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December 8, 2023 at 1:19 PM. The Food and Drug Administration on Friday approved a powerful treatment for sickle cell disease, a devastating illness that affects more than 100,000 Americans, the ...
Casgevy will cost $2.2 million for the one-time treatment, Vertex said in a regulatory filing, while Lyfgenia will cost $3.1 million, bluebird said in a news release.
DrugBank. DB15572. UNII. S53L777GM8. KEGG. D12749. Exagamglogene autotemcel, sold under the brand name Casgevy, is a gene therapy used for the treatment of sickle cell disease [1][3] and transfusion-dependent beta thalassemia. [1] It was developed by Vertex Pharmaceuticals and CRISPR Therapeutics.
The CRISPR treatment for LCA10 (the most common variant of Leber congenital amaurosis which is the leading cause of inherited childhood blindness) modifies the patient's defective photoreceptor gene. In March 2020, the first patient volunteer in this US-based study, sponsored by Editas Medicine , was given a low-dose of the treatment to test ...
The FDA sought the independent panel’s advice, in part, because this would be the first time the FDA would approve a treatment that uses CRISPR technology, but Dr. Fyodor Urnov, a professor in ...
In addition to CRISPR research, the IGI works to advance public understanding of CRISPR and genome engineering and guide the ethical use of these technologies. Free public resources include: CRISPRpedia — a free textbook-style resource for learning about the biology, applications, and ethics of CRISPR and genome editing, with chapters edited ...
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