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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
Enrollment Count 25 (ESTIMATED)
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 2025-11-14

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