문서

연구 주제

병원이 만드는 개인맞춤 치료제

신생항원 백신·펩타이드 치료제·현장 세포치료를 병원이 직접 개발·제조하는 흐름과 그 조건 — Hospital-Developed Therapeutics.

무엇이 옮겨 가고 있나

개인맞춤 치료제는 개념을 지나 임상 현실이 되었고, 그 과정에서 신약 개발의 한 부분이 제약회사에서 병원으로 옮겨 가기 시작했습니다. 세 갈래가 이 이동을 특히 선명하게 보여 줍니다.

  • 신생항원 백신 — 환자의 변이체(mutanome)에서 만든 백신이 흑색종·췌장암·삼중음성 유방암에서 수년에 걸친 T세포 면역과 무재발 생존 신호를 보이고 있습니다.
  • 펩타이드 치료제 — 합성 장쇄 펩타이드 백신, 펩타이드 수용체 방사성리간드 치료, 펩타이드-약물 접합체. 진단과 치료를 한 분자로 잇는 테라노스틱스는 이미 병원 핵의학과의 일상 진료 안에 들어와 있습니다.
  • 세포치료 — CAR-T 와 종양침윤림프구가 병원 GMP 시설에서 현장 제조(point of care) 방식으로 만들어지면서, 채혈에서 투여까지의 간격과 비용이 함께 줄고 있습니다.

왜 병원이 개발자가 되는가

셋을 관통하는 사실은 하나입니다 — 제품 하나가 곧 한 사람 분의 배치(batch of one) 라는 것. 그러면 제조자·연구자·치료 의사를 나누던 전통적인 경계가 유지되기 어렵습니다. 만드는 자리와 쓰는 자리가 같아야 시간이 맞기 때문입니다.

남은 것은 생물학이 아니다

이 연구의 결론은 분명합니다. 지금의 제약 조건은 생물학적 실현 가능성이 아니라 규제의 정합성, 제조의 표준화, 그리고 지속 가능한 재원입니다. 구체적으로는 —

  • 병원 면제(hospital exemption)와 첨단바이오의약품(ATMP) 제도가 서로 맞물리는지,
  • 학술 기관의 GMP 역량과 기술이전 경로가 실제로 받쳐 주는지,
  • 시퀀싱·바이오마커 기반과 분자종양위원회가 판단을 감당할 만큼 갖춰졌는지,
  • 그리고 환자 한 명이 곧 모집단인 시험 설계를 어떻게 평가할 것인지.

contextBio 는 무엇을 하나

이 갈래는 서비스 하나에 대응하지 않습니다 — 나머지 연구가 향하는 도착지에 가깝기 때문입니다. 환자 수준에서 시험을 계산 위에 먼저 올리는 일은 가상병원 이, 맞춤 설계될 분자는 펩타이드 신약·면역조절 펩타이드 이, 그 근거가 되는 오믹스 처리는 AURORA가 맡습니다.

참고문헌

78편

이 쪽의 근거는 리뷰 원고 Hospitals as Developers of Individualized Medicines: Neoantigen Vaccines, Peptide Therapeutics and Point of Care Cell Products 이고, 그 원고가 인용한 문헌 목록입니다.

