CLIMB-121 (exa-cel, sickle cell)
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
CRISPR-Cas9 BCL11A editing (exa-cel) eliminates vaso-occlusive crises in severe sickle cell disease over multi-year follow-up.
Source publication
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
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Sponsor
Vertex Pharmaceuticals Incorporated
Principal investigator
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Population
Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies; n=63
Primary endpoint
Proportion of subjects who have not experienced any severe vaso-occlusive crisis (VOC) for at least 12 consecutive months (VF12)
Methodology notes
Full methodology is under curation. This project aggregates 3 seeded claims across three temporal layers. The replication spec, pre-registered analysis, and version history will appear here as the project matures.
Funding progress
$174K / $1.2M
15%
Funding displayed here is indicative of collaborator interest. Sciync does not broker money, grants, or securities.
Temporal layers
Published findings under validation
1
Active replication in progress
1
Forecast horizons pending resolution
1