EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)

NCTID	NCT05444894 (View at clinicaltrials.gov)
Description	The purpose of this study is to evaluate the safety, tolerability, and efficacy of treatment with EDIT-301 in adult participants with Transfusion Dependent beta Thalassemia (Show More)
Development Status	Inactive
Indication	Beta-Thalassemia Major
Disease Ontology Term	DOID:0080771
Compound Name	EDIT-301
Compound Alias	Renizgamglogene autogedtemcel
Sponsor	Editas Medicine, Inc.
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	9
Results Posted	Not Available

Target Gene/Variant	HBG1/HBG2
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	Viral transduction
Vector Type	LV
Editor Type	ASCas12a mRNA
Dose 1	Min: 5.7E6 CD34+ cells/kg
Dose 2	Max: 11.9E6 CD34+ cells/kg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	8
Locations	Canada,United States

Has US IND	True
FDA Designations	Regenerative Medicine Advanced Therapy
Recent Updates	Ended development of this program due to inability to locate a financial partner