Rapid Fire
FDA Approval to Gene Therapies for Sickle Cell Disease
- 21 Dec 2023
- 1 min read
Food and Drug Administration (FDA), an agency under U.S. Department of Health and Human Services, approved two gene therapies for sickle cell disease: Lyfgenia from bluebird bio and Casgevy by Vertex Pharmaceuticals and CRISPR Therapeutics.
- Sickle cell disease is a genetic blood disorder characterized by an abnormality in hemoglobin, the protein responsible for carrying oxygen in red blood cells (RBC).
- It causes RBC to adopt a sickle or crescent shape, hindering their movement through vessels, leading to potential complications like severe pain, infections, anaemia, and strokes.
- These therapies aim to transform treatment by leveraging CRISPR gene editing technology to either insert modified genes or edit stem cells, potentially offering one-time treatments.
- Concerns exist about the therapies' long-term effectiveness and risks, including the need for high-dose chemotherapy, potential infertility, and concerns about unintended genomic alterations.
Read more: CRISPR-Cas9 for Sickle-Cell Anaemia
