EDIT-301

GENETIC2 trials

Sponsors

Editas Medicine, Inc.

Conditions

HemoglobinopathiesSickle Cell DiseaseThalassemia IntermediaThalassemia MajorTransfusion Dependent Beta Thalassemia

Phase 1

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

Active, not recruitingNCT04853576

Editas Medicine, Inc.Hemoglobinopathies, Sickle Cell Disease

Start: 2021-05-04End: 2025-08-31Target: 45Updated: 2025-01-31

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

Active, not recruitingNCT05444894

Editas Medicine, Inc.Hemoglobinopathies, Thalassemia Intermedia, Thalassemia Major +1

Start: 2022-04-29End: 2025-12-31Target: 9Updated: 2025-04-02

Related Papers

CRISPR-Cas12a gene editing of the HBG1/2 promoters leads to sustained normalization of total hemoglobin and increased fetal hemoglobin in patients with severe sickle cell disease: Updated Results from the RUBY trial

Blood2025-11-03

Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine, Led to Sustained Hemoglobin Normalization and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease Treated in the RUBY Trial

Blood2024-11-053 citations

Reni-Cel, an Investigational AsCas12a Gene-Edited Cell Medicine, Led to Successful Engraftment, Increased Hemoglobin, and Reduced Transfusion Dependence in Patients with Transfusion-Dependent Beta-Thalassemia Treated in the Edithal Trial

Blood2024-11-05

Incremental eligibility criteria for the BMT CTN 1507 haploidentical trial for children with sickle cell disease

Blood Advances2024-10-071 citations

AsCas12a Gene Editing of <i>HBG1/2</i> Promoters with EDIT-301 Results in Rapid and Sustained Normalization of Hemoglobin and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia

Blood2023-11-0214 citations

S264: EDIT-301 SHOWS PROMISING PRELIMINARY SAFETY AND EFFICACY RESULTS IN THE PHASE I/II CLINICAL TRIAL (RUBY) OF PATIENTS WITH SEVERE SICKLE CELL DISEASE USING HIGHLY SPECIFIC AND EFFICIENT ASCAS12A ENZYME

HemaSphere2023-08-0116 citations