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

Phase 1/2 Active not recruiting 45 enrolled Editas Medicine, Inc.

NaSingle-groupOpen-labelTreatment

Summary

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).

Timeline

Start: 2021-05-04
Primary completion: 2025-08
Completion: 2025-08

Publications

Hanna R, Frangoul H, Pineiro L, McKinney C, Mapara M, Dalal J, Rangarajan HG, Atkins H, Sharma A, Chang KH, Jaskolka MC, Kim K, Yu Q, Mei B, Afonja O, Walters MC; RUBY Investigators. CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat Sickle Cell Disease. N Engl J Med. 2026 Apr 2;394(13):1281-1291. doi: 10.1056/NEJMoa2415550.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	RENIZGAMGLOGENE AUTOGEDTEMCEL	Unknown	—	Intravenous

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

Summary

Timeline

Publications

Drugs

Indications