EDIT

Editas Medicine, Inc.

BS score 47.2MEDIUMPHASE2 · mkt cap $282.9M · rev ttm $40.5M

drug hypothesis

EDIT-301 modulates BCL11A (gene editing for fetal hemoglobin reactivation) to treat Transfusion-dependent beta thalassemia (TDT).

moa:CRISPR-Cas12a gene editing of autologous hematopoietic stem cells to disrupt BCL11A and reactivate fetal hemoglobin (HbF) expression, potentially eliminating transfusion dependence in beta thalassemia patients

score breakdown

trial design57

base rate disconnect11

language red flags100

composite47.2

valuation analysis

market cap$282.9M

revenue ttm$40.5M

phasePHASE2

historical base rate15%

disconnect ratio0.4x

lead trialNCT03396042

meta

cik0001650664

first discovered2026-04-09

last updated2026-04-10

statusACTIVE

llm model—

extracted at2026-04-10

trial design

Phase 1, non-randomized, single-arm, open-label trial enrolling 9 patients with transfusion-dependent beta thalassemia. Participants receive autologous EDIT-301 infusion following myeloablative conditioning. Primary focus on engraftment, with secondary endpoints measuring transfusion independence, HbF levels, and genetic modification persistence over 12+ months.

primary endpoint:Proportion of participants achieving neutrophil engraftment (ANC ≥ 0.5 x 10^9/L for 3 consecutive measurements on different days) within 42 days post EDIT-301 infusion

claimed differentiation

CRISPR-Cas12a approach (precision gene editing) versus lentiviral gene addition in competitor therapies (Zynteglo); aims to restore natural fetal hemoglobin switching mechanism

language red flags

Small enrollment (n=9) limits statistical power for efficacy conclusions
Phase 1 trial primarily assesses safety/engraftment, not definitive efficacy
Primary endpoint is engraftment, not transfusion independence - lower bar for 'success'
Very early stage - years from potential approval
No stated preclinical evidence provided in filing
Company claims CRISPR 'precision' without head-to-head data versus existing approved therapy (Zynteglo)
Active not recruiting status with primary completion 2025 suggests slower than expected enrollment or follow-up

company-stated risks

Risks associated with autologous HSCT procedure
Potential for graft failure or delayed engraftment
On-target but off-tumor toxicity
Insertional mutagenesis (theoretical risk)
Immune response to edited cells
Off-target CRISPR editing effects
Transplant-related mortality
Iron overload complications

upcoming catalysts

2025-09Primary completion of NCT05444894