Nucleic acid-based drugs for patients with solid tumours

Siegel, R. L., Miller, K. D., Wagle, N. S. & Jemal, A. Cancer statistics, 2023. CA Cancer J. Clin. 73, 17–48 (2023).

Article  PubMed  Google Scholar 

National Cancer Institute. SEER Training modules: Cancer classification. NIH training.seer.cancer.gov/disease/categories/classification.html (2023).

Sung, H. et al. Global Cancer Statistics 2020: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA Cancer J. Clin. 71, 209–249 (2021).

Article  PubMed  Google Scholar 

Hager, S., Fittler, F. J., Wagner, E. & Bros, M. Nucleic acid-based approaches for tumor therapy. Cells 9, 2061 (2020).

Article  CAS  PubMed  PubMed Central  Google Scholar 

Kulkarni, J. A. et al. The current landscape of nucleic acid therapeutics. Nat. Nanotechnol. 16, 630–643 (2021).

Article  CAS  PubMed  Google Scholar 

Paunovska, K., Loughrey, D. & Dahlman, J. E. Drug delivery systems for RNA therapeutics. Nat. Rev. Genet. 23, 265–280 (2022).

Article  CAS  PubMed  PubMed Central  Google Scholar 

Hou, X., Zaks, T., Langer, R. & Dong, Y. Lipid nanoparticles for mRNA delivery. Nat. Rev. Mater. 6, 1078–1094 (2021).

Article  CAS  PubMed  PubMed Central  Google Scholar 

Loughrey, D. & Dahlman, J. E. Non-liver mRNA delivery. Acc. Chem. Res. 55, 13–23 (2022).

Article  CAS  PubMed  Google Scholar 

Wu, Q., Qian, W., Sun, X. & Jiang, S. Small-molecule inhibitors, immune checkpoint inhibitors, and more: FDA-approved novel therapeutic drugs for solid tumors from 1991 to 2021. J. Hematol. Oncol. 15, 143 (2022).

Article  CAS  PubMed  PubMed Central  Google Scholar 

Kahvejian, A., Quackenbush, J. & Thompson, J. F. What would you do if you could sequence everything? Nat. Biotechnol. 26, 1125–1133 (2008).

Article  CAS  PubMed  PubMed Central  Google Scholar 

Qi, F., Qian, S., Zhang, S. & Zhang, Z. Single cell RNA sequencing of 13 human tissues identify cell types and receptors of human coronaviruses. Biochem. Biophys. Res. Commun. 526, 135–140 (2020).

Article  CAS  PubMed  PubMed Central  Google Scholar 

Van de Sande, B. et al. Applications of single-cell RNA sequencing in drug discovery and development. Nat. Rev. Drug Discov. 22, 496–520 (2023).

Article  PubMed  PubMed Central  Google Scholar 

Paunovska, K., Loughrey, D., Sago, C. D., Langer, R. & Dahlman, J. E. Using large datasets to understand nanotechnology. Adv. Mater. 31, e1902798 (2019).

Article  PubMed  PubMed Central  Google Scholar 

Radmand, A. et al. The transcriptional response to lung-targeting lipid nanoparticles in vivo. Nano Lett. 23, 993–1002 (2023).

Article  CAS  PubMed  PubMed Central  Google Scholar 

Zhao, Z., Anselmo, A. C. & Mitragotri, S. Viral vector-based gene therapies in the clinic. Bioeng. Transl. Med. 7, e10258 (2022).

Article  PubMed  Google Scholar 

Curreri, A., Sankholkar, D., Mitragotri, S. & Zhao, Z. RNA therapeutics in the clinic. Bioeng. Transl. Med. 8, e10374 (2023).

Article  CAS  PubMed  Google Scholar 

Baden, L. R. et al. Efficacy and safety of the mRNA-1273 SARS-CoV-2 vaccine. N. Engl. J. Med. 384, 403–416 (2021).

Article  CAS  PubMed  Google Scholar 

Polack, F. P. et al. Safety and efficacy of the BNT162b2 mRNA Covid-19 vaccine. N. Engl. J. Med. 383, 2603–2615 (2020).

Article  CAS  PubMed  Google Scholar 

Dooling, K. et al. The Advisory Committee on Immunization Practices’ updated interim recommendation for allocation of COVID-19 vaccine – United States, December 2020. MMWR Morb. Mortal. Wkly. Rep. 69, 1657–1660 (2021).

Article  CAS  PubMed  PubMed Central  Google Scholar 

Adams, D. et al. Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis. N. Engl. J. Med. 379, 11–21 (2018).

