Drugs / RENIZGAMGLOGENE AUTOGEDTEMCEL
RENIZGAMGLOGENE AUTOGEDTEMCEL
Regulatory milestones approvals, filings & regulatory actions · 2 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed | US (FDA) | — | sickle cell S-Lepore disease | 2020-12-09 | “submitted an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for the initiation of a Phase 1/2 clinical trial of EDIT-301” editasmedicine.com ↗ |
| Clinical hold | US (FDA) | — | sickle cell S-Lepore disease | — | “FDA removed the previously disclosed partial clinical hold for EDIT-301 RUBY study for the treatment of SCD.” editasmedicine.com ↗ |
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT05444894 | Apr 2022 → Sep 2025 overdue | beta-thalassemia intermedia, beta-thalassemia major | Editas Medicine, Inc. | Active not recruiting | No outcome recorded |
| Phase 1/2 | NCT04853576 | May 2021 → Aug 2025 overdue | sickle cell disease | Editas Medicine, Inc. | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 22
| Date | Issuer | Release |
|---|---|---|
| 2024-12-09 | Editas Medicine, Inc. | Results Editas Medicine Reports Updated Clinical Data from the RUBY Trial of Reni-cel in Patients with Severe Sickle Cell Disease at the American Society of Hematology (ASH) Annual Meeting editasmedicine.com ↗
Since reni-cel treatment, 27 of the 28 patients were free of vaso-occlusive events (VOEs). |
| 2024-06-14 | Editas Medicine, Inc. | Results Editas Medicine Reports New Safety and Efficacy Data from the RUBY Trial of Reni-cel in 18 Patients with Sickle Cell Disease, Presented at the European Hematology Association (EHA) Annual Congress editasmedicine.com ↗ |
| 2023-12-11 | Editas Medicine, Inc. | Results Editas Medicine Announces New EDIT-301 Safety and Efficacy Data in 17 Patients, Presented Today at the American Society of Hematology (ASH) Annual Meeting and in a Company-sponsored Webinar editasmedicine.com ↗
Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today announced new safety and efficacy data in 17 patients treated with EDIT-301, now known as renizgamglogene autogedtemcel (reni-cel), in the RUBY trial for severe sickle cell disease (SCD) (n=11) and in the EdiTHAL trial for transfusion-dependent beta thalassemia (TDT) (n=6). |
| 2023-10-16 | Editas Medicine, Inc. | Regulatory Editas Medicine Granted FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for EDIT-301 for the Treatment of Severe Sickle Cell Disease editasmedicine.com ↗
Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to EDIT-301, an investigational, gene editing medicine, for the treatment of severe sickle cell disease (SCD). |
| 2023-06-09 | Editas Medicine, Inc. | Results Editas Medicine Announces Positive Initial EDIT-301 Safety and Efficacy Data from the First Four Patients Treated in the RUBY Trial and the First Patient Treated in the EdiTHAL Trial editasmedicine.com ↗
Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today announced positive initial safety and efficacy data from the first four patients with sickle cell disease (SCD) treated with EDIT-301 in the RUBY trial and from the first transfusion-dependent beta thalassemia patient treated in the EdiTHAL trial. |
| 2023-05-05 | Editas Medicine, Inc. | Results Editas Medicine Announces First Quarter 2023 Results and Business Updates editasmedicine.com ↗
We entered 2023 with the objective of accelerating the development of EDIT-301 and positioning Editas as a leader in programable in vivo gene editing. |
| 2023-04-27 | Editas Medicine, Inc. | Regulatory Editas Medicine Receives FDA Orphan Drug Designation for EDIT-301 for the Treatment of Sickle Cell Disease editasmedicine.com ↗
Editas Medicine, Inc. (Nasdaq: EDIT), a clinical stage genome editing company, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to EDIT-301, an investigational, gene editing medicine, for the treatment of sickle cell disease. |
| 2022-12-06 | Editas Medicine, Inc. | Results Editas Medicine Announces Positive Safety and Efficacy Data from the First Two Patients Treated in the RUBY Trial of EDIT-301 for the Treatment of Severe Sickle Cell Disease editasmedicine.com ↗
The clinical data includes safety data from the first two patients and efficacy data from the first patient treated. |
| 2022-07-27 | Editas Medicine, Inc. | Results Editas Medicine Announces Clinical Achievements in the Development of EDIT-301 for Sickle Cell Disease editasmedicine.com ↗
Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced the dosing and confirmed successful neutrophil and platelet engraftment of the first patient in the Phase 1/2 RUBY trial of EDIT-301 for the treatment of severe sickle cell disease (SCD). |
| 2022-05-12 | Editas Medicine, Inc. | Regulatory Editas Medicine Receives FDA Orphan Drug Designation for EDIT-301 for the Treatment of Beta Thalassemia editasmedicine.com ↗
Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to EDIT-301, an investigational, gene editing medicine, for the treatment of beta thalassemia. |
| 2022-04-26 | Editas Medicine, Inc. | Regulatory Editas Medicine Receives FDA Rare Pediatric Disease Designation for EDIT-301 for the Treatment of Beta Thalassemia editasmedicine.com ↗
Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced that the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease designation to EDIT-301, an investigational, gene-edited medicine for the treatment of beta thalassemia. |
| 2021-12-20 | Editas Medicine, Inc. | Regulatory Editas Medicine Announces FDA Clearance of Investigational New Drug (IND) Application for EDIT-301 for the Treatment of Transfusion-Dependent Beta Thalassemia editasmedicine.com ↗
Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced that the U.S. Food and Drug Administration (FDA) has cleared the IND for EDIT-301 for the treatment of transfusion-dependent beta thalassemia (TDT), enabling the Company to initiate a Phase 1/2 clinical study of EDIT-301 in TDT patients. |
| 2021-12-12 | Editas Medicine, Inc. | Results Editas Medicine Reports Preclinical Data Demonstrating Robust Tumor Reduction and Clearance Using Novel, Engineered iNK Cells at the American Society of Hematology Annual Meeting editasmedicine.com ↗
AsCas12a edited TDT erythroid cells had improved maturation, health, and higher total hemoglobin content per cell when compared to unedited controls. |
| 2021-11-04 | Editas Medicine, Inc. | Results Editas Medicine to Present Data Demonstrating Progress Towards Transformative Gene Editing Medicines for the Treatment of Hemoglobinopathies and Cancer at the ASH Annual Meeting and Exposition editasmedicine.com ↗
Edited peripheral blood CD34+ cells mobilized from transfusion-dependent beta thalassemia (TDT) patients demonstrated significantly improved red blood cell production and increased hemoglobin content, supporting the development of EDIT-301 for the treatment of TDT; |
| 2021-06-11 | Editas Medicine, Inc. | Results Editas Medicine Presents Preclinical Data Supporting the Initiation of the EDIT-301 Phase 1/2 RUBY Clinical Trial for the Treatment of Sickle Cell Disease at the European Hematology Association Congress editasmedicine.com ↗
The data demonstrated that high levels of editing were achieved, resulting in robust fetal hemoglobin (HbF) induction in erythroid cells with no detectable off-target editing. |
| 2021-01-11 | Editas Medicine, Inc. | Regulatory Editas Medicine Announces the FDA has Cleared Initiation of the EDIT-301 Clinical Trial editasmedicine.com ↗
Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced the U.S. Food and Drug Administration (FDA) has cleared the initiation of the safety phase of the Company’s EDIT-301 clinical trial, and the Company can begin dosing patients. |
| 2020-12-09 | Editas Medicine, Inc. | Regulatory Editas Medicine Announces Submission of IND Application for EDIT-301 with the FDA editasmedicine.com ↗ |
| 2020-12-05 | Editas Medicine, Inc. | Results Editas Medicine Announces Preclinical Data and Large-Scale Manufacturing Process for EDIT-301, in Development for the Treatment of Sickle Cell Disease and Beta-Thalassemia editasmedicine.com ↗
Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced preclinical data and successful development of a large-scale manufacturing process for EDIT-301, a potentially best-in-class, one-time, durable, autologous cell therapy medicine to treat sickle cell disease and beta-thalassemia. |
| 2020-08-24 | Editas Medicine, Inc. | Regulatory Editas Medicine Receives Rare Pediatric Disease Designation for EDIT-301 for the Treatment of Sickle Cell Disease editasmedicine.com ↗
Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for EDIT-301, an experimental, autologous cell medicine, being developed as a potentially best-in-class, durable medicine for sickle cell disease. |
| 2020-06-12 | Editas Medicine, Inc. | Results Editas Medicine Presents Pre-Clinical Data from a Study of EDIT-301 with Sickle Patient Cells for the Potential Treatment of Sickle Cell Disease editasmedicine.com ↗
In particular, editing was highly efficient and reproducible, with approximately 90 percent editing in multiple sickle patient donors. |
| 2019-12-09 | Editas Medicine, Inc. | Results Editas Medicine Announces In Vivo Proof-of-Concept Data for EDIT-301, in Development for the Treatment of Sickle Cell Disease and Beta-Thalassemia editasmedicine.com ↗
Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced in vivo proof-of-concept data supporting the development of EDIT-301 as a potentially best-in-class, durable medicine to treat sickle cell disease and beta-thalassemia. |
| 2019-06-15 | Editas Medicine, Inc. | Results Editas Medicine Presents Pre-Clinical Data for Treatment of Sickle Cell Disease and Beta-Thalassemia at the 24th Congress of the European Hematology Association editasmedicine.com ↗
Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced results from a follow-up study to assess two different CRISPR genome editing strategies, one targeting the BCL11A erythroid enhancer ( BC11Ae ) and one targeting the beta-globin locus, for the treatment of sickle cell disease and beta-thalassemia. |
All press releases naming this drug 28 releases
Evidence & citations 7 cited values
Every value below carries the sentence it was read from. 4 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | RENIZGAMGLOGENE AUTOGEDTEMCEL | ChEMBL registry synonym — accepted as the source's own label CHEMBL6068079 ↗ |
| Known as | EDIT-301 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04853576 ↗ |
| Known as | Renizgamglogen autogedtemcel | ChEMBL registry synonym — accepted as the source's own label CHEMBL6068079 ↗ |
| Action | Restore | “to reactivate fetal hemoglobin production” PMID 41931047 ↗ Apr 2026 |
| Route | Intravenous | “administered as a one-time intravenous infusion” NCT04853576 ↗ |
| Target | HBG1 | “The therapy was designed to disrupt the BCL11A binding sites in the HBG1 and HBG2 promoters to reactivate fetal hemoglobin production for the treatment of sickle cell disease.” PMID 41931047 ↗ Apr 2026 |
| Target | HBG2 | “The therapy was designed to disrupt the BCL11A binding sites in the HBG1 and HBG2 promoters to reactivate fetal hemoglobin production for the treatment of sickle cell disease.” PMID 41931047 ↗ Apr 2026 |