  1. Van Eldere J, Aiuti A, Lluesma SM, Møller BK, Laursen BE, Lebbe C, et al. A roadmap for supporting the development of advanced therapy medicinal products in a European framework. Lancet Reg Health Eur. 2026;66:101688. doi:10.1016/j.lanepe.2026.101688
  2. Priesner C, Hildebrandt M. Advanced Therapy Medicinal Products and the Changing Role of Academia. Transfus Med Hemother. 2022;49(3):158-162. doi:10.1159/000524392
  3. Ott PA, Hu Z, Keskin DB, Shukla SA, Sun J, Bozym DJ, et al. An immunogenic personal neoantigen vaccine for patients with melanoma. Nature. 2017;547(7662):217-221. doi:10.1038/nature22991
  4. Sahin U, Derhovanessian E, Miller M, Kloke BP, Simon P, Löwer M, et al. Personalized RNA mutanome vaccines mobilize poly-specific therapeutic immunity against cancer. Nature. 2017;547(7662):222-226. doi:10.1038/nature23003
  5. Weber JS, Carlino MS, Khattak A, Meniawy T, Ansstas G, Taylor MH, et al. Individualised neoantigen therapy mRNA-4157 (V940) plus pembrolizumab versus pembrolizumab monotherapy in resected melanoma (KEYNOTE-942): a randomised, phase 2b study. Lancet. 2024;403(10427):632-644. doi:10.1016/S0140-6736(23)02268-7
  6. Sartor O, de Bono J, Chi KN, Fizazi K, Herrmann K, Rahbar K, et al. Lutetium-177-PSMA-617 for Metastatic Castration-Resistant Prostate Cancer. N Engl J Med. 2021;385(12):1091-1103. doi:10.1056/NEJMoa2107322
  7. Strosberg JR, Caplin ME, Kunz PL, Ruszniewski PB, Bodei L, Hendifar A, et al. 177Lu-Dotatate plus long-acting octreotide versus high‑dose long-acting octreotide in patients with midgut neuroendocrine tumours (NETTER-1): final overall survival and long-term safety results from an open-label, randomised, controlled, phase 3 trial. Lancet Oncol. 2021;22(12):1752-1763. doi:10.1016/S1470-2045(21)00572-6
  8. Oliver-Caldés A, González-Calle V, Cabañas V, Español-Rego M, Rodríguez-Otero P, Reguera JL, et al. Fractionated initial infusion and booster dose of ARI0002h, a humanised, BCMA-directed CAR T-cell therapy, for patients with relapsed or refractory multiple myeloma (CARTBCMA-HCB-01): a single-arm, multicentre, academic pilot study. Lancet Oncol. 2023;24(8):913-924. doi:10.1016/S1470-2045(23)00222-X
  9. Kekre N, Hay KA, Webb JR, Mallick R, Balasundaram M, Sigrist MK, et al. CLIC-01: Manufacture and distribution of non-cryopreserved CAR-T cells for patients with CD19 positive hematologic malignancies. Front Immunol. 2022;13:1074740. doi:10.3389/fimmu.2022.1074740
  10. Luanpitpong S, Klaihmon P, Janan M, Kungwankiattichai S, Owattanapanich W, Kunacheewa C, et al. Point-of-care manufacturing of anti-CD19 CAR-T cells using a closed production platform: Experiences of an academic in Thailand. Mol Ther Oncol. 2024;32(4):200889. doi:10.1016/j.omton.2024.200889
  11. Salazar-Riojas R, Alvarado-Navarro DM, Chávez-Estrada YO, Hernández-Navarro AK, Ake-Uc MB, Moncada-Saucedo NK, et al. Decentralized Point-of-Care Manufacturing of CD19 Chimeric Antigen Receptor T Cells in Mexico. JCO Glob Oncol. 2025;11:e2400581. doi:10.1200/GO-24-00581
  12. Keyer V, Kydyrbayeva A, Kulatay T, Zauatbayeva G, Bazhenov D, Ingirbay B, et al. Introducing CAR-T Therapy in Kazakhstan: Establishing Academic-Scale Lentiviral Vector and CAR-T Cell Production. Biomolecules. 2025;15(8). doi:10.3390/biom15081166
