • Jian W, Kang L, Burton L, Weng N. 2016. A workflow for absolute quantitation of large therapeutic proteins in biological samples at intact level using LC-HRMS. Bioanalysis. 8(16):1679-1691.
  • Jing X, Arya V, Reynolds KS, Rogers H. 2023. Clinical Pharmacology of RNA Interference-Based Therapeutics: A Summary Based on Food and Drug Administration-Approved Small Interfering RNAs. Drug Metab Dispos. 51(2):193-198.
  • Kaur S, Xu K, Saad OM, Dere RC, Carrasco-Triguero M. 2013. Bioanalytical assay strategies for the development of antibody-drug conjugate biotherapeutics. Bioanalysis. 5(2):201-226.
  • Kellie JF, Kehler JR, Szapacs ME. 2016. Application of high-resolution MS for development of peptide and large-molecule drug candidates. Bioanalysis. 8(3):169-177.
  • Kumar P, Ban HS, Kim SS, Wu H, Pearson T, Greiner DL, Laouar A, Yao J, Haridas V, Habiro K et al. 2008. T cell-specific siRNA delivery suppresses HIV-1 infection in humanized mice. Cell. 134(4):577-586.
  • Lanshoeft C, Cianferani S, Heudi O. 2017. Generic Hybrid Ligand Binding Assay Liquid Chromatography High-Resolution Mass Spectrometry-Based Workflow for Multiplexed Human Immunoglobulin G1 Quantification at the Intact Protein Level: Application to Preclinical Pharmacokinetic Studies. Anal Chem. 89(4):2628-2635.
  • Li H, Ortiz R, Tran LT, Salimi-Moosavi H, Malella J, James CA, Lee JW. 2013. Simultaneous analysis of multiple monoclonal antibody biotherapeutics by LC-MS/MS method in rat plasma following cassette-dosing. AAPS J. 15(2):337-346.
  • Li P, Gong Y, Kim J, Liu X, Gilbert J, Kerns HM, Groth R, Rooney M. 2020. Hybridization Liquid Chromatography-Tandem Mass Spectrometry: An Alternative Bioanalytical Method for Antisense Oligonucleotide Quantitation in Plasma and Tissue Samples. Anal Chem. 92(15):10548-10559.
  • Li X, Stuckert P, Bosch I, Marks JD, Marasco WA. 2001. Single-chain antibody-mediated gene delivery into ErbB2-positive human breast cancer cells. Cancer Gene Ther. 8(8):555-565.
  • Li XQ, Elebring M, Dahlen A, Weidolf L. 2023. In Vivo Metabolite Profiles of an N-Acetylgalactosamine-Conjugated Antisense Oligonucleotide AZD8233 Using Liquid Chromatography High-Resolution Mass Spectrometry: A Cross-Species Comparison in Animals and Humans. Drug Metab Dispos. 51(10):1350-1361.
  • Liu WL, Stevenson M, Seymour LW, Fisher KD. 2009. Quantification of siRNA using competitive qPCR. Nucleic Acids Res. 37(1):e4.
  • Malecova B, Burke RS, Cochran M, Hood MD, Johns R, Kovach PR, Doppalapudi VR, Erdogan G, Arias JD, Darimont B et al. 2023. Targeted tissue delivery of RNA therapeutics using antibody-oligonucleotide conjugates (AOCs). Nucleic Acids Res. 51(12):5901-5910.
  • Mangala LS, Rodriguez-Aguayo C, Bayraktar E, Jennings NB, Lopez-Berestein G, Sood AK. 2021. Assessment of In Vivo siRNA Delivery in Cancer Mouse Models. Methods Mol Biol. 2372:157-168.
  • McDougall R, Ramsden D, Agarwal S, Agarwal S, Aluri K, Arciprete M, Brown C, Castellanos-Rizaldos E, Charisse K, Chong S et al. 2022. The Nonclinical Disposition and Pharmacokinetic/Pharmacodynamic Properties of N-Acetylgalactosamine-Conjugated Small Interfering RNA Are Highly Predictable and Build Confidence in Translation to Human. Drug Metab Dispos. 50(6):781-797.
  • McGinnis AC, Cummings BS, Bartlett MG. 2013. Ion exchange liquid chromatography method for the direct determination of small ribonucleic acids. Anal Chim Acta. 799:57-67.
  • Mou S, Huang Y, Rosenbaum AI. 2018. ADME Considerations and Bioanalytical Strategies for Pharmacokinetic Assessments of Antibody-Drug Conjugates. Antibodies (Basel). 7(4).
