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The UK drug regulator approved Casgevy, the gene therapy to treat people above 12 with sickle cell disease and beta thalassemia.
The U.S. FDA has approved two gene therapies — Casgevy and Lyfgenia — to treat sickle cell disease in patients over 12.
What is Casgevy and Lyfgenia
Casgevy and Lyfgenia are groundbreaking gene therapies approved in December 2023 by the U.S. Food and Drug Administration (FDA) for the treatment of sickle cell disease (SCD) in patients aged 12 and older.
They offer a potentially curative option for individuals suffering from this debilitating genetic disorder.
Casgevy
Developed by: Vertex Pharmaceuticals and CRISPR Therapeutics
Technology: CRISPR/Cas9 gene editing
Mechanism: Edits the patient's own hematopoietic stem cells (HSCs) to produce functional adult hemoglobin (HbA), which is deficient in SCD patients.
Clinical trial results: In a Phase 3 study, 87% of patients receiving Casgevy experienced a 75%
or greater reduction in vaso-occlusive crises (VOCs), the painful and debilitating events characteristic of SCD.
Cost: $2.2 million per patient
Lyfgenia:
Developed by: bluebird bio
Technology: Lentiviral vector technology
Mechanism: Uses a lentiviral vector to deliver a gene for HbA T87Q, a modified form of hemoglobin that functions similarly to normal HbA, into the patient's HSCs.
Clinical trial results: In a Phase 3 study, 88% of patients receiving Lyfgenia achieved a 75% or greater reduction in VOCs.
Cost: $3.1 million per patient
Both therapies offer significant potential benefits for SCD patients,
Reduced frequency and severity of VOCs
Improved quality of life
Reduced need for pain medication and blood transfusions
Potential for long-term remission or even cure
It's important to note that these therapies are still in their early stages and come with some potential risks and limitations,
High cost
Need for long-term follow-up to assess safety and efficacy.
Potential for off-target effects of gene editing.
Limited availability due to complex manufacturing process
Casgevy and Lyfgenia represent a major breakthrough in the treatment of SCD and offer hope for a brighter future for patients living with this disease.
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