Antibody–siRNA conjugates (ARC): Emerging siRNA drug formulation

Huggins, 2019, Site selective antibody-oligonucleotide conjugation via microbial transglutaminase, Molecules, 24, 10.3390/molecules24183287 Liu, 2017, Responsive nanocarriers as an emerging platform for cascaded delivery of nucleic acids to cancer, Adv Drug Deliv Rev, 115, 98, 10.1016/j.addr.2017.03.004 Cortes, 2020, Prevention, recognition, and management of adverse events associated with gemtuzumab ozogamicin use in acute myeloid leukemia, J Hematol Oncol, 13, 137, 10.1186/s13045-020-00975-2 Goldenson, 2021, Gemtuzumab ozogamicin for the treatment of acute myeloid leukemia in adults, Expert Opin Biol Ther, 21, 849, 10.1080/14712598.2021.1825678 Choi, 2020, Advances in therapy for relapsed or refractory Hodgkin lymphoma, Curr Oncol Rep, 22, 6, 10.1007/s11912-020-0866-3 Chen, 2013, Targeted therapy for Hodgkin lymphoma and systemic anaplastic large cell lymphoma: focus on brentuximab vedotin, Onco Targets Ther, 7, 45 Amiri-Kordestani, 2014, FDA approval: ado-trastuzumab emtansine for the treatment of patients with HER2-positive metastatic breast cancer, Clin Cancer Res, 20, 4436, 10.1158/1078-0432.CCR-14-0012 Khongorzul, 2020, Antibody-drug conjugates: a compreheNSIVE REView, Mol Cancer Res, 18, 3, 10.1158/1541-7786.MCR-19-0582 Lambert, 2018, Antibody-drug conjugates for cancer treatment, Annu Rev Med, 69, 191, 10.1146/annurev-med-061516-121357 Yarian, 2019, Antibody-drug therapeutic conjugates: Potential of antibody-siRNAs in cancer therapy, J Cell Physiol, 234, 16724, 10.1002/jcp.28490 Bai, 2019, Redox-responsive polymeric RNAi based on multivalent conjugation of siRNA for improved intracellular delivery, Bioconjug Chem, 30, 2777, 10.1021/acs.bioconjchem.9b00680 Manzano, 2020, Antibody-drug conjugates: A promising novel therapy for the treatment of ovarian cancer, Cancers (Basel), 12, 10.3390/cancers12082223 Dennler, 2014, Transglutaminase-based chemo-enzymatic conjugation approach yields homogeneous antibody-drug conjugates, Bioconjug Chem, 25, 569, 10.1021/bc400574z Donaghy, 2016, Effects of antibody, drug and linker on the preclinical and clinical toxicities of antibody-drug conjugates, MAbs, 8, 659, 10.1080/19420862.2016.1156829 Mehrling, 2018, Challenges in optimising the successful construction of antibody drug conjugates in cancer therapy, Antibodies (Basel), 7 Dovgan, 2019, Antibody-oligonucleotide conjugates as therapeutic, imaging, and detection agents, Bioconjug Chem, 30, 2483, 10.1021/acs.bioconjchem.9b00306 Zhao, 2020, Recent advances of antibody drug conjugates for clinical applications, Acta Pharm Sin B, 10, 1589, 10.1016/j.apsb.2020.04.012 Behrens, 2015, Antibody-drug conjugates (ADCs) derived from interchain cysteine cross-linking demonstrate improved homogeneity and other pharmacological properties over conventional heterogeneous ADCs, Mol Pharm, 12, 3986, 10.1021/acs.molpharmaceut.5b00432 Junutula, 2010, Engineered thio-trastuzumab-DM1 conjugate with an improved therapeutic index to target human epidermal growth factor receptor 2-positive breast cancer, Clin Cancer Res, 16, 4769, 10.1158/1078-0432.CCR-10-0987 Li, 2017, Stable and potent selenomab-drug conjugates, Cell Chem Biol, 24, 433, 10.1016/j.chembiol.2017.02.012 Okeley, 2013, Metabolic engineering of monoclonal antibody carbohydrates for antibody-drug conjugation, Bioconjug Chem, 24, 1650, 10.1021/bc4002695 Amani, 2020, ADCs, as novel revolutionary weapons for providing a step forward in targeted therapy of malignancies, Curr Drug Deliv, 17, 23, 10.2174/1567201816666191121145109 Lee, 2012, Cell-specific siRNA delivery by peptides and antibodies, Methods Enzymol, 502, 91, 10.1016/B978-0-12-416039-2.00005-7 Tatiparti, 2017, siRNA delivery strategies: A comprehensive review of recent developments, Nanomaterials (Basel), 7, 10.3390/nano7040077 Ibtehaj, 2017, High-dose BAFF receptor specific mAb-siRNA conjugate generates Fas-expressing B cells in lymph nodes and high-affinity serum autoantibody in a myasthenia mouse model, Clin Immunol, 176, 122, 10.1016/j.clim.2017.01.005 Kumar, 2008, T cell-specific siRNA delivery suppresses HIV-1 infection