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-401, EDIT-301, and EDIT-301. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
EDIT-401 FDA Regulatory Timeline and Events
EDIT-401 is a drug developed by Editas Medicine for the following indication: Gene and Cell Therapy.
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-401
- Announced Date:
- May 14, 2026
- Indication:
- Gene and Cell Therapy
Announcement
Editas Medicine, Inc. shared new preclinical data supporting the continued advancement of Editas' lead in vivo development candidate, EDIT-401, and its potential as a one-time treatment for hyperlipidemia, as well as the broader potential of the Company's differentiated upregulation strategy.
AI Summary
Editas Medicine said new preclinical data support moving forward with EDIT-401, its lead in vivo development candidate. The company believes EDIT-401 could one day become a one-time treatment for hyperlipidemia, a condition linked to high levels of fat in the blood and a higher risk of heart disease. The new findings add to earlier results and strengthen the case for Editas’ gene-editing approach that aims to turn genes up, rather than simply turn them off.
The company also said the data support its broader upregulation platform, which is designed to help the body fight disease by boosting alternate or compensatory biological pathways. Editas added that DNA large language prediction models may help speed up and improve the development of these gene-editing strategies. Overall, the results suggest the company’s approach could have wider use beyond EDIT-401.
Read Announcement- Drug:
- EDIT-401
- Announced Date:
- April 27, 2026
- Indication:
- Gene and Cell Therapy
Announcement
Editas Medicine, Inc. announced that five abstracts have been accepted for presentation, including one oral presentation and two poster presentations at the American Society of Gene and Cell Therapy (ASGCT) 2026 Annual Meeting, one oral presentation at TIDES USA 2026: Oligonucleotide and Peptide Therapeutics Conference, and one oral presentation at the 94th European Atherosclerosis Society (EAS) Congress.
AI Summary
Editas Medicine announced that five abstracts were accepted for presentation at three upcoming scientific meetings. At the American Society of Gene and Cell Therapy (ASGCT) 2026 Annual Meeting, the company will give one oral presentation titled “Preclinical Development of EDIT‑401, a Durable In Vivo CRISPR Gene Editing Therapy That Upregulates LDLR Protein to Lower LDL‑C,” plus two poster presentations, including one on the pharmacokinetics and pharmacodynamics of an in vivo gene editing therapy to lower LDL‑C in mice.
In addition, Editas will present an oral talk at TIDES USA 2026 titled “Transformative LDL Cholesterol Lowering In Vivo CRISPR Gene Editing Approach for Hyperlipidemia and Atherosclerotic Cardiovascular Disease,” and one oral presentation at the 94th European Atherosclerosis Society (EAS) Congress. Abstracts will be available on the conference websites and presentations will be posted on the Editas website during the meetings.
Read Announcement- Drug:
- EDIT-401
- Announced Date:
- October 9, 2025
- Indication:
- Gene and Cell Therapy
Announcement
Editas Medicine today reported in vivo preclinical proof-of-concept data for EDIT-401, an experimental, potential best-in-class, one-time therapy to significantly reduce LDL-cholesterol (LDL-C), at the 32nd Annual European Society of Gene and Cell Therapy (ESGCT) Congress in Seville, Spain.
AI Summary
Editas Medicine reported in vivo preclinical proof-of-concept data for EDIT-401, a one-time gene editing therapy designed to cut LDL cholesterol (LDL-C). The results were shared at the 32nd Annual European Society of Gene and Cell Therapy Congress in Seville, Spain.
In non-human primates, a single dose of EDIT-401 achieved at least 90% LDL-C reduction within 48 hours. Similar results appeared in mice with high LDL-C and low LDL receptor (LDLR) function. The therapy uses CRISPR/Cas9 nuclease with dual guide RNAs delivered by lipid nanoparticles to disrupt negative control sites in the 3′ UTR of the LDLR gene. This boosted LDLR mRNA stability and led to a sixfold rise in liver LDLR protein. The effect lasted three months in mouse models.
These findings highlight EDIT-401’s potential as a durable, leading treatment for people living with high LDL cholesterol.
Read Announcement- Drug:
- EDIT-401
- Announced Date:
- October 6, 2025
- Indication:
- Gene and Cell Therapy
Announcement
Editas Medicine, Inc. announced an oral presentation at the upcoming European Society of Gene and Cell Therapy (ESGCT) Congress, to be held October 7-10, 2025, in Seville, Spain.
AI Summary
Editas Medicine announced an oral presentation at the European Society of Gene and Cell Therapy (ESGCT) Congress, October 7–10, 2025, in Seville, Spain. The company will share new data on a CRISPR gene editing agent designed to lower LDL cholesterol.
Abstract OR069, titled “A transformative LDL-cholesterol–lowering CRISPR gene editing medicine,” is set for October 9 at 5:00 p.m. CEST (11:00 a.m. ET) in room A. Linda Burkly, Ph.D., Executive VP and CSO, will deliver the talk. Registered attendees can view abstracts on the ESGCT site. Editas will post the presentation on its website afterward for ongoing access.
Editas management will also speak at investor events in October, including the H.C. Wainwright Genetic Medicines Virtual Conference on October 14 and the Chardan Genetic Medicines Conference in New York on October 21. Live webcasts and 30-day replays will be available in the Investors section of the company’s website.
Read Announcement
EDIT-301 (RUBY) FDA Regulatory 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.
Read Announcement
EDIT-301 FDA Regulatory 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.
Read Announcement