  13. Rojas LA, Sethna Z, Soares KC, Olcese C, Pang N, Patterson E, et al. Personalized RNA neoantigen vaccines stimulate T cells in pancreatic cancer. Nature. 2023;618(7963):144-150. doi:10.1038/s41586-023-06063-y
  14. Fried S, Shouval R, Varda-Bloom N, Besser MJ, Yerushalmi R, Shem-Tov N, et al. Point-of-care CAR T-cell therapy as salvage strategy for out-of-specification tisagenlecleucel. Leuk Lymphoma. 2022;63(14):3385-3393. doi:10.1080/10428194.2022.2123232
  15. Marchetti P, Curigliano G, Westphalen CB, Biffoni M, Lonardi S, Scagnoli S, et al. The role of the molecular tumor board: learnings from the ROME trial. NPJ Precis Oncol. 2026;10(1). doi:10.1038/s41698-026-01386-1
  16. Fountzilas E, Tsimberidou AM, Vo HH, Kurzrock R. Clinical trial design in the era of precision medicine. Genome Med. 2022;14(1):101. doi:10.1186/s13073-022-01102-1
  17. Contreras J, Herschbein L, Mathur N, Guzman ML. Access to CAR-T therapy in Latin America: Barriers, gaps, and pathways forward. Semin Immunol. 2026;82:102029. doi:10.1016/j.smim.2026.102029
  18. Shah M, Krull A, Odonnell L, de Lima MJ, Bezerra E. Promises and challenges of a decentralized CAR T-cell manufacturing model. Front Transplant. 2023;2:1238535. doi:10.3389/frtra.2023.1238535
  19. Xie N, Shen G, Gao W, Huang Z, Huang C, Fu L. Neoantigens: promising targets for cancer therapy. Signal Transduct Target Ther. 2023;8(1):9. doi:10.1038/s41392-022-01270-x
  20. Katsikis PD, Ishii KJ, Schliehe C. Challenges in developing personalized neoantigen cancer vaccines. Nat Rev Immunol. 2024;24(3):213-227. doi:10.1038/s41577-023-00937-y
  21. Blass E, Ott PA. Advances in the development of personalized neoantigen-based therapeutic cancer vaccines. Nat Rev Clin Oncol. 2021;18(4):215-229. doi:10.1038/s41571-020-00460-2
  22. Kumar A, Dixit S, Srinivasan K, M D, Vincent PMDR. Personalized cancer vaccine design using AI-powered technologies. Front Immunol. 2024;15:1357217. doi:10.3389/fimmu.2024.1357217
  23. Alburquerque-González B, López-Abellán MD, Luengo-Gil G, Montoro-García S, Conesa-Zamora P. Design of Personalized Neoantigen RNA Vaccines Against Cancer Based on Next-Generation Sequencing Data. Methods Mol Biol. 2022;2547:165-185. doi:10.1007/978-1-0716-2573-6_7
  24. Hackenbruch C, Bauer J, Heitmann JS, Maringer Y, Nelde A, Denk M, et al. FusionVAC22_01: a phase I clinical trial evaluating a DNAJB1-PRKACA fusion transcript-based peptide vaccine combined with immune checkpoint inhibition for fibrolamellar hepatocellular carcinoma and other tumor entities carrying the oncogenic driver fusion. Front Oncol. 2024;14:1367450. doi:10.3389/fonc.2024.1367450
  25. Trivedi V, Yang C, Klippel K, Yegorov O, von Roemeling C, Hoang-Minh L, et al. mRNA-based precision targeting of neoantigens and tumor-associated antigens in malignant brain tumors. Genome Med. 2024;16(1):17. doi:10.1186/s13073-024-01281-z
  26. Jiang Q, Braun DA, Clauser KR, Ramesh V, Shirole NH, Duke-Cohan JE, et al. HIF regulates multiple translated endogenous retroviruses: Implications for cancer immunotherapy. Cell. 2025;188(7):1807-1827.e34. doi:10.1016/j.cell.2025.01.046
