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Abstract
Keywords
Abstract
Cell and gene therapy is a method used to treat genetically based diseases that currently have no effective treatments. The number of products available for cell and gene therapy has been rising annually in recent years. By 2025, the FDA predicts that it will have approved 10 to 20 novel cell and gene therapy products annually. The FDA's current list of authorized gene treatments includes 26 gene and cell-based products. The FDA and EMA have approved 8 cell-based and gene therapy products in 2022. We summarized the gene therapy medications authorized in 2022 in this review. Two gene therapy drugs (Skysona and Zynteglo) that were approved this year were previously approved and stopped marketing in Europe since the European market price could not be agreed upon. This year, the FDA approved the use of these two medications.
Keywords
- 1) Kaji EH, Leiden JM. Gene and Stem Cell Therapies. JAMA. 2001;285(5):545–550. doi:10.1001/jama.285.5.545
- 2) Center for Biologics Evaluation and Research. (n.d.). What isgene therapy? U.S. Food and Drug Administration. RetrievedDecember 13, 2022, from https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/what-gene-therapy
- 3) Center for Biologics Evaluation and Research. (n.d.).Approved cellular and gene therapy products. U.S. Food andDrug Administration. Retrieved December 13, 2022, from https://www.fda.gov/vaccines-blood-biologics/cellular-gene-
- therapy-products/approved-cellular-and-gene-therapy-products4) Yıldırım, S., & Kocabaş, F. (2021). Gene therapy productsreached to market by 2021. Gene Editing, 2(2), 1–21. https://doi.org/10.29228/genediting.54101
- 5) Cristina Santoro, Gabriele Quinta Valle, Erminia Baldacci,Antonietta Ferrett, Federica Riccardi: Inhibitors in Hemophilia B, Semin Thromb Hemost 2018; 44(06): 578-589
- 6) Biggs R, Douglas AS, Macfarlane RG, Dacie JV, Pitney WR,Merskey C. Christmas disease – a condition previously mistaken for haemophilia. Br Med J 1952; 2: 1378–82.
- 7) Goodeve, A. C. (2015). Hemophilia b: MolecularPathogenesis and mutation analysis. Journal of Thrombosis andHaemostasis, 13(7), 1184–1195. https://doi.org/10.1111/jth.12958
- 8) Annette Von Drygalski, Adam Giermasz, GiancarloCastaman, Nigel S. Key, Susan Lattimore, Frank W. G. Leebeek,Wolfgang Miesbach, Michael Recht Alison Long, Robert Gut,Eileen K. Sawyer, Steven W. Pipe, Etranacogene dezaparvovec(AMT-061 phase 2b): normal/near normal fix activity and bleed cessation in hemophilia B, OCTOBER 30, 2019.
- 9) Courtney D. Thornburg, Etranacogene dezaparvovec forhemophilia B gene therapy, First published online on November 24, 2021.
- 10) Mr. Sachin Navale, Miss. Kaveri Bhosale, Mr. MaheshMohite, & Mrs. Swapnali Navale. (2022). Hemgenix as firstgene therapy for treatment of haemophilia B. InternationalJournal of Advanced Research in Science, Communication and Technology, 89–94. https://doi.org/10.48175/ijarsct-7657
- December 8, 2022, from https://www.fda.gov/vaccines-blood- biologics/vaccines/hemgenix
- 12) Naddaf, M. (2022, December 6). Researchers welcome $3.5-million haemophilia gene therapy - but questions remain. NatureNews. Retrieved December 8, 2022, from https://www.nature.com/articles/d41586-022-04327-7
- 13) Mannucci PM, Tuddenham EGD. The hemophiliac – fromroyal genes to gene therapy. N Engl J Med. 2001;344:1773–1779. doi: 10.1056/NEJM200106073442307.
- 14) Sheridan, C. For hemophilia and thalassemia, a new era of‘one-and-done’ gene therapies has arrived. Nat Biotechnol 40,1531–1533 (2022). https://doi.org/10.1038/s41587-022-01555-0
- 15) Ema. (2022, November 9). Roctavian. European MedicinesAgency. Retrieved December 8, 2022, fromhttps://www.ema.europa.eu/en/medicines/human/EPAR/roctavi an-0
- 16) ICER publishes final report and policy recommendations forhemophilia a therapies. ICER. (2022, January 11). RetrievedDecember 8, 2022, from https://icer.org/news-insights/press- releases/icer-publishes-final-report-and-policy-
- 17) Roctavian: Withdrawn Application | European MedicinesAgency. (n.d.). Retrieved December 8, 2022, fromhttps://www.ema.europa.eu/en/medicines/human/withdrawn- applications/roctavian
- 18) Himmelreich N, Montioli R, Bertoldi M, et al. Aromaticamino acid decarboxylase defciency: molecular and metabolicbasis and therapeutic outlook. Mol Genet Metab. 2019;127(1):12–22.
- 19) Hwu PW, Kiening K, Anselm I, et al. Gene therapy in theputamen for curing AADC defciency and Parkinson’s disease. EMBO Mol Med. 2021;13(9): e14712.
- 20) Ema. (2022, October 24). Upstaza. European Medicineshttps://www.ema.europa.eu/en/medicines/human/EPAR/upstaza21) European Medicines Agency. (n.d.). Retrieved December 8,2022, from https://www.ema.europa.eu/en/documents/assessment-
- 22) Chien YH, Lee NC, Tseng SH, et al. Efcacy and safety ofAAV2 gene therapy in children with aromatic L-amino aciddecarboxylase defciency: an open-label, phase 1/2 trial. Lancet Child Adolesc Health. 2017;1(4):265–73
- 23) Keam, S. J. (2022). Eladocagene Exuparvovec: First approval. Drugs. https://doi.org/10.1007/s40265-022-01775-3
- 24) Lichtman MA. The Non-Hodgkin Lymphomas. WilliamsHematology, 7th ed. 2005;1407– 1459. New York: McGraw-Hill.
- 25) Armitage JO, Weisenburger DD. New approach to classifyingnon-Hodgkin’s lymphomas: clinical features of the majorhistologic subtypes. Non-Hodgkin’s Lymphoma Classification Project. J Clin Oncol. 1998;16:2780-2795.
- Medicines Agency. (n.d.). Retrieved December 9, 2022, from https://www.ema.europa.eu/en/documents/product-
- 27) Ema. (2022, May 20). Breyanzi. European Medicines Agency.Retrieved December 9, 2022, fromhttps://www.ema.europa.eu/en/medicines/human/EPAR/breyan zi
- 28) Ema. (2020, November 3). European public assessmentreports: Background and context - medicines agency. EuropeanMedicines Agency. Retrieved December 9, 2022, from https://www.ema.europa.eu/en/medicines/what-we-publish-
- 29) Raab MS, Podar K, Breitkreutz I, Richardson PG, AndersonKC. Multiple myeloma. Lancet. 2009 Jul 25;374(9686):324-39.doi: 10.1016/S0140-6736(09)60221-X. Epub 2009 Jun 21. PMID: 19541364.
- 30) Ema. (2022, September 28). Carvykti. European Medicineshttps://www.ema.europa.eu/en/medicines/human/EPAR/carvykt i
- 33) Ema. (2022, August 24). Abecma. European Medicineshttps://www.ema.europa.eu/en/medicines/human/EPAR/abecma
