U.K. Is First to Approve a CRISPR-Based Therapy, Covering Two Blood Disorders
The UK has authorized a gene-editing medicine for treating sickle cell disease and beta thalassemia, marking the first approval worldwide for a therapy based on CRISPR technology.
The therapy, known as Casgevy, involves editing a gene in a patient's stem cells to produce high levels of fetal hemoglobin.
Casgevy offers a potentially curative treatment option for these inherited blood disorders, reducing the need for chronic therapies and blood transfusions. [ more ]
UK authorizes first gene therapy for treating sickle cell disease
The UK's MHRA has approved the use of a gene-editing therapy for patients with sickle cell disease and beta thalassemia.
The therapy, called Casgevy, is the first-ever approved therapy that uses CRISPR-based gene editing technology.
The therapy has shown high efficacy in relieving severe pain associated with these blood disorders and could potentially replace the current standard of care. [ more ]
UK authorizes first gene therapy for treating sickle cell disease
The UK's MHRA has approved the use of a gene-editing therapy for patients with sickle cell disease and beta thalassemia.
The therapy, called Casgevy, is the first-ever approved therapy that uses CRISPR-based gene editing technology.
The therapy has shown high efficacy in relieving severe pain associated with these blood disorders and could potentially replace the current standard of care. [ more ]
Casgevy: UK approves gene-editing drug for blood disorders
The UK has approved a gene therapy using the Crispr gene-editing tool to treat sickle cell disease and beta thalassemia, a first in the world.
The treatment involves removing stem cells from a patient's bone marrow and using Crispr to disable the faulty gene, allowing the body to produce functioning haemoglobin.
Trials have shown promising results, with the majority of patients experiencing relief from symptoms and no longer needing blood transfusions. [ more ]
Casgevy: UK approves gene-editing drug for blood disorders
The UK has approved a gene therapy using the Crispr gene-editing tool to treat sickle cell disease and beta thalassemia, a first in the world.
The treatment involves removing stem cells from a patient's bone marrow and using Crispr to disable the faulty gene, allowing the body to produce functioning haemoglobin.
Trials have shown promising results, with the majority of patients experiencing relief from symptoms and no longer needing blood transfusions. [ more ]