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

Phase 1/2 Active not recruiting 9 enrolled Editas Medicine, Inc.

NaSingle-groupOpen-labelTreatment

Summary

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

Timeline

Start: 2022-04-29
Primary completion: 2025-09
Completion: 2025-12

Publications

Frangoul H, Hanna R, Walters MC, Kao RL, Carroll C, McManus M, Chang KH, Jaskolka MC, Kim K, Yu Q, Badamosi N, Mei B, Afonja O, Thompson A; EdiThal Investigators. CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat beta-Thalassemia. N Engl J Med. 2026 Apr 2;394(13):1292-1301. doi: 10.1056/NEJMoa2501277.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	RENIZGAMGLOGENE AUTOGEDTEMCEL	Unknown	—	Intravenous

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

Summary

Timeline

Publications

Drugs

Indications