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
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