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Gene Editing Therapy

All articles tagged with #gene editing therapy

healthcare2 years ago•0 min saved

"FDA Approves CRISPR Gene-Editing Therapy for Beta-Thalassemia and Sickle Cell Disease"

The FDA has approved Vertex Pharmaceuticals and CRISPR Therapeutics' Casgevy, a gene editing therapy, to treat beta-thalassemia, marking the first-ever CRISPR-edited therapy for the rare genetic blood disorder. Priced at $2.2 million for the one-time ex vivo treatment, the approval is for patients 12 years and older who require regular blood transfusions to survive, and it will compete with a gene-edited therapy from startup bluebird bio.

via Endpoints News|

#beta-thalassemia #crispr-therapeutics #fda

science-and-technology2 years ago•10 min saved

"2023's Unforgettable Science Breakthroughs, Stories, and Headlines"

The year 2023 witnessed significant advancements in various scientific fields. Some notable breakthroughs include the discovery of a new type of brain cell that could aid in understanding brain protection and memory consolidation, the rise of ChatGPT as a versatile language model, the detection of a long-sought-after organic molecule in space by the James Webb Space Telescope, the creation of synthetic human embryos for research purposes, the successful transplantation of a pig organ into a human body, and the development of a gene-editing therapy that could revolutionize high-cholesterol treatment. Other breakthroughs include the measurement of a muon's wobble challenging the Standard Model of physics, the first-ever whole-eye and partial-face transplant, and the progress made in achieving controlled nuclear fusion at the National Ignition Facility.

via Inverse|

#brain-cell #chatgpt #gene-editing-therapy

health2 years ago•9 min saved

"Vertex and CRISPR's Gene-Editing Therapy Nears FDA Approval, Paving the Way for a Patent Battle"

The gene-editing therapy Casgevy, developed by Vertex and CRISPR Therapeutics, is expected to receive FDA approval for the treatment of severe cases of sickle cell disease. Casgevy would be the first gene-editing therapy authorized by US regulators and uses the CRISPR tool. In clinical trials, Casgevy demonstrated remarkable results, completely relieving 29 out of 30 sickle cell patients of debilitating pain episodes for at least one year. However, the expected high price tag raises concerns about equity in medicine. While Casgevy offers hope for a potential cure, ethical concerns and access to the treatment remain important considerations.

via The Boston Globe|

#casgevy #crispr #fda-approval