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EDIT-301

Phase 1

Transfusion Dependent Beta Thalassemia | Gene therapy | Hematology |Editas Medicine, Inc.|Last Updated: Apr 2, 2025

Success Probability
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Trial Design
UNCONTROLLEDDMC
Total Trials1
Total Enrollment9
FDA Designations
ORPHAN_DRUGRARE_PEDIATRIC_DISEASERMAT
Clinical trial landscape

EDIT-301 · 2 trials · 5 indications

Phase 1 2
NCT05444894EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)Transfusion Dependent Beta Thalassemia
ACTIVE NOT_RECRUITING9 Analytics
NCT04853576A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)Sickle Cell Disease
ACTIVE NOT_RECRUITING45 Analytics
PHASE1ACTIVE NOT_RECRUITING
EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)
Transfusion Dependent Beta Thalassemia
NCT054448949
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PHASE1ACTIVE NOT_RECRUITING
A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)
Sickle Cell Disease
NCT0485357645
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Study Endpoints
Primary Endpoints
Proportion of participants achieving engraftment defined as neutrophil engraftment (defined as demonstrating absolute neutrophil count (ANC) ≥ 0.5 x 10^9/L post EDIT-301 infusion for 3 consecutive measurements obtained on different days)
EDIT-301 infusion (Day 0) to 42 days post EDIT-301 infusion
Frequency and severity of adverse events (AEs) (incidence of AEs and Grade 3 or higher serious adverse events, using National Cancer Institute Common Terminology Criteria for Adverse Events [NCI CTCAE] v.5.0)
Screening through up to 24 months post EDIT-301 infusion
Proportion of subjects achieving complete resolution of severe vaso-occlusive events (VOEs)
from Month 6 through Month 18 post EDIT-301 infusion
Secondary Endpoints
Kinetics of HSPC engraftment
EDIT-301 infusion (Day 0) to first day in which 3 consecutive measurements obtained on different days demonstrate ANC ≥ 0.5 x 10^9/L up to 24 months post EDIT-301 infusion
Incidence of transplant related mortality
EDIT-301 infusion (Day 0) through Day 100 post EDIT-301 infusion and from EDIT-301 infusion (Day 0) through 12 months post EDIT-301 infusion
Incidence of all-cause mortality
Screening through up to 24 months post EDIT-301 infusion
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
EDIT-301EXPERIMENTALEDIT-301 (autologous gene edited (CD)34+ hematopoietic stem cells) will be administered as a one-time intravenous infusion.
Interventions
NameTypeDescription
EDIT-301GENETICAdministered by intravenous infusion after myeloablative conditioning with busulfan.
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Eligibility Criteria
Age Range18 Years to 35 Years
SexALL
Healthy VolunteersNo
Study Sites8

Key Inclusion Criteria: Diagnosis of Transfusion Dependent B-Thalassemia as defined by: * Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE) based on historical data in medical records, and * History of at least 100 mL/kg/year or 1...

Countries:United StatesCanada
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT04853576primaryCompletionDate: changed
LOWMay 26, 2026NCT05444894primaryCompletionDate: changed
LOWMay 24, 2026NCT04853576studyFirstPostDate: changed
LOWMay 24, 2026NCT05444894studyFirstPostDate: changed