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

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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 Aug 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