  27. Berraondo P, Cuesta R, Sanmamed MF, Melero I. Immunogenicity and Efficacy of Personalized Adjuvant mRNA Cancer Vaccines. Cancer Discov. 2024;14(11):2021-2024. doi:10.1158/2159-8290.CD-24-1196
  28. Zaidi N, Jaffee EM, Yarchoan M. Recent advances in therapeutic cancer vaccines. Nat Rev Cancer. 2025;25(7):517-533. doi:10.1038/s41568-025-00820-z
  29. Sethna Z, Guasp P, Reiche C, Milighetti M, Ceglia N, Patterson E, et al. RNA neoantigen vaccines prime long-lived CD8+ T cells in pancreatic cancer. Nature. 2025;639(8056):1042-1051. doi:10.1038/s41586-024-08508-4
  30. Sahin U, Schmidt M, Derhovanessian E, Cortini A, Vogler I, Omokoko T, et al. Individualized mRNA vaccines evoke durable T cell immunity in adjuvant TNBC. Nature. 2026;651(8107):1088-1096. doi:10.1038/s41586-025-10004-2
  31. Blass E, Keskin DB, Tu CR, Forman C, Vanasse A, Sax HE, et al. A multi-adjuvant personal neoantigen vaccine generates potent immunity in melanoma. Cell. 2025;188(19):5125-5141.e27. doi:10.1016/j.cell.2025.06.019
  32. Fan T, Xu C, Wu J, Cai Y, Cao W, Shen H, et al. Lipopolyplex-formulated mRNA cancer vaccine elicits strong neoantigen-specific T cell responses and antitumor activity. Sci Adv. 2024;10(41):eadn9961. doi:10.1126/sciadv.adn9961
  33. Mendez-Gomez HR, DeVries A, Castillo P, von Roemeling C, Qdaisat S, Stover BD, et al. RNA aggregates harness the danger response for potent cancer immunotherapy. Cell. 2024;187(10):2521-2535.e21. doi:10.1016/j.cell.2024.04.003
  34. Liu C, Liu X, Xiang X, Pang X, Chen S, Zhang Y, et al. A nanovaccine for antigen self-presentation and immunosuppression reversal as a personalized cancer immunotherapy strategy. Nat Nanotechnol. 2022;17(5):531-540. doi:10.1038/s41565-022-01098-0
  35. Yao R, Xie C, Xia X. Recent progress in mRNA cancer vaccines. Hum Vaccin Immunother. 2024;20(1):2307187. doi:10.1080/21645515.2024.2307187
  36. Chandra S, Wilson JC, Good D, Wei MQ. mRNA vaccines: a new era in vaccine development. Oncol Res. 2024;32(10):1543-1564. doi:10.32604/or.2024.043987
  37. Grippin AJ, Marconi C, Copling S, Li N, Braun C, Woody C, et al. SARS-CoV-2 mRNA vaccines sensitize tumours to immune checkpoint blockade. Nature. 2025;647(8089):488-497. doi:10.1038/s41586-025-09655-y
  38. Naghavian R, Faigle W, Oldrati P, Wang J, Toussaint NC, Qiu Y, et al. Microbial peptides activate tumour-infiltrating lymphocytes in glioblastoma. Nature. 2023;617(7962):807-817. doi:10.1038/s41586-023-06081-w
  39. Yang X, Garner LI, Zvyagin IV, Paley MA, Komech EA, Jude KM, et al. Autoimmunity-associated T cell receptors recognize HLA-B*27-bound peptides. Nature. 2022;612(7941):771-777. doi:10.1038/s41586-022-05501-7
  40. Muttenthaler M, King GF, Adams DJ, Alewood PF. Trends in peptide drug discovery. Nat Rev Drug Discov. 2021;20(4):309-325. doi:10.1038/s41573-020-00135-8
  41. Kuo PH, Morris MJ, Hesterman J, Kendi AT, Rahbar K, Wei XX, et al. Quantitative 68Ga-PSMA-11 PET and Clinical Outcomes in Metastatic Castration-resistant Prostate Cancer Following 177Lu-PSMA-617 (VISION Trial). Radiology. 2024;312(2):e233460. doi:10.1148/radiol.233460
