Dr. Stuart Orkin's research on identifying genes regulating fetal hemoglobin led to a revolutionary gene therapy for sickle cell disease using CRISPR technology. [ 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 ]
World's first gene therapy for sickle cell and thalassemia approved in the U.K.
The UK has authorized the first gene therapy treatment for sickle cell disease, offering potential relief to thousands of patients.
Casgevy, made by Vertex Pharmaceuticals and CRISPR Therapeutics, is the first medicine licensed using the gene editing tool.
Gene therapy offers a potential cure for sickle cell disease and thalassemia, which have previously only been manageable through difficult treatments. [ more ]
U.K. Becomes First Country to Approve a CRISPR Disease Treatment
UK medicines regulator approves CRISPR gene editing therapy for sickle-cell disease and -thalassaemia.
Casgevy completely relieved 28 out of 29 participants of pain from sickle-cell disease for at least one year after treatment.
For at least one year after treatment with Casgevy, 93% of participants with a severe form of -thalassaemia did not need a red-blood-cell transfusion. [ more ]
Landmark sickle cell treatment is axed by regulators
A drug approved to treat sickle cell disease has been scrapped by the UK government, despite being hailed as the first new drug in over 20 years for the condition.
The decision has sparked criticism from campaigners who argue that it leaves a reliance on only one licensed NHS treatment for sickle cell patients. [ 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 ]
Former Patriot Devin McCourty honored as 'Hero Among Us' at Celtics game
Devin McCourty, former Patriots safety, was honored with the Celtics' 'Hero Among Us' award for his work in the community.
McCourty and his twin brother co-founded 'Tackle Sickle Cell' over 10 years ago to raise awareness for the disease and have raised over $1.2 million for Boston Medical Center. [ more ]
FDA approves first CRISPR therapy-here's how it works against sickle cell
The FDA has approved two gene therapies for the treatment of sickle cell disease, one of which is the first CRISPR/Cas9-based treatment to be approved in the US.
Sickle cell disease affects around 100,000 people in the US and can lead to anemia, organ damage, and early death. [ more ]
Breakthrough Gene Treatments For Sickle Cell Disease Approved
Regulators have approved two new gene therapies for sickle cell disease, offering hope for a cure for the painful blood disorder
The treatments are designed for patients 12 and older with severe forms of the disease, and one of them is the first therapy based on CRISPR gene editing [ more ]
Gene-editing technology CRISPR has successfully treated a patient with sickle-cell disease, providing hope for future treatments.
The treatment resulted in the patient living virtually symptom-free, with 29 out of 30 eligible patients experiencing zero pain crises after treatment. [ more ]
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 ]