A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)

NCTID	NCT04853576 (View at clinicaltrials.gov)
Description	The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult and adolescent participants with severe sickle cell disease (SCD). (Show More)
Indication	Sickle Cell Disease, Hemoglobinopathies
Compound Name	Renizgamglogene autogedtemcel (EDIT-301)
Sponsor	Editas Medicine, Inc.
Funder Type	Industry
Status	Active not recruiting
Enrollment Count	45

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
Vector Type
Editor Type	ASCas12a
Dose 1	Min: 2.9E6 CD34+ cells/kg
Dose 2	Max: 10.0E6 CD34+ cells/kg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	24
Locations	Canada,United States

Has US IND	True
Recent Updates	Paused development and initiated efforts to outlicense reni-cel; possible BLA submission by Q1 2025