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- renizgamglogene autogedtemcel (EDIT-301) / Editas Medicine
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 () - Dec 7, 2024 - Abstract #ASH2024ASH_9484;
P1/2
After plerixafor and filgrastim mobilization, autologous CD34+ hematopoietic stem and progenitor cells are collected by apheresis and edited at the HBG1 and HBG2 promoters...The safety profile of reni-cel was consistent with myeloablative conditioning with busulfan...Additionally, patients have been transfusion-independent for up to 14.5 months. These findings, which include additional outcomes, build on clinical evidence to support the continued investigation of reni-cel in the EdiThal clinical trial.
- renizgamglogene autogedtemcel (EDIT-301) / Editas Medicine
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 (Halls G-H (San Diego Convention Center)) - Nov 6, 2024 - Abstract #ASH2024ASH_4393;
P1/2
After plerixafor mobilization, autologous CD34+ hematopoietic stem and progenitor cells are collected by apheresis and edited at the HBG1/2 promoters...The safety profile of reni-cel was consistent with myeloablative conditioning with busulfan...The data also demonstrate an early increase in the percentage of F-cells, improvements in markers of hemolysis, sustained high levels of editing, resolution of VOEs, and a favorable safety profile, with successful engraftment in all patients. These findings, which are based on a larger cohort of patients and additional outcomes, build on clinical evidence that support the continued investigation of reni-cel in the RUBY trial.
- | renizgamglogene autogedtemcel (EDIT-301) / Editas Medicine
Enrollment closed: A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY) (clinicaltrials.gov) - Oct 7, 2024
P1/2, N=45, Active, not recruiting, Sponsor: Editas Medicine, Inc.
These findings, which are based on a larger cohort of patients and additional outcomes, build on clinical evidence that support the continued investigation of reni-cel in the RUBY trial. Recruiting --> Active, not recruiting
- |||||| renizgamglogene autogedtemcel (EDIT-301) / Editas Medicine
Enrollment open: EDIT-301-LTFU-001: A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent ?-Thalassemia Who Received EDIT-301 (clinicaltrials.gov) - Sep 19, 2024
P=N/A, N=54, Enrolling by invitation, Sponsor: Editas Medicine, Inc.
Recruiting --> Active, not recruiting Not yet recruiting --> Enrolling by invitation
- |||||| renizgamglogene autogedtemcel (EDIT-301) / Editas Medicine
New trial: EDIT-301-LTFU-001: A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent ?-Thalassemia Who Received EDIT-301 (clinicaltrials.gov) - Apr 11, 2024
P=N/A, N=54, Not yet recruiting, Sponsor: Editas Medicine, Inc.
- EDIT-301 / Editas Medicine
AsCas12a Gene Editing of HBG1/2 Promoters with Edit-301 Results in Early and Sustained Normalization of Hemoglobin and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia () - Dec 11, 2023 - Abstract #TCTASTCTCIBMTR2024TCT_ASTCT_CIBMTR_982;
P1/2
Not yet recruiting --> Enrolling by invitation After pharmacokinetically adjusted myeloablative busulfan conditioning, pts received a single infusion of EDIT-301 (?3
- EDIT-301 / Editas Medicine
AsCas12a Gene Editing of HBG1/2 Promoters with EDIT-301 Results in Rapid and Sustained Normalization of Hemoglobin and Increased Fetal Hemoglobin in Patients with Severe Sickle Cell Disease ... (SDCC - Halls G-H) - Nov 3, 2023 - Abstract #ASH2023ASH_6461;
P1/2
In addition, there were improvements in key markers of hemolysis (SCD) and a favorable safety profile in EDIT-301-treated patients. EDIT-301 treatment showed promising results for the first clinical use of AsCas12a in both SCD and TDT patients after gene editing of the ?-globin gene ( HBG1/2 ) promoters.
- |||| EDIT-301 / Editas Medicine
Clinical: Congrats to @editasmed team @RabiHannaMD @hfrangoul et al exciting new results with EDIT-301 in Ruby trial for patients with #sicklecelldisease . Delighted to be part of this important study. @ColumbiaMed. (Twitter) - Jun 10, 2023
- ||| EDIT-301 / Editas Medicine
Great presentation by Rabi Hanna reporting the initial results of EDIT-301 at #EHA2023 #Frankfurt (Twitter) - Jun 10, 2023
- EDIT-301 / Editas Medicine
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 (Harmony 1) - May 12, 2023 - Abstract #EHA2023EHA_2407;
P1/2
These preliminary data demonstrated clinical proof of concept and are promising for the first clinical use of AsCas12a-based gene editing of the globin gene ( HBG1/HBG2 ) promoters, thereby supporting further investigation of EDIT-301 in the RUBY clinical trial. Updated data will be presented.
