First CRISPR medicines
The first approved gene-editing medicine frees many sickle cell patients from crises; custom editing is now being tried.
Open in the interactive tree →The UK approved Casgevy (exagamglogene autotemcel) in November 2023 and the FDA followed on 8 December 2023, the first approved CRISPR therapy; it edits a patient's blood stem cells to switch on fetal hemoglobin. In the trial 29 of 31 evaluable sickle cell patients had no severe pain crises for at least 12 months. The treatment needs chemotherapy conditioning and has a US list price of about 2.2 million dollars.
As of October 2026
Uptake is slow but rising: 64 patients received Casgevy infusions in 2025 (30 of them in the fourth quarter), 147 started cell collection, and 2025 revenue was 116 million US dollars; second-quarter 2026 revenue was 76 million. By mid-2026 the FDA had extended approval to children aged 2 and older with sickle cell disease or transfusion-dependent beta thalassemia, 53 days after filing. In February 2025 the Penn and CHOP team gave a first personalized base-editing treatment to infant KJ Muldoon under an FDA single-patient authorization, and in March 2026 it was discussing a platform trial for urea cycle disorders with the FDA. Editors that work inside the body and avoid chemotherapy are in clinical trials.
Open steps
- Editing stem cells without chemotherapy Medium AI leverageCasgevy needs toxic chemotherapy to clear marrow; antibody-based conditioning or direct in-body stem-cell editing could make treatment safer and wider.
- Agents that plan editing experiments High AI leverageChoosing the editor, guide and delivery for each disease target is expert labour; AI tools could cut design time and improve specificity.
- Spotting large unintended changes Medium AI leverageCuts can cause large deletions or chromosome rearrangements; detection methods must find rare events in edited cells before patients receive them.
- Cheaper manufacturing of edited cells Low AI leverageEx vivo therapies need weeks in a specialised plant; automated closed-system manufacturing and in-body alternatives are needed to reach most patients.
Where AI could help
Medium AI leverage. AI improves editor and guide design, but price, chemotherapy before infusion and delivery limit uptake more than design does.
- Designing new editors with fewer off-target cuts
- Guide design and off-target prediction per patient
- Agents that plan experiments and analyze editing data
- Faster build of custom therapies for single patients
Shown so far
- In July 2025 Nature published OpenCRISPR-1, a gene editor designed with language models trained on CRISPR-Cas sequences, which edited the human genome (company-developed, Profluent). source
- In July 2025 Nature Biomedical Engineering published CRISPR-GPT, an LLM agent for gene-editing experiment design; two junior researchers used it to run gene knockouts and activations in cell lines. source
Prerequisites
Unlocks
- Curing HIVopen