A Phase 1/2/3 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease

Primary endpoint

Proportion of subjects who have not experienced any severe vaso-occlusive crisis (VOC) for at least 12 consecutive months (VF12)

Population

Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies; n=63

Historical

Current

Future

3 noemes

Historical

1 noeme

Published findings from this trial. Vote whether the expert body still believes each noeme stands given current evidence.

The CLIMB-121 (exa-cel, sickle cell) trial's stated primary conclusion — CRISPR-Cas9 BCL11A editing (exa-cel) eliminates vaso-occlusive crises in severe sickle cell disease over multi-year follow-up. — replicates in independent cohorts.

gene-therapy

Current

1 noeme

Noemes tracking active replication, subgroup analysis, or long-term follow-up. Vote on the likelihood the original effect holds.

Recent follow-up analyses of CLIMB-121 (exa-cel, sickle cell) are confirming the original effect size in real-world data.

gene-therapy

Future

1 noeme

Open forecast horizons tied to this trial. Vote your probability that each resolves TRUE by the declared horizon.

By 2028, exa-cel or a comparable ex vivo gene-edited therapy will have treated >500 SCD patients globally with a durable VOC-free rate >90% at 3 years.

gene-therapy

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