Study Summary
This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.
Want to learn more about this trial?
Request More InfoInterventions
nula-cel Drug ProductGENETIC
nula-cel is administered via IV infusion following a myeloablative conditioning regimen
Study Locations
| Facility | City | State | Country |
|---|---|---|---|
| Children's Hospital Los Angeles | Los Angeles | California | United States |
| Lucile Packard Children's Hospital | Palo Alto | California | United States |
| Washington University | St Louis | Missouri | United States |
| Nationwide Children's Hospital | Columbus | Ohio | United States |