Article  CAS  PubMed  Google Scholar 

Balwani, M. et al. Phase 3 trial of RNAi therapeutic givosiran for acute intermittent porphyria. N. Engl. J. Med. 382, 2289–2301 (2020).

Article  CAS  PubMed  Google Scholar 

Lorentzen, C. L., Haanen, J. B., Met, Ö. & Svane, I. M. Clinical advances and ongoing trials on mRNA vaccines for cancer treatment. Lancet Oncol. 23, e450–e458 (2022).

Article  CAS  PubMed  PubMed Central  Google Scholar 

Mullard, A. 2020 FDA drug approvals. Nat. Rev. Drug. Discov. 20, 85–90 (2021).

Article  CAS  PubMed  Google Scholar 

Mullard, A. Cancer drug approvals and setbacks in 2021. Nat. Cancer 2, 1246–1247 (2021).

Article  PubMed  Google Scholar 

Huayamares, S. G., Lokugamage, M. P., Da Silva Sanchez, A. J. & Dahlman, J. E. A systematic analysis of biotech startups that went public in the first half of 2021. Curr. Res. Biotechnol. 4, 392–401 (2022).

Article  CAS  Google Scholar 

Barbier, A. J., Jiang, A. Y., Zhang, P., Wooster, R. & Anderson, D. G. The clinical progress of mRNA vaccines and immunotherapies. Nat. Biotechnol. 40, 840–854 (2022).

Article  CAS  PubMed  Google Scholar 

Food and Drug Administration. Cellular & gene therapy guidances. FDA https://www.fda.gov/vaccines-blood-biologics/biologics-guidances/cellular-gene-therapy-guidances (2024).

Gene therapy needs a long-term approach. Nat. Med. 27, 563 (2021).

Food and Drug Administration. Establishment of the Office of Therapeutic Products. FDA https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/establishment-office-therapeutic-products (2023).

Wang, L. L. et al. Cell therapies in the clinic. Bioeng. Transl. Med. 6, e10214 (2021).

Article  PubMed  PubMed Central  Google Scholar 

Bashor, C. J., Hilton, I. B., Bandukwala, H., Smith, D. M. & Veiseh, O. Engineering the next generation of cell-based therapeutics. Nat. Rev. Drug. Discov. 21, 655–675 (2022).

Article  CAS  PubMed  PubMed Central  Google Scholar 

Cappell, K. M. & Kochenderfer, J. N. Long-term outcomes following CAR T cell therapy: what we know so far. Nat. Rev. Clin. Oncol. 20, 359–675 (2023).

Article  CAS  PubMed  PubMed Central  Google Scholar 

Zhang, W. W. et al. The first approved gene therapy product for cancer Ad-p53 (Gendicine): 12 years in the clinic. Hum. Gene Ther. 29, 160–179 (2018).

Article  CAS  PubMed  Google Scholar 

Daley, J. Gene therapy arrives. Nature 576, S12–S13 (2019).

Article  CAS  Google Scholar 

Cattaneo, R., Miest, T., Shashkova, E. V. & Barry, M. A. Reprogrammed viruses as cancer therapeutics: targeted, armed and shielded. Nat. Rev. Microbiol. 6, 529–540 (2008).

Article  CAS  PubMed  PubMed Central  Google Scholar 

Gordon, E. M. & Hall, F. L. Noteworthy clinical case studies in cancer gene therapy: tumor-targeted Rexin-G advances as an efficacious anti-cancer agent. Int. J. Oncol. 36, 1341–1353 (2010).

Article  CAS  PubMed  Google Scholar 

Rehman, H., Silk, A. W., Kane, M. P. & Kaufman, H. L. Into the clinic: talimogene laherparepvec (T-VEC), a first-in-class intratumoral oncolytic viral therapy. J. Immunother. Cancer 4, 53 (2016).

Article  PubMed  PubMed Central  Google Scholar 

Maruyama, Y. et al. Regulatory issues: PMDA – review of Sakigake designation products: oncolytic virus therapy with delytact injection (Teserpaturev) for malignant glioma. Oncologist 28, 664–670 (2023).

Article  PubMed  PubMed Central  Google Scholar 

Food and Drug Administration. Highlights of prescribing information: ADSTILADRIN® (nadofaragene firadenovec-vncg). FDA www.fda.gov/media/164029/download (2022).

Sibbald, B. Death but one unintended consequence of gene-therapy trial. CMAJ 164, 1612 (2001).

CAS  PubMed  PubMed Central  Google Scholar 

Hacein-Bey-Abina, S. et al. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J. Clin. Invest. 118, 3132–3142 (2008).

Article  CAS  PubMed  PubMed Central 

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