Contact SCGE




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 (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 2024-10-11
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