- | EDIT-301 / Editas Medicine
No #ASH22 abstract on $EDIT Edit-301 (Twitter) - Nov 3, 2022
- renizgamglogene autogedtemcel (EDIT-301) / Editas Medicine
New P1/2 trial: EdiThal: EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT) (clinicaltrials.gov) - Jul 6, 2022
P1/2, N=6, Recruiting, Sponsor: Editas Medicine, Inc.
- ||||| EDIT-301 / Editas Medicine
FDA event, IND: Editas Medicine Announces FDA Clearance of Investigational New Drug (IND) Application for EDIT-301 for the Treatment of Transfusion-Dependent Beta Thalassemia https://t.co/a51NT4LhGY (Twitter) - Dec 20, 2021
- EDIT-301 / Editas Medicine
Preclinical Development of EDIT301, an Autologous Cell Therapy Comprising AsCas12a-RNP Modified Mobilized Peripheral Blood-CD34+ Cells for the Potential Treatment of Transfusion Dependent Beta Thalassemia (GWCC - Hall B5, Level 1) - Nov 5, 2021 - Abstract #ASH2021ASH_3449;
These data support that a single administration of EDIT-301 may have the potential to safely and effectively reverse dyserythropoiesis and ameliorate anemia in individuals with TDT long-term. Clinical studies to demonstrate the safety and efficacy of EDIT-301 in the treatment of TDT are currently being planned.
- Zarzio (filgrastim biosimilar) / Novartis, EDIT-301 / Editas Medicine
[VIRTUAL] PRECLINICAL DATA SUPPORTING THE INITIATION OF THE EDIT-301 PHASE I RUBY CLINICAL TRIAL FOR THE POTENTIAL TREATMENT OF SICKLE CELL DISEASE () - May 13, 2021 - Abstract #EHA2021EHA_537;
In vivo, edited CD34+ cells led to polyclonal engraftment and reconstituted all blood cell lineages at levels comparable to unedited cells. These results support the dosing of EDIT301 in the upcoming RUBY trial, a phase 1/2 study of patients with severe SCD.
- || renizgamglogene autogedtemcel (EDIT-301) / Editas Medicine
Enrollment open: A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY) (clinicaltrials.gov) - Apr 29, 2021
P1/2, N=40, Recruiting, Sponsor: Editas Medicine, Inc.
These results support the dosing of EDIT301 in the upcoming RUBY trial, a phase 1/2 study of patients with severe SCD. Active, not recruiting --> Recruiting
- || renizgamglogene autogedtemcel (EDIT-301) / Editas Medicine
New P1/2 trial: A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY) (clinicaltrials.gov) - Apr 20, 2021
P1/2, N=40, Active, not recruiting, Sponsor: Editas Medicine, Inc.
- EDIT-301 / Editas Medicine
[VIRTUAL] Robust Pre-Clinical Results and Large-Scale Manufacturing Process for Edit-301: An Autologous Cell Therapy for the Potential Treatment of SCD (Poster Hall (Virtual Meeting)) - Nov 5, 2020 - Abstract #ASH2020ASH_1354;
Finally, a robust large-scale manufacturing process has been developed to supply material for the clinical setting. Based on these results, we are completing the activities required to assess EDIT-301 in the clinic as treatment for SCD.
- EDIT-301 / Editas Medicine
[VIRTUAL] EDIT-301: AN AUTOLOGOUS CELL THERAPY TO PROMOTE FETAL HEMOGLOBIN EXPRESSION FOR THE POTENTIAL TREATMENT OF SICKLE CELL DISEASE () - May 16, 2020 - Abstract #EHA2020EHA_557;
These cells readily engrafted and reconstituted all blood cell lineages at levels comparable to unedited cells. Production has been scaled up from research to clinical scale in preparation for progressing EDIT-301 towards the clinic.
- EDIT-301 / Editas Medicine
EDIT-301: An Experimental Autologous Cell Therapy Comprising Cas12a-RNP Modified mPB-CD34+ Cells for the Potential Treatment of SCD (Hall B, Level 2 (Orange County Convention Center)) - Nov 7, 2019 - Abstract #ASH2019ASH_6393;
These edited CD34+ cells led to long-term polyclonal multilineage engraftment and therapeutically meaningful levels of 40-50% HbF in vivo . Based on these results, IND-enabling activities have been initiated for EDIT-301: an experimental autologous cell therapy comprising Cas12a-RNP modified mPB-CD34+ cells for the potential treatment of SCD.