  42. Armstrong AJ, Sartor O, de Bono J, Chi K, Fizazi K, Krause BJ, et al. Association of Declining Prostate-specific Antigen Levels with Clinical Outcomes in Patients with Metastatic Castration-resistant Prostate Cancer Receiving [177Lu]Lu-PSMA-617 in the Phase 3 VISION Trial. Eur Urol. 2024;86(6):552-562. doi:10.1016/j.eururo.2024.08.021
  43. Gafita A, Martin AJ, Emmett L, Eiber M, Iravani A, Fendler WP, et al. Validation of Prognostic and Predictive Models for Therapeutic Response in Patients Treated with [177Lu]Lu-PSMA-617 Versus Cabazitaxel for Metastatic Castration-resistant Prostate Cancer (TheraP): A Post Hoc Analysis from a Randomised, Open-label, Phase 2 Trial. Eur Urol Oncol. 2025;8(1):21-28. doi:10.1016/j.euo.2024.03.009
  44. Calais J, Czernin J, Thin P, Gartmann J, Nguyen K, Armstrong WR, et al. Safety of PSMA-Targeted Molecular Radioligand Therapy with 177Lu-PSMA-617: Results from the Prospective Multicenter Phase 2 Trial RESIST-PC (NCT03042312). J Nucl Med. 2021;62(10):1447-1456. doi:10.2967/jnumed.121.262543
  45. Belabaci Z, Schmidt L, Sleiay M, Couñago F, López Campos F, Tolba M, et al. Efficacy and safety of rechallenge therapy with [177Lu]Lu-PSMA in metastatic castration-resistant prostate cancer: a systematic review and meta-analysis. Eur J Nucl Med Mol Imaging. 2025;53(1):93-104. doi:10.1007/s00259-025-07438-1
  46. Munzur AD, Herberts C, Kwan EM, Emmett L, Sandhu S, Buteau JP, et al. Clonal Hematopoiesis after 177Lu-PSMA-617 Radioligand Therapy in Prostate Cancer. Clin Cancer Res. 2026;32(13):2644-2652. doi:10.1158/1078-0432.CCR-25-4001
  47. Alas M, Saghaeidehkordi A, Kaur K. Peptide-Drug Conjugates with Different Linkers for Cancer Therapy. J Med Chem. 2021;64(1):216-232. doi:10.1021/acs.jmedchem.0c01530
  48. Wang M, Liu J, Xia M, Yin L, Zhang L, Liu X, et al. Peptide-drug conjugates: A new paradigm for targeted cancer therapy. Eur J Med Chem. 2024;265:116119. doi:10.1016/j.ejmech.2023.116119
  49. Armstrong A, Coburn F, Nsereko Y, Al Musaimi O. Peptide-Drug Conjugates: A New Hope for Cancer. J Pept Sci. 2025;31(8):e70040. doi:10.1002/psc.70040
  50. Javid H, Oryani MA, Rezagholinejad N, Esparham A, Tajaldini M, Karimi-Shahri M. RGD peptide in cancer targeting: Benefits, challenges, solutions, and possible integrin-RGD interactions. Cancer Med. 2024;13(2):e6800. doi:10.1002/cam4.6800
  51. Petruzzella A, Bruand M, Santamaria-Martínez A, Katanayeva N, Reymond L, Wehrle S, et al. Antibody-peptide conjugates deliver covalent inhibitors blocking oncogenic cathepsins. Nat Chem Biol. 2024;20(9):1188-1198. doi:10.1038/s41589-024-01627-z
  52. Rohaan MW, Borch TH, van den Berg JH, Met Ö, Kessels R, Geukes Foppen MH, et al. Tumor-Infiltrating Lymphocyte Therapy or Ipilimumab in Advanced Melanoma. N Engl J Med. 2022;387(23):2113-2125. doi:10.1056/NEJMoa2210233
  53. Chesney J, Lewis KD, Kluger H, Hamid O, Whitman E, Thomas S, et al. Efficacy and safety of lifileucel, a one-time autologous tumor-infiltrating lymphocyte (TIL) cell therapy, in patients with advanced melanoma after progression on immune checkpoint inhibitors and targeted therapies: pooled analysis of consecutive cohorts of the C-144-01 study. J Immunother Cancer. 2022;10(12). doi:10.1136/jitc-2022-005755
