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FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease

Author: U.S. Food & Drug Administration

Published: December 08, 2023

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FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease

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The FDA approved the first cell-based gene therapies, Casgevy and Lyfgenia, for the treatment of sickle cell disease in patients 12 years and older.

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The FDA has approved two new gene therapies, Casgevy and Lyfgenia, for treating sickle cell disease (SCD) in patients aged 12 and older. Casgevy, uses technology to modify blood stem cells, increasing fetal hemoglobin to prevent sickling. Lyfgenia, uses a lentiviral vector to produce a hemoglobin that reduces sickling. Both treatments involve collecting patients' stem cells, using chemotherapy to prepare the bone marrow, and re-infusing the modified cells. These approvals are major advances for treating SCD, a disease with limited options (U.S. Food & Drug Administration, 2023).

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1. Potential Cure: Gene therapy aims to correct the genetic defect as opposed to traditional treatment of symptomatic management.

2. Improve Quality of Life: Gene therapy can decrease or eliminate symptoms associated with crises, thus greatly improving patients' quality of life.

3. Single Treatment: Gene therapy is given back as a one-time single-dose infusion, as opposed to ongoing treatments and routine medication.

4. Decreased Transfusion-Associated Risk: When gene therapy is successful the need for regular blood transfusions is decreased or eliminated, therefore lowering transfusion-associated risk.

5. Personalized Treatment: The patient's genetic makeup is targeted in gene therapy, making each treatment personalized to the individual.

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1. High Cost: Gene therapy treatments are expensive and not all patients in need may be able to afford treatment.

2. Chemotherapy-associated Risk: Chemotherapy required for stem cell infusions can often have severe side effects.

3. Long-term Monitoring: Gene therapy is relatively new making the long-term effectiveness and potential late-onset side effects not completely understood, thus requiring lifelong monitoring.

4. Potential Adverse Reaction: There is a risk of the patients' immune system rejecting the modified genes and this could cause adverse reactions.

5. Ethical Concern: When genetic alterations are made, ethical issues and concerns may arise.

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Sickle cell disease significantly impacts a large population, causing considerable negative effects. Gene therapy has the potential to revolutionize the treatment and possibly cure hemoglobinopathies like sickle cell disease. The recent innovations, Casgevy and Lyfgenia, could profoundly improve the lives of those affected by the condition. However, the groundbreaking potential of these therapies must be carefully considered and thoroughly evaluated as highlighted in the cons.

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Cleveland Clinic. (n.d.). Gene Therapy for Hemoglobinopathies. [Video]. https://www.youtube.com/watch?v=eSxErZICEXg&t=116s

U.S. Food and Drug Administration. (2023, December 8). FDA approves first gene therapies to treat patients with sickle cell disease. U.S. Food and Drug Administration. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease