Siegel, R. L., Miller, K. D., Wagle, N. S. & Jemal, A. Cancer statistics, 2023. CA Cancer J. Clin. 73, 17–48 (2023).
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).
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).
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).
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).
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).
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).
Comments (0)