Clinical Trial

Disease: Sickle Cell Disease, SCD, (NCT04853576)

Disease info:

Sickle cell disease is a group of disorders that affects haemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body. People with this disorder have atypical haemoglobin molecules called haemoglobin S, which can distort red blood cells into a sickle or crescent shape.

The production of haemoglobin A, which is the principle type of haemoglobin in humans, is governed by 3 genes: HBA1, HBA2, and HBB. Each haemoglobin A molecule consists of two alpha and two beta chains, and mutations in either of the HBA or the HBB genes may result in abnormal haemoglobin molecules with reduced or diminshed function. Sickle cell diseaase arises from a single point mutation in the 6th codon of the beta-globin gene (HBB), which results in a valine instead of a glutamic acid in the haemoglobin beta-chain.

Abnormal haemoglobin ultimately leads to anaemia as well as other symptoms, depending on the exact mutations present. Diseases caused by defective haemoglobin fall into a larger category of diseases known as the "haemoglobinopathies" which also include the thalassemias, a related group of diseases that are characterised by reduced or deficient rather than abnormal haemoglobin. 



Sickle cell disease affects approximately 100,000 individuals in the USA and more than 3 million worldwide.
Official title:
A Phase 1/2 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited CD34+ Human Hematopoietic Stem and Progenitor Cells (EDIT-301) in Subjects With Severe Sickle Cell Disease

Contact: Editas Medicine's Clinical Trial Team

Phone: 617-401-9007






United States, California

UCSF Benioff Children's Hospital, Oakland, California, United States, 94609


United States, Colorado

Children's Hospital Colorado, Aurora, Colorado, United States, 80045


United States, Florida

Johns Hopkins All Children's Hospital, Saint Petersburg, Florida, United States, 33701


United States, Georgia

Children's Healthcare of Atlanta, Atlanta, Georgia, United States, 30322


United States, Illinois

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States, 60611


United States, Mississippi

University of Mississippi Medical Center, Jackson, Mississippi, United States, 39216


United States, New York

Columbia University Medical Center - Department of Pediatrics, New York, New York, United States, 10032

Columbia University Medical Center, New York, New York, United States, 10032


United States, North Carolina

The University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, United States, 27599

Atrium Health, Charlotte, North Carolina, United States, 28204


United States, Ohio

University Hospitals Rainbow Babies & Children's Hospital, Cleveland, Ohio, United States, 44106

Cleveland Clinic, Cleveland, Ohio, United States, 44195

Nationwide Children's Hospital, Columbus, Ohio, United States, 43205


United States, Pennsylvania

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States, 19104


United States, South Carolina

Medical University of South Carolina, Charleston, South Carolina, United States, 29425


United States, Tennessee

Tristar Medical Group Children's Specialists/Sarah Cannon Center for Blood Cancers, Nashville, Tennessee, United States, 37203


United States, Texas

Texas Oncology - Baylor Charles A. Sammons Cancer Center, Dallas, Texas, United States, 75246

Cook Children's, Fort Worth, Texas, United States, 76104


Canada, Ontario

Ottawa Hospital Research Institute, Ottawa, Ontario, Canada, K1H 8L6

Princess Margaret Cancer Centre, Toronto, Ontario, Canada, M5G 2M9


Canada, Quebec

Centre Hospitalier Universitaire Sainte-Justine, Montréal, Quebec, Canada, H3T 1C5



Study start:
May. 4, 2021
40 participants
Gene editing method:
Type of edit:
Gene disruption
Haemoglobin Subunit Gamma 1 and 2 (HBG1/2 promoter)
Delivery method:
Electroporation - Ex-vivo
EDIT-301 is currently being investigated in clinical studies in patients with severe sickle cell disease (RUBY trial, NCT04853576) and transfusion-dependent beta thalassemia (EDITHAL trial, NCT05444894).
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting


The RUBY Trial is a single-arm, open-label, multi-center Phase 1/2 study designed to assess the safety and efficacy of EDIT-301 in people with severe sickle cell disease. Enrolled patients will receive a single administration of EDIT-301.

EDIT-301 is an experimental, autologous cell therapy medicine under investigation for the treatment of sickle cell disease. EDIT-301 is comprised of sickle patient CD34+ cells that are genetically modified using a highly specific and efficient CRISPR/Cas12a (also known as Cpf1) ribonucleoprotein (RNP) to edit the HBG1/2 promoter region in the beta-globin locus. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in foetal haemoglobin (HbF) production, which has the potential to provide a durable treatment benefit for people living with sickle cell disease.

Last updated: Dec. 28, 2023
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