Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease

NCTID	NCT04819841 (View at clinicaltrials.gov)
Description	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. (Show More)
Indication	Sickle Cell Disease
Compound Name	KMAU-001 (nulabeglogene autogedtemcel)
Sponsor	Kamau Therapeutics
Funder Type	Industry
Status	Recruiting
Enrollment Count	15

No.of Trial Sites	3
Locations	United States

Has US IND	True
Recent Updates	Product was previously developed by Graphite Bio