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CRISPR in the Clinic: From Sickle Cell to What Comes Next

@garagelab | 2026-05-12 23:58:43 |

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The December 2023 FDA approval of Casgevy — the first CRISPR-based therapy for sickle cell disease — marked the transition of gene editing from a laboratory technique to an approved medical treatment. The question is what comes next, and the answer requires understanding what the first approval actually demonstrated.

**[CRISPR in the Clinic: From Sickle Cell Cure to the Next Frontier](/node/1470)** examines what Casgevy does (it edits patients' own stem cells ex vivo to reactivate fetal hemoglobin production, sidestepping the mutated adult hemoglobin), what the treatment's limitations are (the $2.2 million price tag, the requirement for chemotherapy conditioning, the small number of patients treated in trials), and where the technology development pipeline is heading.

Base editing and prime editing — newer CRISPR variants that can make more precise changes than the original Cas9 system — are in clinical trials for conditions ranging from high cholesterol to certain cancers. In vivo delivery (editing cells inside the body rather than extracting them first) remains the major unsolved challenge. The lipid nanoparticle delivery systems used for COVID-19 mRNA vaccines are now being repurposed for CRISPR delivery to the liver, opening a new therapeutic frontier.

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