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Clinical Trial Report

Gene Therapy Trial Report

Summary

Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe Β-Hemoglobinopathies


NCTID NCT06647979 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Sickle Cell Disease, Beta-Thalassemia
Disease Ontology Term DOID:0081445; DOID:0080771
Compound Name Autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein
Sponsor Daniel Bauer
Funder Type Other
Recruitment Status
Not yet recruiting
Enrollment Count 10
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 2024-10-11
Completion Date 2030-12
Last Update 2025-02-12

Participation Criteria


Eligible Age 13 Years - 40 Years
Standard Ages Child, 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 Includes genotyping for Variant rs114518452 as part of the inclusion/exclusion criteria

Resources/Links