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Clinical Trial Report

Gene Therapy Trial Report

Summary

Gene Editing For Sickle Cell Disease


NCTID NCT06506461 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Sickle Cell Disease
Disease Ontology Term DOID:0081445
Compound Name Gene-modified CD34+ cells
Sponsor St. Jude Children's Research Hospital
Funder Type Other
Recruitment Status
Recruiting
Enrollment Count 25
Results Posted Not Available

Therapy Information


Target Gene/Variant BCL11A
Therapy Type Gene editing
Therapy Route Ex-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Intravenous
Drug Product Type Autologous cells
Target Tissue/Cell CD34+ cells
Delivery System Electroporation
Vector Type
Editor Type SpCas9 mRNA
Dose 1 Transduced CD34+ cells
Dose 2
Dose 3
Dose 4
Dose 5

Study Record Dates


Current Stage Phase1
Submit Date 2024-06-14
Completion Date 2032-12
Last Update 2025-03-25

Participation Criteria


Eligible Age 18 Years - 24 Years
Standard Ages Adult
Sexes Eligible for Study ALL

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates

Resources/Links