Study title:
A Phase I/II study of the safety and efficacy of a single dose of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells in subjects with severe sickle disease
Long title:
A Phase I/II study of the safety and efficacy of a single dose of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells in subjects with severe sickle disease
Date receipt dossier:
20 aoû 2018
EudraCT number:
2018-001320-19
Company / Sponsor:
CRISPR Therapeutic AG
Phase:
I/II
Treated organism:
Humans
Indication category:
Hematologic disorder
Disease:
Severe Sickle Cell Disease (SCD)
Therapeutic approach:
Gene therapy
Genetic modification:
Modified cells expressing Hemoglobin F (HbF)
Method of transfer of nucleic acid of interest:
CRISPR-Cas9
Administered biological material:
Modified cells expressing HbF
Route of administration:
Intravenous
Locations in Belgium:
Hopital Universitaire des Enfants Reine Fabiola
Nr of subjects:
5
Foreseen duration:
3 years and 10 months
Type of procedure:
Contained use only
Current status:
Assessed