  54. Medina T, Chesney JA, Kluger HM, Hamid O, Whitman ED, Cusnir M, et al. Long-Term Efficacy and Safety of Lifileucel Tumor-Infiltrating Lymphocyte Cell Therapy in Patients With Advanced Melanoma: A 5-Year Analysis of the C-144-01 Study. J Clin Oncol. 2025;43(33):3565-3572. doi:10.1200/JCO-25-00765
  55. Betof Warner A, Hamid O, Komanduri K, Amaria R, Butler MO, Haanen J, et al. Expert consensus guidelines on management and best practices for tumor-infiltrating lymphocyte cell therapy. J Immunother Cancer. 2024;12(2). doi:10.1136/jitc-2023-008735
  56. Jain H, Karulkar A, Kalra D, Ravikumar S, Shah S, Firfiray A, et al. Talicabtagene autoleucel for relapsed or refractory B-cell malignancies: results from an open-label, multicentre, phase 1/2 study. Lancet Haematol. 2025;12(4):e282-e293. doi:10.1016/S2352-3026(24)00377-6
  57. Narula G, Keerthivasagam S, Jain H, Punatar S, Chichra A, Dhamne C, et al. Novel humanized CD19-CAR-T (Now talicabtagene autoleucel, Tali-cel™) cells in relapsed/ refractory pediatric B-acute lymphoblastic leukemia- an open-label single-arm phase-I/Ib study. Blood Cancer J. 2025;15(1):75. doi:10.1038/s41408-025-01279-9
  58. Gupta D, Shaz B. Surveying local CAR T-cell manufacturing processes to facilitate standardization and expand accessibility. J Transl Med. 2025;23(1):507. doi:10.1186/s12967-025-06400-x
  59. Chiesa R, Georgiadis C, Syed F, Zhan H, Etuk A, Gkazi SA, et al. Base-Edited CAR7 T Cells for Relapsed T-Cell Acute Lymphoblastic Leukemia. N Engl J Med. 2023;389(10):899-910. doi:10.1056/NEJMoa2300709
  60. Chiesa R, Georgiadis C, Rashed H, Preece R, Hardefeldt P, Chu J, et al. Universal Base-Edited CAR7 T Cells for T-Cell Acute Lymphoblastic Leukemia. N Engl J Med. 2026;394(2):152-165. doi:10.1056/NEJMoa2505478
  61. Mamia K, Kolbeinsdottir S, Labun K, Li Z, Komisarczuk A, Keskitalo S, et al. Precision T cell correction platform for inborn errors of immunity. Mol Ther. 2025;33(11):5624-5643. doi:10.1016/j.ymthe.2025.08.018
  62. Giorgioni L, Ambrosone A, Cometa MF, Salvati AL, Nisticò R, Magrelli A. Revolutionizing CAR T-Cell Therapies: Innovations in Genetic Engineering and Manufacturing to Enhance Efficacy and Accessibility. Int J Mol Sci. 2024;25(19). doi:10.3390/ijms251910365
  63. Kaminskiy Y, Degtyarev V, Stepanov A, Maschan M. CAR T cell therapy for central nervous system solid tumors: current progress and future directions. Front Immunol. 2025;16:1600403. doi:10.3389/fimmu.2025.1600403
  64. Hills A, Awigena-Cook J, Genenz K, Ostertag M, Butler S, Eggimann AV, et al. An assessment of the hospital exemption landscape across European Member States: regulatory frameworks, use and impact. Cytotherapy. 2020;22(12):772-779.e1. doi:10.1016/j.jcyt.2020.08.011
  65. Coppens DGM, Hoekman J, De Bruin ML, Slaper-Cortenbach ICM, Leufkens HGM, Meij P, et al. Advanced therapy medicinal product manufacturing under the hospital exemption and other exemption pathways in seven European Union countries. Cytotherapy. 2020;22(10):592-600. doi:10.1016/j.jcyt.2020.04.092
