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graphic of scissors cutting two strands of DNA

2023 was an important year for patients with sickle cell disease(link is external)Prior to CRISPR(link is external), the only cure for the life-long ailment was a bone marrow transplant(link is external), which is notoriously dangerous and costly. This month, the FDA approved(link is external) Vertex’s “Casgevy,” a CRISPR-based therapy(link is external) for the treatment of sickle cell disease in patients 12 and older. The landmark approval made the therapeutic the first genetically edited therapy to reach the general market.

Casgevy, which also received the greenlight from regulators in the UK for another blood disorder called beta thalassemia, works by being administered in a single-infusion of genetically modified stem cells to a patient. Clinical study participants that took Casgevy were free from symptoms associated with sickle cell disease, like periodic episodes of extreme pain(link is external) due to blocked blood flow through vessels, for up to a year.

CRISPR(link is external), which modifies precise regions of a human’s DNA strands, was once thought to be a far off scientific innovation. Human cells were first modified using CRISPR in clinical trials in China back in 2016(link is external). Less than a decade...