This section highlights FDA-related milestones and regulatory updates for drugs developed by Editas Medicine (EDIT).
Over the past two years, Editas Medicine has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
EDIT-301 and EDIT-301. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
EDIT-301 - FDA Regulatory Timeline and Events
EDIT-301 is a drug developed by Editas Medicine for the following indication: Transfusion-Dependent Beta Thalassemia.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- EDIT-301
- Announced Date:
- June 14, 2024
- Indication:
- Transfusion-Dependent Beta Thalassemia
Announcement
Editas Medicine, Inc. announced new safety and efficacy data in 7 patients with transfusion-dependent beta thalassemia (TDT) treated with renizgamglogene autogedtemcel (reni-cel; formerly known as EDIT-301) in the Phase 1/2 EdiTHAL clinical trial. Reni-cel, the first investigational AsCas12a gene-edited cell therapy medicine, is being studied in the EdiTHAL trial as a potential one-time, durable gene editing medicine for people living with TDT.
AI Summary
Editas Medicine, Inc. recently shared promising safety and efficacy data from the Phase 1/2 EdiTHAL clinical trial, which focused on patients with transfusion-dependent beta thalassemia (TDT). In this study, 7 patients were treated with renizgamglogene autogedtemcel (reni-cel), the first investigational AsCas12a gene-edited cell therapy. Unlike traditional treatments, reni-cel is designed as a one-time, durable gene editing medicine that may offer lasting benefits for people living with TDT.
The early results from the trial are encouraging, showing that the treatment was well tolerated and demonstrated a favorable safety profile. These findings support further investigation into reni-cel’s potential to improve hemoglobin production and overall outcomes for TDT patients, marking a significant step forward for gene editing therapies in the treatment of serious blood disorders.
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EDIT-301 (RUBY) - FDA Regulatory Timeline and Events
EDIT-301 (RUBY) is a drug developed by Editas Medicine for the following indication: Sickle Cell Disease.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- EDIT-301 (RUBY)
- Announced Date:
- December 9, 2024
- Indication:
- Sickle Cell Disease
Announcement
Editas Medicine, Inc.will present updated safety and efficacy data in 28 patients living with severe sickle cell disease (SCD) treated with renizgamglogene autogedtemcel (reni-cel; formerly known as EDIT-301) in the Phase 1/2/3 RUBY clinical trial.
AI Summary
Editas Medicine will present updated safety and efficacy results from the Phase 1/2/3 RUBY clinical trial at the American Society of Hematology Annual Meeting. The poster presentation focuses on data from 28 patients with severe sickle cell disease treated with renizgamglogene autogedtemcel (reni‐cel), formerly known as EDIT-301.
The data show that reni-cel was well-tolerated, with 27 of the 28 patients reporting no vaso-occlusive events following treatment. Patients experienced rapid, robust increases in total hemoglobin—from an average of 9.8 g/dL at baseline to 13.8 g/dL at month 6—and significant, sustained improvements in fetal hemoglobin levels and red blood cell quality. Additionally, patients reported better outcomes in pain management, physical function, and social activities, supporting the potential of reni-cel as a one-time, durable treatment option for severe sickle cell disease.
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