  • Mu R, Yuan J, Huang Y, Meissen JK, Mou S, Liang M, Rosenbaum AI. 2022. Bioanalytical Methods and Strategic Perspectives Addressing the Rising Complexity of Novel Bioconjugates and Delivery Routes for Biotherapeutics. BioDrugs. 36(2):181-196.
  • Mullard A. 2022. Antibody-oligonucleotide conjugates enter the clinic. Nat Rev Drug Discov. 21(1):6-8.
  • Myler H, Rangan VS, Wang J, Kozhich A, Cummings JA, Neely R, Dail D, Liu A, Wang B, Vezina HE et al. 2015. An integrated multiplatform bioanalytical strategy for antibody-drug conjugates: a novel case study. Bioanalysis. 7(13):1569-1582.
  • Nair JK, Attarwala H, Sehgal A, Wang Q, Aluri K, Zhang X, Gao M, Liu J, Indrakanti R, Schofield S et al. 2017. Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc-siRNA conjugates. Nucleic Acids Res. 45(19):10969-10977.
  • Nanna AR, Kel'in AV, Theile C, Pierson JM, Voo ZX, Garg A, Nair JK, Maier MA, Fitzgerald K, Rader C. 2020. Generation and validation of structurally defined antibody-siRNA conjugates. Nucleic Acids Res. 48(10):5281-5293.
  • Paunovska K, Loughrey D, Dahlman JE. 2022. Drug delivery systems for RNA therapeutics. Nat Rev Genet. 23(5):265-280.
  • Setten RL, Rossi JJ, Han SP. 2019. The current state and future directions of RNAi-based therapeutics. Nat Rev Drug Discov. 18(6):421-446.
  • Sugo T, Terada M, Oikawa T, Miyata K, Nishimura S, Kenjo E, Ogasawara-Shimizu M, Makita Y, Imaichi S, Murata S et al. 2016. Development of antibody-siRNA conjugate targeted to cardiac and skeletal muscles. J Control Release. 237:1-13.
  • Tai TS, Tien N, Shen HY, Chu FY, Wang CCN, Lu CH, Yu HI, Kung FP, Chuang HH, Lee YR et al. 2019. Sesamin, a Naturally Occurring Lignan, Inhibits Ligand-Induced Lipogenesis through Interaction with Liver X Receptor Alpha (LXRalpha) and Pregnane X Receptor (PXR). Evid Based Complement Alternat Med. 2019:9401648.
  • Takakusa H, Iwazaki N, Nishikawa M, Yoshida T, Obika S, Inoue T. 2023. Drug Metabolism and Pharmacokinetics of Antisense Oligonucleotide Therapeutics: Typical Profiles, Evaluation Approaches, and Points to Consider Compared with Small Molecule Drugs. Nucleic Acid Ther. 33(2):83-94.
  • Tan M, Vernes JM, Chan J, Cuellar TL, Asundi A, Nelson C, Yip V, Shen B, Vandlen R, Siebel C et al. 2012. Real-time quantification of antibody-short interfering RNA conjugate in serum by antigen capture reverse transcription-polymerase chain reaction. Anal Biochem. 430(2):171-178.
  • Thayer MB, Lade JM, Doherty D, Xie F, Basiri B, Barnaby OS, Bala NS, Rock BM. 2019. Application of Locked Nucleic Acid Oligonucleotides for siRNA Preclinical Bioanalytics. Sci Rep. 9(1):3566.
  • Tian Q, Rogness J, Meng M, Li Z. 2017. Quantitative determination of a siRNA (AD00370) in rat plasma using peptide nucleic acid probe and HPLC with fluorescence detection. Bioanalysis. 9(11):861-872.
  • Toshima A, Shiraishi Y, Shinmi D, Kagawa Y, Enokizono J. 2023. Comprehensive Analyses of the Intracellular and in Vivo Disposition of Fab- Small Interfering RNA Conjugate to Identify Key Issues to Improve Its in Vivo Activity. Drug Metab Dispos. 51(3):338-347.
  • Vasicek LA, Spellman DS, Hsieh S, Seghezzi W, Zhang S, Santostefano M, Bateman KP. 2018. Quantitation of a Therapeutic Antibody in Serum Using Intact Sequential Affinity Capture, Trypsin Digestion, and LC-MS/MS. Anal Chem. 90(1):866-871.
  • Whitehead KA, Langer R, Anderson DG. 2009. Knocking down barriers: advances in siRNA delivery. Nat Rev Drug Discov. 8(2):129-138.

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.