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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 ...
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.
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.
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 ...
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 price for the treatment was set at €594,000, twice the annual cost of enzyme replacement therapy injections. [20] Enzyme replacement therapy for ADA requires weekly injections and costs about US$4.25 million for one patient over ten years.
Researchers at Oregon Health & Science University used an experimental CRISPR-based gene editing treatment in participants with a rare eye disorder that causes low vision and blindness. The ...
CRISPR Therapeutics. CRISPR Therapeutics AG is a Swiss–American biotechnology company headquartered in Zug, Switzerland. It was one of the first companies formed to utilize the CRISPR gene editing platform to develop medicines for the treatment of various rare and common diseases. [2][3] The company has approximately 500 employees and has ...
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