in humanized mice, Cell, 134, 577, 10.1016/j.cell.2008.06.034 Hoy, 2018, Patisiran: first global approval, Drugs, 78, 1625, 10.1007/s40265-018-0983-6 Scott, 2020, Givosiran: first approval, Drugs, 80, 335, 10.1007/s40265-020-01269-0 Baumer, 2017, Immunoprotein-mediated siRNA delivery, Mol Pharm, 14, 1339, 10.1021/acs.molpharmaceut.6b01039 Chen, 2018, RNA interference-based therapy and its delivery systems, Cancer Metastasis Rev, 37, 107, 10.1007/s10555-017-9717-6 Shi, 2019, Therapeutic effects of human monoclonal PSMA antibody-mediated TRIM24 siRNA delivery in PSMA-positive castration-resistant prostate cancer, Theranostics, 9, 1247, 10.7150/thno.29884 Baumer, 2016, Antibody-coupled siRNA as an efficient method for in vivo mRNA knockdown, Nat Protoc, 11, 22, 10.1038/nprot.2015.137 Baumer, 2015, Antibody-mediated delivery of anti-KRAS-siRNA in vivo overcomes therapy resistance in colon cancer, Clin Cancer Res, 21, 1383, 10.1158/1078-0432.CCR-13-2017 Mehta, 2015, A new approach for the treatment of arthritis in mice with a novel conjugate of an anti-C5aR1 antibody and C5 small interfering RNA, J Immunol, 194, 5446, 10.4049/jimmunol.1403012 Song, 2005, Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors, Nat Biotechnol, 23, 709, 10.1038/nbt1101 Su, 2013, PSMA specific single chain antibody-mediated targeted knockdown of Notch1 inhibits human prostate cancer cell proliferation and tumor growth, Cancer Lett, 338, 282, 10.1016/j.canlet.2013.05.035 Ye, 2017, High-yield synthesis of monomeric LMWP (CPP)-siRNA covalent conjugate for effective cytosolic delivery of siRNA, Theranostics, 7, 2495, 10.7150/thno.19863 Yu, 2018, Improved method for synthesis of low molecular weight protamine-siRNA conjugate, Acta Pharmaceutica Sinica B, 8, 116, 10.1016/j.apsb.2017.11.011 Rengaswamy, 2016, RGD liposome-protamine-siRNA (LPR) nanoparticles targeting PAX3-FOXO1 for alveolar rhabdomyosarcoma therapy, J Control Release, 235, 319, 10.1016/j.jconrel.2016.05.063 Cuellar, 2015, Systematic evaluation of antibody-mediated siRNA delivery using an industrial platform of THIOMAB-siRNA conjugates, Nucleic Acids Res, 43, 1189, 10.1093/nar/gku1362 Shim, 2010, Efficient and targeted delivery of siRNA in vivo, FEBS J, 277, 4814, 10.1111/j.1742-4658.2010.07904.x Tushir-Singh, 2016, Antibody-siRNA conjugates: drugging the undruggable for anti-leukemic therapy, Expert Opin Biol Ther, 17, 325, 10.1080/14712598.2017.1273344 Sugo, 2016, Development of antibody-siRNA conjugate targeted to cardiac and skeletal muscles, J Control Release, 237, 1, 10.1016/j.jconrel.2016.06.036 Nanna, 2020, Generation and validation of structurally defined antibody-siRNA conjugates, Nucleic Acids Res, 48, 5281, 10.1093/nar/gkaa286 Im, 2017, Variable heavy chain domain derived from a cell-penetrating anti-DNA monoclonal antibody for the intracellular delivery of biomolecules, Immunol Invest, 46, 500, 10.1080/08820139.2017.1301466 Hauser, 2010, Novel siRNA delivery system to target podocytes in vivo, PLoS ONE, 5, 10.1371/journal.pone.0009463 Xia, 2007, Intravenous siRNA of brain cancer with receptor targeting and avidin-biotin technology, Pharm Res, 24, 2309, 10.1007/s11095-007-9460-8 Xia, 2009, Antibody-mediated targeting of siRNA via the human insulin receptor using avidin-biotin technology, Mol Pharm, 6, 747, 10.1021/mp800194y Craig, 2018, Recent preclinical and clinical advances in oligonucleotide conjugates, Expert Opin Drug Deliv, 15, 629, 10.1080/17425247.2018.1473375 Ye, 2018, Cellular uptake mechanism and comparative in vitro cytotoxicity studies of monomeric LMWP-siRNA conjugate, J Ind Eng Chem, 63, 103, 10.1016/j.jiec.2018.02.005 Tietz, 2022, Tricyclic cell-penetrating peptides for efficient delivery of functional antibodies into cancer cells, Nat Chem, 14, p. 284-+, 10.1038/s41557-021-00866-0 Yu, 2021, Antibody-siRNA conjugates (ARCs) using multifunctional peptide as a tumor enzyme cleavable linker mediated effective intracellular delivery of siRNA, Int J Pharm, 606, 10.1016/j.ijpharm.2021.120940 Guo, 2010, Engineering RNA for targeted siRNA delivery and medical application, Adv Drug Deliv Rev, 62, 650, 10.1016/j.addr.2010.03.008