The first genome-edited cell product receives FDA approval
https://doi.org/10.60043/2949-5938-2023-2-55-59
Abstract
The promise of genome editing technologies (GETs) for gene therapy is one of the key drivers for development of this field to treat monogenic hereditary diseases. However, the general consensus of research community and many physicians is that in some cases the balance of risk and benefit of GET-based drugs and CRISPR/Cas9, in particular, has not been fully studied to date. However, on December 03, 2023, the US Food and Drug Administration (FDA) approved marketing of two products for sickle cell disease. One of them (“Casgevy”) is a cellular product consisting of CD34+ autologous hematopoietic stem cells, in which, using the CRISPR/Cas9 system, it was possible to increase the production of fetal hemoglobin, which leads to compensation of patient’s condition. Thus, for the first time in world practice, national regulator has approved not just a genetically modified cell product, but a product obtained using GET. This short message is dedicated to this historical event and its importance, as well as possible consequences.
Supporting agencies: The work was carried under state assignment of Lomonosov Moscow State University and the prospective direction of scientific and educational development of M.V. Lomonosov Moscow State University “Molecular technologies of living systems and synthetic biology”
About the Author
P. I. Makarevich
Lomonosov Moscow State University
Russian Federation
Russian Federation
Pavel I. Makarevich — M.D., Ph.D., Laboratory of Gene and Cell Therapy, Institute for Regenerative Medicine, Medical Research and Education Center
Moscow, Leninskiye Gory, 1,
References
1. U.S. Food and drug administration (2023). https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease [Accessed December, 12, 2024].
2. Румянцев А.Г., Токарев Ю.Н., Сметанина Н.С. Гемоглобинопатии и талассемические синдромы. М.: Практическая медицина, 2015. 448 с.
3. CASGEVY (exagamglogene autotemcel), suspension for intravenous infusion (2023). https://www.fda.gov/media/174615/download?attachment [Accessed December, 12, 2024].
4. Cancellieri S, Zeng J, Lin LY, et al. Human genetic diversity alters off-target outcomes of therapeutic gene editing. Nat Genet. 2023; 55(1):34–43.
5. Livshits G. Landmark CRISPR Approvals and the Rise of Epigenome Editing. GEN Biotechnology. 2023;2(6):476–478.
6. Макаревич П.И. Три десятилетия развития генной терапии: вехи и перспективы. Регенерация органов и тканей. 2023;1(1):16–24.
7. Intellia Therapeutics Announces Positive Interim Clinical Data for its Second Systemically Delivered Investigational CRISPR Candidate, NTLA-2002 for the Treatment of Hereditary Angioedema (HAE) (2022). https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-announces-positive-interim-clinical-data [Accessed December, 12, 2024].
Review
For citations:
Makarevich P.I. The first genome-edited cell product receives FDA approval. Регенерация органов и тканей. 2023;1(2):55-59. (In Russ.) https://doi.org/10.60043/2949-5938-2023-2-55-59
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