Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients with Severe Sickle Cell Disease

NCTID	NCT04774536 (View at clinicaltrials.gov)
Description	This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years old to 35 years old severe Sickle Cell Disease (SCD). The study will evaluate the hematopoietic stem cell transplantation (HSCT) using CRISPR/Cas9 edited red blood cells (known as CRISPR_SCD001 Drug Product). (Show More)
Indication	Sickle Cell Disease
Compound Name	CRISPR_SCD001
Sponsor	Mark Walters, MD
Funder Type	Other
Status	Recruiting
Enrollment Count	9

Target Gene/Variant	HBB
Therapy Type	Gene editing
Therapy Route	Ex-vivo
Mechanism of Action	Mutation correction
Route of Administration	Intravenous
Drug Product Type	Autologous cells
Target Tissue/Cell	CD34+ cells
Delivery System	Electroporation
Vector Type	none
Editor Type	Cas9 mRNA
Dose 1	Transduced CD34+ cells (range: 3 - 20 x 10^6 cells/kg)
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites	2
Locations	United States

Has US IND	True
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