  66. Coppens DG, Gardarsdottir H, Bruin ML, Meij P, Gm Leufkens H, Hoekman J. Regulating advanced therapy medicinal products through the Hospital Exemption: an analysis of regulatory approaches in nine EU countries. Regen Med. 2020;15(8):2015-2028. doi:10.2217/rme-2020-0008
  67. Johanna I, Daudeij A, Devina F, Nijenhuis C, Nuijen B, Romberg B, et al. Basics of advanced therapy medicinal product development in academic pharma and the role of a GMP simulation unit. Immunooncol Technol. 2023;20:100411. doi:10.1016/j.iotech.2023.100411
  68. Marolt Presen D, Lainšček D, Kinghorn J, Sebestyen Z, Kuball J, Amini L, et al. CTGCT, Centre of Excellence for the Technologies of Gene and Cell Therapy: Collaborative translation of scientific discoveries into advanced treatments for neurological rare genetic diseases and cancer. Comput Struct Biotechnol J. 2025;27:10-16. doi:10.1016/j.csbj.2024.11.051
  69. Pakzad M, Hassani SN, Abbasi F, Hajizadeh-Saffar E, Taghiyar L, Fallah N, et al. A Roadmap for the Production of a GMP-Compatible Cell Bank of Allogeneic Bone Marrow-Derived Clonal Mesenchymal Stromal Cells for Cell Therapy Applications. Stem Cell Rev Rep. 2022;18(7):2279-2295. doi:10.1007/s12015-022-10351-x
  70. Barker RA, Carpenter M, Jamieson CHM, Murry CE, Pellegrini G, Rao RC, et al. Lessons learnt, and still to learn, in first in human stem cell trials. Stem Cell Reports. 2023;18(8):1599-1609. doi:10.1016/j.stemcr.2022.11.019
  71. Lau LMS, Khuong-Quang DA, Mayoh C, Wong M, Barahona P, Ajuyah P, et al. Precision-guided treatment in high-risk pediatric cancers. Nat Med. 2024;30(7):1913-1922. doi:10.1038/s41591-024-03044-0
  72. Nikanjam M, Kato S, Kurzrock R. Liquid biopsy: current technology and clinical applications. J Hematol Oncol. 2022;15(1):131. doi:10.1186/s13045-022-01351-y
  73. Kim J, Hu C, Moufawad El Achkar C, Black LE, Douville J, Larson A, et al. Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease. N Engl J Med. 2019;381(17):1644-1652. doi:10.1056/NEJMoa1813279
  74. Musunuru K, Grandinette SA, Wang X, Hudson TR, Briseno K, Berry AM, et al. Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease. N Engl J Med. 2025;392(22):2235-2243. doi:10.1056/NEJMoa2504747
  75. Aartsma-Rus A, Garanto A, van Roon-Mom W, McConnell EM, Suslovitch V, Yan WX, et al. Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides. Nucleic Acid Ther. 2023;33(1):17-25. doi:10.1089/nat.2022.0060
  76. Lemaitre MM. Individualized Antisense Oligonucleotide Therapies: How to Approach the Challenge of Manufacturing These Oligos from a Chemistry, Manufacturing, and Control-Regulatory Standpoint. Nucleic Acid Ther. 2022;32(2):101-110. doi:10.1089/nat.2021.0030
  77. Synofzik M, van Roon-Mom WMC, Marckmann G, van Duyvenvoorde HA, Graessner H, Schüle R, et al. Preparing n-of-1 Antisense Oligonucleotide Treatments for Rare Neurological Diseases in Europe: Genetic, Regulatory, and Ethical Perspectives. Nucleic Acid Ther. 2022;32(2):83-94. doi:10.1089/nat.2021.0039
  78. Aartsma-Rus A, de Gusmao CM, Demarest S, van Roon-Mom W, Graessner H, Bush LW, et al. Individualized antisense oligonucleotide treatment eligibility of patients living with neurodevelopmental diseases. Cell Rep Med. 2026;7(9):102989. doi:10.1016/j.xcrm.2026.102989

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